Trial Outcomes & Findings for Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure (NCT NCT02105766)

Last Updated: 2025-03-11

Results Overview

Number of patients who have sustained donor type hemoglobin at one year post transplant. Sustained donor type hemoglobin is based on hemoglobin electrophoresis for patients with SCD and transfusion independence for patients with beta-thalassemia

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

56 participants

Primary outcome timeframe

1 year

Results posted on

2025-03-11

Participant Flow

Participant milestones

Participant milestones
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor Participants received filgrastim to mobilize peripheral blood stem cells for apheresis collection. Collected stem cells of donor will then be infused to HLA matched sibling. Filgrastim: mobilize peripheral blood stem cells for apheresis collection
Overall Study STARTED	11	18	27
Overall Study COMPLETED	7	16	27
Overall Study NOT COMPLETED	4	2	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant
Overall Study Physician Decision	1	1
Overall Study Lost to Follow-up	2	0
Overall Study Death	1	1

Baseline Characteristics

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor n=27 Participants Participants received filgrastim to mobilize peripheral blood stem cells for apheresis collection. Collected stem cells of donor will then be infused to HLA matched sibling. Filgrastim: mobilize peripheral blood stem cells for apheresis collection	Total n=56 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants	8 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	11 Participants n=5 Participants	15 Participants n=7 Participants	22 Participants n=5 Participants	48 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Sex: Female, Male Female	11 Participants n=5 Participants	5 Participants n=7 Participants	8 Participants n=5 Participants	24 Participants n=4 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	13 Participants n=7 Participants	19 Participants n=5 Participants	32 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=5 Participants	17 Participants n=7 Participants	25 Participants n=5 Participants	52 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	4 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants	4 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	10 Participants n=5 Participants	16 Participants n=7 Participants	23 Participants n=5 Participants	49 Participants n=4 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants
Region of Enrollment United States	11 participants n=5 Participants	18 participants n=7 Participants	27 participants n=5 Participants	56 participants n=4 Participants

PRIMARY outcome

Timeframe: 1 year

Population: Analysis is intended per protocol for cohort 1 participants that received a stem cell transplant (female participants with male donors)

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Number of Patients Who Have Sustained Donor Type Hemoglobin at One Year Post Transplant	10 Participants	—

PRIMARY outcome

Timeframe: 2 years

Population: Analysis is intended per protocol to include those participants from cohort 2 (patients with pre-existing antibody to donor red cells)

Number of participants with donor red cells at 2 years post stem cell transplant. Number of participants with donor red cells is detected by hemoglobin electrophoresis or donor type red cell antigen, and reticulocyte count ≥30 k/uL at 2 years post-transplant.

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=18 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Number of Participants With Donor Red Cells at 2 Years Post Stem Cell Transplant	16 Participants	—

SECONDARY outcome

Timeframe: Day 0 up to Day 1

Mean CD34+ cell dose, filgrastim mobilized peripheral blood hematopoietic cells, unselected

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Mean CD34+ Cell Dose	19.08 x10^6 cells/kg Standard Deviation 8.38	14.98 x10^6 cells/kg Standard Deviation 5.95

SECONDARY outcome

Timeframe: Day 0 up to Day 1

Mean CD3+ cell dose, filgrastim mobilized peripheral blood hematopoietic cells, unselected

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Mean CD3+ Cell Dose	3.12 x10^8 cells/kg Standard Deviation 1.37	3.29 x10^8 cells/kg Standard Deviation 1.57

SECONDARY outcome

Timeframe: day 30, day 60 , day 100, 1 year and 2 year

Population: Analysis includes those participants who have sustained engraftment or did not experience graft failure.

Median Percent of donor T-cells and myeloid chimerism. Leukocytes are selected by magnetic beads for CD3 (T-cells) and CD14/15 (myeloid cells), then microsatellite PCR analyses are performed to obtain donor chimerism percent.

