Trial Outcomes & Findings for Combined T Cell Depleted Haploidentical Peripheral Blood Stem Cell and Unrelated Umbilical Cord Blood Transplantation in Patients With Hematologic Malignancies Using a Total Lymphoid Irradiation Based Preparative Regimen (NCT NCT02199041)

NCT ID: NCT02199041

Last Updated: 2018-02-07

Results Overview

Neutrophil engraftment is defined as absolute neutrophil count (ANC) recovery of ≥ 0.5 x 10\^9/L (500/mm\^3) for three consecutive laboratory values obtained on different days (derived from either donor). Date of engraftment is the date of the first of the three consecutive laboratory values. The number of patients engrafted by day +42 post-transplant is provided.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 24 participants
• Primary outcome timeframe: Until day 42 post-transplant
• Results posted on: 2018-02-07

Participant Flow

Twelve participants meeting eligibility criteria and 12 of their blood donors were enrolled at St. Jude Children's Research Hospital between July 2014 and January 2015.

The 12 enrolled blood donors did not undergo transplantation and are therefore not included in the results reported here.

Participant milestones

Measure	Treatment Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, granulocyte colony-stimulating factor (G-CSF), mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.
Overall Study STARTED	12
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Combined T Cell Depleted Haploidentical Peripheral Blood Stem Cell and Unrelated Umbilical Cord Blood Transplantation in Patients With Hematologic Malignancies Using a Total Lymphoid Irradiation Based Preparative Regimen

Baseline characteristics by cohort

Measure	Treatment n=12 Participants Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.
Age, Continuous	5.0 years n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Race/Ethnicity, Customized Ethnicity · NOS Spanish, Hispanic, Latino	1 Participants n=5 Participants
Race/Ethnicity, Customized Ethnicity · Non Spanish Speaking, Non Hispanic	8 Participants n=5 Participants
Race/Ethnicity, Customized Ethnicity · Puerto Rican	1 Participants n=5 Participants
Race/Ethnicity, Customized Ethnicity · South or Central American	2 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black	3 Participants n=5 Participants
Race/Ethnicity, Customized Race · Multiple Race (NOS)	1 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	8 Participants n=5 Participants
Diagnosis Acute Erythroleukemia, FAB M6, BM	1 Participants n=5 Participants
Diagnosis AL, Lymphoblastic, Pre-B, BM	2 Participants n=5 Participants
Diagnosis AL, Lymphoblastic, Pre-T Cell, BM	1 Participants n=5 Participants
Diagnosis AL, Megakaryoblastic, FAB M7, BM	2 Participants n=5 Participants
Diagnosis AL, Monocytic, FAB M5, BM	1 Participants n=5 Participants
Diagnosis AL, Myeloid, Minimal Differentiation, FAB M0, BM	1 Participants n=5 Participants
Diagnosis AL, Myeloid, NOS, BM	2 Participants n=5 Participants
Diagnosis AL, Myeloid, With Maturation, FAB M2, BM	1 Participants n=5 Participants
Diagnosis Lymphoma, Non-Hodgkin's, Anaplastic Large Cell	1 Participants n=5 Participants
Disease Status at HSCT CR1	2 Participants n=5 Participants
Disease Status at HSCT CR2	3 Participants n=5 Participants
Disease Status at HSCT CR3	2 Participants n=5 Participants
Disease Status at HSCT Refractory	4 Participants n=5 Participants
Disease Status at HSCT Relapse 2	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Until day 42 post-transplant

Neutrophil engraftment is defined as absolute neutrophil count (ANC) recovery of ≥ 0.5 x 10^9/L (500/mm^3) for three consecutive laboratory values obtained on different days (derived from either donor). Date of engraftment is the date of the first of the three consecutive laboratory values. The number of patients engrafted by day +42 post-transplant is provided.

Outcome measures

Measure

Treatment n=12 Participants Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Number of Participants With Neutrophil Engraftment

11 Participants

SECONDARY outcome

Timeframe: One year after transplantation

Relapse was evaluated using standard World Health Organization (WHO) criteria for each disease. The estimate of cumulative incidence of relapse will be estimated using Kalbfleisch-Prentice method. Relapse defined as the recurrence of original disease. Death is the competing risk event. The analysis will be implemented using Statistical Analysis System (SAS) macro (bmacro252-Excel2007\cin).

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced malignant relapse is provided

Outcome measures

Measure

Treatment n=12 Participants Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Number of Participants With Malignant Relapse

7 Participants

SECONDARY outcome

Timeframe: One year after transplantation

The Kaplan-Meier estimate of EFS with relapse, death due to any cause and graft failure as events along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007\kme) available in the Department of Biostatistics at St. Jude, where EFS = min (date of last follow-up, date of relapse, date of graft failure, date of death due to any cause) - date of transplant, and all participants surviving at the time of analysis without events will be censored. The number of participants who did not experience any of these events through one year post-transplant is given.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who did not experience any events defined above is provided.

