Trial Outcomes & Findings for Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders (NCT NCT00919503)

NCT ID: NCT00919503

Last Updated: 2021-08-13

Results Overview

Number of patients engrafted (\>5% donor CD3+ peripheral blood chimerisms) at 1 year following transplant

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

98 participants

Primary outcome timeframe

1 year following transplant

Results posted on

2021-08-13

Participant Flow

Participant milestones

Participant milestones
Measure	Regimen A (PBSCT and BMT) CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Overall Study STARTED	84	14
Overall Study COMPLETED	84	14
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Regimen A (PBSCT and BMT) n=84 Participants CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=14 Participants CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV	Total n=98 Participants Total of all reporting groups
Age, Categorical <=18 years	70 Participants n=93 Participants	13 Participants n=4 Participants	83 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	14 Participants n=93 Participants	1 Participants n=4 Participants	15 Participants n=27 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Sex: Female, Male Female	28 Participants n=93 Participants	3 Participants n=4 Participants	31 Participants n=27 Participants
Sex: Female, Male Male	56 Participants n=93 Participants	11 Participants n=4 Participants	67 Participants n=27 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=93 Participants	3 Participants n=4 Participants	9 Participants n=27 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	77 Participants n=93 Participants	10 Participants n=4 Participants	87 Participants n=27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=93 Participants	1 Participants n=4 Participants	2 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	4 Participants n=93 Participants	2 Participants n=4 Participants	6 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) Black or African American	8 Participants n=93 Participants	1 Participants n=4 Participants	9 Participants n=27 Participants
Race (NIH/OMB) White	67 Participants n=93 Participants	9 Participants n=4 Participants	76 Participants n=27 Participants
Race (NIH/OMB) More than one race	4 Participants n=93 Participants	1 Participants n=4 Participants	5 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants
Region of Enrollment United States	84 participants n=93 Participants	14 participants n=4 Participants	98 participants n=27 Participants

PRIMARY outcome

Timeframe: 1 year following transplant

Population: Regimen B: Three patients expired without CD3+ chimerisms being performed, therefore could not be analyzed for primary efficacy

Number of patients engrafted (\>5% donor CD3+ peripheral blood chimerisms) at 1 year following transplant

Outcome measures

Outcome measures
Measure	Regimen A (PBSCT and BMT) n=84 Participants CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=11 Participants CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Preliminary Efficacy	83 Participants	10 Participants

SECONDARY outcome

Timeframe: 1 year following transplant

Number of patients who experienced non-relapse mortality by 1 year following transplant

Outcome measures

Outcome measures
Measure	Regimen A (PBSCT and BMT) n=84 Participants CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=14 Participants CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Non-relapse Mortality	2 Participants	5 Participants

SECONDARY outcome

Timeframe: Day 100 post transplant

Population: Regimen B: 2 patients expired too early for evaluation for acute GVHD and could not be evaluated for this outcome

Number of patients diagnosed with overall grade II-IV acute GVHD by Day 100 post transplant

Outcome measures

Outcome measures
Measure	Regimen A (PBSCT and BMT) n=84 Participants CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=12 Participants CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Number of Patients With Grade II-IV Acute Graft-versus-host Disease	44 Participants	8 Participants

SECONDARY outcome

Timeframe: 1 year following transplant

Population: Regimen B: 3 patients expired too early for evaluation for chronic GVHD and could not be evaluated for this outcome

Number of patients diagnosed with chronic GVHD and requiring systemic immunosuppression within 1 year following transplant

Outcome measures

Outcome measures
Measure	Regimen A (PBSCT and BMT) n=84 Participants CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=11 Participants CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Number of Patients With of Chronic Graft-versus-host Disease	29 Participants	5 Participants

SECONDARY outcome

Timeframe: Day 100 post transplant

Population: Regimen B: 3 patients expired without CD3+ chimerisms being performed and could not be evaluated for this outcome

Number of patients with peripheral blood donor chimerism for CD3 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant.

Outcome measures

Outcome measures
Measure	Regimen A (PBSCT and BMT) n=84 Participants CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=11 Participants CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Donor Chimerism CD3 at 100 Days Post Transplant Greater than equal to 95%	49 Participants	7 Participants
Donor Chimerism CD3 at 100 Days Post Transplant 50 - 94%	29 Participants	2 Participants
Donor Chimerism CD3 at 100 Days Post Transplant 5-49%	6 Participants	1 Participants
Donor Chimerism CD3 at 100 Days Post Transplant Less than 5%	0 Participants	1 Participants

SECONDARY outcome

Timeframe: 1 year following transplant

Population: Regimen B: 3 patients expired prior to disease response being evaluated and could not be evaluated for this outcome

Number of patients with no evidence of disease at one year following transplant

Outcome measures

Outcome measures
Measure	Regimen A (PBSCT and BMT) n=84 Participants CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=11 Participants CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Disease Response at One Year Following Hematopoietic Cell Transplantation	78 Participants	8 Participants