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=10 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=16 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Median Percent of Donor T-cells and Myeloid Chimerism donor T-cells Year 2	74.5 percentage of donor cells Interval 53.0 to 84.0	62 percentage of donor cells Interval 27.0 to 94.0
Median Percent of Donor T-cells and Myeloid Chimerism myeloid chimerism D30	100 percentage of donor cells Interval 80.0 to 100.0	99.5 percentage of donor cells Interval 68.0 to 100.0
Median Percent of Donor T-cells and Myeloid Chimerism myeloid chimerism D60	100 percentage of donor cells Interval 54.0 to 100.0	100 percentage of donor cells Interval 29.0 to 100.0
Median Percent of Donor T-cells and Myeloid Chimerism myeloid chimerism D100	100 percentage of donor cells Interval 42.0 to 100.0	100 percentage of donor cells Interval 16.0 to 100.0
Median Percent of Donor T-cells and Myeloid Chimerism myeloid chimerism Year 1	100 percentage of donor cells Interval 47.0 to 100.0	100 percentage of donor cells Interval 54.0 to 100.0
Median Percent of Donor T-cells and Myeloid Chimerism myeloid chimerism Year 2	100 percentage of donor cells Interval 44.0 to 100.0	99 percentage of donor cells Interval 45.0 to 100.0
Median Percent of Donor T-cells and Myeloid Chimerism donor T-cells D30	65.5 percentage of donor cells Interval 16.0 to 94.0	25 percentage of donor cells Interval 1.0 to 85.0
Median Percent of Donor T-cells and Myeloid Chimerism donor T-cells D60	39 percentage of donor cells Interval 2.0 to 86.0	23 percentage of donor cells Interval 1.0 to 100.0
Median Percent of Donor T-cells and Myeloid Chimerism donor T-cells D100	34 percentage of donor cells Interval 0.0 to 91.0	22.5 percentage of donor cells Interval 2.0 to 96.0
Median Percent of Donor T-cells and Myeloid Chimerism donor T-cells Year 1	70 percentage of donor cells Interval 29.0 to 78.0	43 percentage of donor cells Interval 24.0 to 100.0

SECONDARY outcome

Timeframe: Up to Day 100

Population: Analysis includes those participants who have sustained engraftment or did not experience graft failure.

Number of participants who developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV as defined by CIMBTR criteria for Organ Stages of Acute GVHD. Grades are defined as: Grade I: Skin = Maculopapular rash\< 25% of body surface area (BSA); Liver = Total Bilirubin 2-3 mg/dL; Lower GI = stool output/day is 500-999 mL/day. Grade II: Skin = rash on 25-50 percent body surface area; Liver = Total Bilirubin 3.1-6.0 mg/dL; Lower GI = Diarrhea 1001-1500 mL/day. Grade III: Skin = Rash on \>50% of body surface; Liver = Total Bilirubin 6.1 - 15.0 mg/dL; Lower GI = Diarrhea \> 1500 mL/day. Grade IV: Skin = Generalized erythroderma plus bullous formation; Liver = Total Bilirubin \>15 mg/dL; Lower GI = Severe abdominal pain with or without ileus. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=10 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=16 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Number of Participants Who Developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV Grade I	0 Participants	0 Participants
Number of Participants Who Developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV Grade II	0 Participants	1 Participants
Number of Participants Who Developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV Grade III	1 Participants	0 Participants
Number of Participants Who Developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV Grade IV	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Day 100 up to 2 years

Population: Analysis includes those participants who have sustained engraftment or did not experience graft failure.

Number of Participants Who Developed Moderate or Severe Chronic Graft vs Host Disease (GVHD) up to 5 years. Moderate chronic GVHD involves EITHER 3 organs/sites with no clinically significant functional impairment OR a less than or equal to 1 organ/site with clinically significant functional impairment, but no major disability. Severe GVHD is associated with a major disability caused by chronic GVHD.

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=10 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=16 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Number of Participants Who Developed Moderate or Severe Chronic Graft vs Host Disease (GVHD) Moderate	0 Participants	0 Participants
Number of Participants Who Developed Moderate or Severe Chronic Graft vs Host Disease (GVHD) Severe	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 2 years

Number of participants that experienced graft failure or graft rejection, or red cell aplasia at 2 years after transplant. Graft failure or graft rejection is defined as \<5% donor cells in both CD3 and myeloid chimerism. Red cell aplasia is defined as reticulocyte \<30 k/uL and requiring red cell transfusions.

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Number of Participants That Experienced Graft Failure or Graft Rejection, or Red Cell Aplasia at 2 Years After Transplant graft failure	1 Participants	0 Participants
Number of Participants That Experienced Graft Failure or Graft Rejection, or Red Cell Aplasia at 2 Years After Transplant red cell aplasia	0 Participants	2 Participants

SECONDARY outcome

Timeframe: Up to 2 years

Number of Participants That Experienced Regimen Failure. Regimen failure is defined as those participants that experienced grade 3 or higher acute GVHD, moderate/severe chronic GVHD, graft failure/rejection, or red cell aplasia. Together any one of these count toward the combined endpoint of regimen failure.