Outcome measures

Measure

Treatment n=12 Participants Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Number of Participants With Event-free Survival (EFS)

4 Participants

SECONDARY outcome

Timeframe: One year after transplantation

The Kaplan-Meier estimate of OS with relapse, death due to any cause and graft failure as events along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007\kme) available in the Department of Biostatistics at St. Jude, where OS = min (date of last follow-up, date of death) - date of hematopoietic cell transplantation (HCT) and all participants surviving after 1 year post-transplant will be considered as censored.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who did not die at 1 year post-transplant is provided.

Outcome measures

Measure

Treatment n=12 Participants Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Number of Participants With Overall Survival (OS)

6 Participants

SECONDARY outcome

Timeframe: 100 days after transplantation

Cumulative incidence of acute GVHD was estimated using Kalbfleisch-Prentice method. Death is the competing risk event. SAS macro (bmacro252-Excel2007\cin) available at St. Jude was used for analysis. Severity of GVHD and stage were determined using the Clinical Oncology Group (COG) Stem Cell Committee Consensus Guidelines for establishing organ stage and overall grade of acute GVHD. Participants are graded on a scale from I to IV, with I being mild and IV being severe.

Overall Clinical Grade (based on the highest stage obtained):

Grade 0: No stage 1-4 of any organ. Grade I: Stage 1-2 skin and no liver or gut involvement. Grade II: Stage 3 skin, or Stage I liver involvement, or Stage 1 gastrointestinal (GI).

Grade III: Stage 0-3 skin, with Stage 2-3 liver, or Stage 2-3 GI. Grade IV: Stage 4 skin, liver or GI involvement.

Due to early close of study, a small number of patients were enrolled. The number of patients who experienced acute GVHD is provided.

Outcome measures

Measure	Treatment n=12 Participants Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.
Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT No Acute GVHD	8 Participants
Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT Grade I	0 Participants
Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT Grade III	2 Participants
Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT Grade IV	1 Participants
Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT Grade II	1 Participants

SECONDARY outcome

Timeframe: 100 days after transplantation

Cumulative incidence of acute and chronic GVHD was estimated using Kalbfleisch-Prentice method. Death is competing risk event. SAS macro (bmacro252-Excel2007\cin) available St. Jude was used for such analysis. Severity of chronic GVHD was evaluated using National Institutes of Health (NIH) Consensus Global Severity Scoring. Mild is considered a better outcome with severe being the worst.

Criteria for grading chronic GVHD:

Mild: 1-2 organs/sites, maximum organ score of 1, and lung score of 1. Moderate: 3 or more organs/sites and maximum organ score of 1 and lung score of 1, OR at least 1 organ/site and maximum organ score of 2 and lung score of 1. Severe: At least 1 organ/site and maximum organ score of 3 and lung score of 2-3.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced chronic GVHD is provided.

Outcome measures

Measure	Treatment n=12 Participants Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.
Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT No Chronic GVHD	11 Participants
Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT Mild	0 Participants
Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT Moderate	0 Participants
Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT Severe	1 Participants

SECONDARY outcome

Timeframe: 100 days after transplantation

The cumulative incidence of secondary graft failure will be estimated using the Kalbfleisch-Prentice method. Deaths due to toxicity and relapse before day 100 are the competing events.

Secondary graft failure or graft rejection will be defined as no evidence of donor chimerism by umbilical cord blood (UCB) and/or haploidentical donor (<10%), or too few cells to perform adequate chimerism analysis, in research participants with prior neutrophil engraftment.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced secondary graft failure is provided.

Outcome measures

Measure

Treatment n=12 Participants Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Number of Participants With Secondary Graft Failure

0 Participants

SECONDARY outcome

Timeframe: 100 days after transplantation

TRM is any death in remission and related to protocol therapy. The cumulative incidence of TRM was estimated using the Kalbfleisch-Prentice method. Deaths before day 100 because of other reasons are the completing events.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced TRM is provided.

Outcome measures

Measure

Treatment n=12 Participants Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Number of Participants With Transplant-related Mortality (TRM)

0 Participants

SECONDARY outcome

Timeframe: 100 days after transplantation

Any patient who had adverse events listed either as probable or definite in the first 100 days post-transplant are counted as transplant-related morbidity. The cumulative incidence of transplant-related morbidity will be estimated using the Kalbfleisch-Prentice method. Deaths before day 100 are the competing risk events.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced at least one-transplant-related morbidity is provided.