SECONDARY outcome

Timeframe: 1 year following transplant

Population: Regimen A: 11 patients did not have lymphocyte subsets drawn post-transplant and could not be evaluated for outcome; Regimen B: 3 patients expired prior to lymphocyte subsets being evaluated and 2 patients did not have lymphocyte subsets drawn post-transplant and could not be evaluated for outcome

Number of patients with immune reconstitution (defined by a normal range CD3) at 1 year post transplant

Outcome measures

Outcome measures
Measure	Regimen A (PBSCT and BMT) n=73 Participants CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=9 Participants CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Immune Reconstitution Following Hematopoietic Cell Transplantation	39 Participants	4 Participants

SECONDARY outcome

Timeframe: 100 days post transplant

Number of participants with clinically significant infections (bacterial, fungal, viral) requiring treatment within 100 days following transplant

Outcome measures

Outcome measures
Measure	Regimen A (PBSCT and BMT) n=84 Participants CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=14 Participants CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Number of Participants With Infections	57 Participants	9 Participants

SECONDARY outcome

Timeframe: 1 year following transplant

Number of patients alive at 1 year following transplant

Outcome measures

Outcome measures
Measure	Regimen A (PBSCT and BMT) n=84 Participants CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=14 Participants CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Overall Survival	80 Participants	9 Participants

SECONDARY outcome

Timeframe: 100 days post transplant

Population: Regimen B: 2 patients expired without CD33+ chimerisms being performed and could not be evaluated for this outcome

Number of patients with peripheral blood donor chimerism for CD33 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant.

Outcome measures

Outcome measures
Measure	Regimen A (PBSCT and BMT) n=84 Participants CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=12 Participants CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Donor Chimerism CD33 at Day 100 Post Transplant Greater than equal to 95%	72 Participants	7 Participants
Donor Chimerism CD33 at Day 100 Post Transplant 50-94%	8 Participants	2 Participants
Donor Chimerism CD33 at Day 100 Post Transplant 5-49%	4 Participants	2 Participants
Donor Chimerism CD33 at Day 100 Post Transplant Less than 5%	0 Participants	1 Participants

Adverse Events

Regimen A (PBSCT and BMT)

Serious events: 17 serious events

Other events: 53 other events

Deaths: 7 deaths

Regimen B (UBCT)

Serious events: 5 serious events

Other events: 10 other events

Deaths: 6 deaths

Serious adverse events

Other adverse events

Additional Information

Dr. Lauri Burroughs

Fred Hutchinson Cancer Research Center

Phone: 206-667-2396

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Regimen A (PBSCT and BMT) n=84 participants at risk CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV	Regimen B (UBCT) n=14 participants at risk CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
Gastrointestinal disorders Gastrointestinal Other: Pneumatosis	0.00% 0/84 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	7.1% 1/14 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Respiratory, thoracic and mediastinal disorders Pneumonitis	0.00% 0/84 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	7.1% 1/14 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Infections and infestations Sepsis with multiorgan failure	0.00% 0/84 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	14.3% 2/14 • Number of events 2 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Respiratory, thoracic and mediastinal disorders Apnea: Requiring intubation following BAL; attributed to underlying infection	0.00% 0/84 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	7.1% 1/14 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Immune system disorders Anaphylaxis reaction (rATG)	2.4% 2/84 • Number of events 2 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Cardiac disorders Cardiac Asystole following central venous catheter placement	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Vascular disorders Hypotension: requiring intervention	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Vascular disorders Hypotension: shock, pressors	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Gastrointestinal disorders Abdominal Pain	1.2% 1/84 • Number of events 2 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Gastrointestinal disorders Nausea	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Gastrointestinal disorders Diarrhea (associated with GVHD): >15ml/kg per day with abdominal pain	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Blood and lymphatic system disorders Febrile neutropenia	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
General disorders Fever (not neutropenic): attributed to rATG	2.4% 2/84 • Number of events 2 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
General disorders Fever (not neutropenic)	2.4% 2/84 • Number of events 2 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Blood and lymphatic system disorders Febrile neutropenia: attributed to rATG	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Blood and lymphatic system disorders Febrile Neutropenia: with life- threatening sepsis	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Infections and infestations Infections and infestations -Other, specify: parainfluenza or sinusitis	1.2% 1/84 • Number of events 2 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Infections and infestations Infections and infestations -Other, specify: CMV meningitis/encephalo	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Infections and infestations Infections and infestations -Other, specify: Clostridium septicum sepsis	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Respiratory, thoracic and mediastinal disorders Adult respiratory distress syndrome (ARDS)	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Renal and urinary disorders Acute kidney injury (reversible, requiring CRRT/dialysis)	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Respiratory, thoracic and mediastinal disorders Dyspnea	1.2% 1/84 • Number of events 1 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
Respiratory, thoracic and mediastinal disorders Hypoxia: Decreased O2 sat requiring support	4.8% 4/84 • Number of events 4 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).	0.00% 0/14 • Day 100 post initiation of conditioning therapy Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).