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Number of Participants That Experienced Regimen Failure	2 Participants	2 Participants

SECONDARY outcome

Timeframe: Up to 2 years

Number of participants that experienced transplant-related mortality. Transplant-related mortality is defined as death that is at least possibly related to the transplant (GVHD, toxicity, infection, other causes).

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Number of Participants That Experienced Transplant-related Mortality	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 2 years

Percentage of participant with disease-free survival following stem cell transplant. Disease-free survival is defined as alive and free acute complications related to sickle cell disease.

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Percentage of Participant With Disease-free Survival Following Stem Cell Transplant	9 Participants	16 Participants

SECONDARY outcome

Timeframe: Up to 2 years

Percentage of Participant Overall Survival up to year 2 following stem cell transplant. Overall survival is defined as being alive following stem cell transplant.

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Percentage of Participant Overall Survival Following Stem Cell Transplant	10 Participants	17 Participants

SECONDARY outcome

Timeframe: Up to Day 100

Median Day to Neutrophil recovery. Neutrophil recovery is defined as the first of three consecutive days of neutrophil count \>0.5 x 10\^9 cells/uL.

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Median Day to Neutrophil Recovery	22 days Interval 17.0 to 67.0	20.5 days Interval 8.0 to 25.0

SECONDARY outcome

Timeframe: Up to Day 120

Median Days to Platelet Recovery. Platelet recovery is defined as count \>50 cells/uL and 7 days from the last platelet transfusion.

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Median Days to Platelet Recovery	21.5 days Interval 16.0 to 69.0	22.5 days Interval 10.0 to 112.0

SECONDARY outcome

Timeframe: Up to 2 years

Median Days to Red Cell Recovery. Red cell recovery defined as days to recovery of reticulocyte count .30 k/uL, detection of donor red cells, transfusion independence.

Outcome measures

Outcome measures
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 Participants Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 Participants Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Median Days to Red Cell Recovery	22 days Interval 15.0 to 75.0	19 days Interval 8.0 to 195.0

Adverse Events

Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor

Serious events: 9 serious events

Other events: 11 other events

Deaths: 1 deaths

Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant

Serious events: 13 serious events

Other events: 18 other events

Deaths: 1 deaths

Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Matthew Hsieh M.D.

National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI)

Phone: 301.402.7687

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Serious adverse events
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 participants at risk Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 participants at risk Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor n=27 participants at risk Participants received filgrastim to mobilize peripheral blood stem cells for apheresis collection. Collected stem cells of donor will then be infused to HLA matched sibling. Filgrastim: mobilize peripheral blood stem cells for apheresis collection
Metabolism and nutrition disorders Anorexia	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Musculoskeletal and connective tissue disorders Arthralgia	18.2% 2/11 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Musculoskeletal and connective tissue disorders Avascular necrosis	18.2% 2/11 • Number of events 5 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Bacteremia	27.3% 3/11 • Number of events 6 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Clostridioides difficile colitis	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Catheter related infection	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Cytomegalovirus (CMV) Reactivation	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	16.7% 3/18 • Number of events 3 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Diarrhea	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	16.7% 3/18 • Number of events 3 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Nervous system disorders Dizziness	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	11.1% 2/18 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Respiratory, thoracic and mediastinal disorders Dyspnea	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Epstein-Barr virus (EBV) viremia	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Endocrine disorders Micro Papillary Thyroid Carcinoma	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Enterocolitis infectious	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	11.1% 2/18 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
General disorders Fatigue	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
General disorders Fever	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	11.1% 2/18 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Gastroparesis	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Herpes simplex reactivation	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Metabolism and nutrition disorders Hyperkalemia	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Endocrine disorders Hyperthyroidism	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 4 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Investigations Hypokalemia	9.1% 1/11 • Number of events 7 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Typhoid fever	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Malaria	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Blood and lymphatic system disorders Neutropenia	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Musculoskeletal and connective tissue disorders Joint effusion	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Lung infection	27.3% 3/11 • Number of events 3 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Meningitis	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Nausea	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	3.7% 1/27 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Musculoskeletal and connective tissue disorders Non-cardiac chest pain	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Musculoskeletal and connective tissue disorders Procedural pain	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Musculoskeletal and connective tissue disorders Pain	18.2% 2/11 • Number of events 11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	33.3% 6/18 • Number of events 8 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	3.7% 1/27 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Musculoskeletal and connective tissue disorders Vaso-occlusive pain crisis	18.2% 2/11 • Number of events 3 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	16.7% 3/18 • Number of events 5 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Psychiatric disorders Psychosis	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Nervous system disorders Reversible posterior leukoencephalopathy syndrome	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Sepsis	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Skin and subcutaneous tissue disorders Furunculosis	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Skin infection	18.2% 2/11 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Respiratory, thoracic and mediastinal disorders Sleep apnea	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Surgical and medical procedures Liver biopsy	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Surgical and medical procedures Plasma exchange	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Surgical and medical procedures Teeth extractions	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.