Outcome measures

Measure

Treatment n=12 Participants Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Number of Participants With Transplant-related Morbidity

12 Participants

Adverse Events

Treatment

Serious events: 5 serious events

Other events: 12 other events

Deaths: 6 deaths

Blood Donors

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Treatment n=12 participants at risk Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.	Blood Donors n=12 participants at risk Blood donors were enrolled on the study to provide cells for transplantation used in the Treatment Arm.
Cardiac disorders Hypotension	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Cardiac disorders Pericardial Effusion	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Blood and lymphatic system disorders Thrombotic Thrombocytopenic Purpura	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
General disorders Fever without Neutropenia	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Investigations Weight Loss	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Gastrointestinal disorders Diarrhea	8.3% 1/12 • Number of events 2 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Enterobacter Cloacae, Blood	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Fungal and Viral, Pneumonia	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Staphylococcus Aureus, Blood	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Pneumonia	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Pneumonia, Staph Aureus	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Nervous system disorders Seizure	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.

Other adverse events

Measure	Treatment n=12 participants at risk Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.	Blood Donors n=12 participants at risk Blood donors were enrolled on the study to provide cells for transplantation used in the Treatment Arm.
Immune system disorders Acute Infusion Reaction, Stem Cells	16.7% 2/12 • Number of events 2 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Immune system disorders Allergic Reaction, Platelet Transfusion	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Immune system disorders Allergy to Diary Product (Disorder)	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Cardiac disorders Hypertension	50.0% 6/12 • Number of events 8 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Cardiac disorders Left Ventricular Cardiac Dysfunction (Disorder)	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Cardiac disorders Pericardial Effusion	25.0% 3/12 • Number of events 4 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Blood and lymphatic system disorders Disseminated Intravascular Coagulation	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Investigations Weight Loss	16.7% 2/12 • Number of events 2 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Skin and subcutaneous tissue disorders Rash	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Skin and subcutaneous tissue disorders Rash, Skin	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Gastrointestinal disorders Colitis (Disorder)	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Gastrointestinal disorders Dehydration	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Gastrointestinal disorders Diarrhea	16.7% 2/12 • Number of events 2 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Gastrointestinal disorders Esophagitis	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Gastrointestinal disorders Hypernatremic Dehydration (Disorder)	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Gastrointestinal disorders Mucositis	16.7% 2/12 • Number of events 2 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Gastrointestinal disorders Nausea	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Gastrointestinal disorders Vomiting (Disorder)	16.7% 2/12 • Number of events 2 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Hepatobiliary disorders Veno-Occlusive Disease, Hepatic	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Cellulitis, Gastrostomy Site	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Fever without Neutropenia	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Febrile Neutropenia	100.0% 12/12 • Number of events 13 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Hepatitis	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Adenovirus, Respiratory Tract	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Adenovirus, Stool	33.3% 4/12 • Number of events 5 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, BK Virus, Blood	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, BK Virus, Urine	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, CMV, Respiratory/Nasopharynx	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Candida Albicans, Oral Mucosa	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Candida Lipolytica, Oral Mucosa	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Clostridium Difficile Colitis	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Clostridium Difficile, Stool	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Cytomegalovirus, Blood	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Due to Klebsiella, Bone Marrow Site	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Enterobacter Asburiae, Blood	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Enterobacter Cloacae, Blood	8.3% 1/12 • Number of events 2 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Enterobacter asburiae and Coagulase Negative Staphylococcus, Gastrostomy Wound Site	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Fungal, Liver	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Herpes Simplex Virus, Oral Mucosa	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Human Herpes Simplex Virus, Oral	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Human Herpes Virus 6, Blood	66.7% 8/12 • Number of events 8 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Human Herpes Virus 6, Cerebrospinal Fluid	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Klebsiella pneumoniae, Colon	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Malassezia Furfur, Blood	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Norovirus, Stool	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Pediococcus Pentosaceua	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Presumed Viral, Oral Mucosa	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Rotavirus, Stool	25.0% 3/12 • Number of events 6 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Infection, Staphylococcus Epidermidis, Blood	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Reactivation, Human Herpes Virus 6, Blood	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Hypoxia	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Infections and infestations Ulceration, Larynx	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Metabolism and nutrition disorders Hyperglycemia	16.7% 2/12 • Number of events 3 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Psychiatric disorders Anxiety	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Respiratory, thoracic and mediastinal disorders Adult Respiratory Distress Syndrome	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Renal and urinary disorders Acute renal failure	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Renal and urinary disorders Renal Insufficiency	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Renal and urinary disorders Renal, Acute Kidney Injury	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
Immune system disorders Engraftment Syndrome	16.7% 2/12 • Number of events 2 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
General disorders Pain, Abdomen	16.7% 2/12 • Number of events 2 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
General disorders Pain, Neuropathic, Legs and Feet, Bilateral	8.3% 1/12 • Number of events 1 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.	0.00% 0/12 • Adverse events were collected from participants beginning with the on-study date through one year post-transplantation. Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.

Additional Information

Brandon Triplett, MD

St. Jude Children's Research Hospital

Phone: 901-595-2766

Email: brandon.triplett@stjude.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place