Other adverse events
Measure	Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor n=11 participants at risk Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant n=18 participants at risk Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0. Alemtuzumab: Immunosuppressant Sirolimus: Immunosuppressant Cyclophosphamide: Immunosuppressant Pentostatin: Immunosuppressant Radiotherapy: Immunosuppressant and myelosuppressant	Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor n=27 participants at risk Participants received filgrastim to mobilize peripheral blood stem cells for apheresis collection. Collected stem cells of donor will then be infused to HLA matched sibling. Filgrastim: mobilize peripheral blood stem cells for apheresis collection
Gastrointestinal disorders Gastroparesis	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Colitis	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Blood and lymphatic system disorders Acute GVHD	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Blood and lymphatic system disorders Anemia	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	27.8% 5/18 • Number of events 8 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Blood and lymphatic system disorders CD4 lymphocytes decreased	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Constipation	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Blood and lymphatic system disorders Febrile neutropenia	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	16.7% 3/18 • Number of events 5 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Diarrhea	27.3% 3/11 • Number of events 3 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Blood and lymphatic system disorders Hemolysis	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Blood and lymphatic system disorders Lymphocyte count decreased	81.8% 9/11 • Number of events 16 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	55.6% 10/18 • Number of events 25 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Blood and lymphatic system disorders Neutrophil count decreased	72.7% 8/11 • Number of events 15 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	44.4% 8/18 • Number of events 28 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Blood and lymphatic system disorders Platelet count decreased	54.5% 6/11 • Number of events 9 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	27.8% 5/18 • Number of events 11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Blood and lymphatic system disorders White blood cell decreased	45.5% 5/11 • Number of events 6 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	11.1% 2/18 • Number of events 4 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Cardiac disorders Hypertension	18.2% 2/11 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	22.2% 4/18 • Number of events 4 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Cardiac disorders Sinus tachycardia	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Eye disorders Cataract	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Eye disorders Eye pain	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Eye disorders Hemianopsia	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Anal fistula	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Mucositis oral	18.2% 2/11 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Nausea	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Toothache	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Upper gastrointestinal hemorrhage	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Gastrointestinal disorders Vomiting	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
General disorders Agitation	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
General disorders Chills	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
General disorders Infusion related reaction	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
General disorders Nausea	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Musculoskeletal and connective tissue disorders Non-cardiac chest pain	18.2% 2/11 • Number of events 7 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Musculoskeletal and connective tissue disorders Pain	36.4% 4/11 • Number of events 6 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	33.3% 6/18 • Number of events 13 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Immune system disorders Cytokine release syndrome	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Immune system disorders Hypogammaglobulinemia	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Anorectal infection	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Catheter related infection	18.2% 2/11 • Number of events 2 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Cytomegalovirus (CMV) Reactivation	27.3% 3/11 • Number of events 3 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	22.2% 4/18 • Number of events 6 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Endocarditis infective	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations BK Virus Infection	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Methicillin-resistant Staphylococcus aureus (MRSA) infection	9.1% 1/11 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/18 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Nasal vestibulitis	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	5.6% 1/18 • Number of events 1 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.
Infections and infestations Lung infection	0.00% 0/11 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	16.7% 3/18 • Number of events 3 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.	0.00% 0/27 • 5 years Information on AEs will be solicited from subjects through questions from study personnel and/or information volunteered by the subject. Grade 3 or higher adverse event recording will start at the time of conditioning regimen, will be followed until satisfactory resolution, through final study visit.