Trial Outcomes & Findings for Single vs Double Umbilical Cord Blood Transplants in Children With High Risk Leukemia and Myelodysplasia (BMT CTN 0501) (NCT NCT00412360)

NCT ID: NCT00412360

Last Updated: 2021-10-28

Results Overview

Overall survival is defined as survival of death from any cause.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 224 participants
• Primary outcome timeframe: 1 year post-randomization
• Results posted on: 2021-10-28

Participant Flow

Participant milestones

Measure	Single UCB Transplant Single Cord Blood Unit Transplantation: Unrelated donor, single cord blood unit	Double UCB Transplant Double Cord Blood Unit Transplantation: Unrelated donor, double cord blood unit
Overall Study STARTED	113	111
Overall Study COMPLETED	112	108
Overall Study NOT COMPLETED	1	3

Reasons for withdrawal

Measure	Single UCB Transplant Single Cord Blood Unit Transplantation: Unrelated donor, single cord blood unit	Double UCB Transplant Double Cord Blood Unit Transplantation: Unrelated donor, double cord blood unit
Overall Study Lost to Follow-up	0	1
Overall Study Not Transplanted	1	2

Baseline Characteristics

Participants with AML

Baseline characteristics by cohort

Measure	Single UCB Transplant n=113 Participants Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=111 Participants Double Umbilical Cord Blood Unit Transplantation	Total n=224 Participants Total of all reporting groups
Age, Continuous	10.4 years STANDARD_DEVIATION 5.1 • n=113 Participants	10.4 years STANDARD_DEVIATION 5.1 • n=111 Participants	10.4 years STANDARD_DEVIATION 5.1 • n=224 Participants
Sex: Female, Male Female	55 Participants n=113 Participants	41 Participants n=111 Participants	96 Participants n=224 Participants
Sex: Female, Male Male	58 Participants n=113 Participants	70 Participants n=111 Participants	128 Participants n=224 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	22 Participants n=113 Participants	20 Participants n=111 Participants	42 Participants n=224 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	88 Participants n=113 Participants	88 Participants n=111 Participants	176 Participants n=224 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=113 Participants	3 Participants n=111 Participants	6 Participants n=224 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=113 Participants	0 Participants n=111 Participants	1 Participants n=224 Participants
Race (NIH/OMB) Asian	4 Participants n=113 Participants	5 Participants n=111 Participants	9 Participants n=224 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=113 Participants	0 Participants n=111 Participants	0 Participants n=224 Participants
Race (NIH/OMB) Black or African American	13 Participants n=113 Participants	11 Participants n=111 Participants	24 Participants n=224 Participants
Race (NIH/OMB) White	80 Participants n=113 Participants	85 Participants n=111 Participants	165 Participants n=224 Participants
Race (NIH/OMB) More than one race	3 Participants n=113 Participants	3 Participants n=111 Participants	6 Participants n=224 Participants
Race (NIH/OMB) Unknown or Not Reported	12 Participants n=113 Participants	7 Participants n=111 Participants	19 Participants n=224 Participants
Primary Disease Acute Myelogenous Leukemia (AML)	39 Participants n=113 Participants	38 Participants n=111 Participants	77 Participants n=224 Participants
Primary Disease Acute Lymphoblastic Leukemia (ALL)	61 Participants n=113 Participants	58 Participants n=111 Participants	119 Participants n=224 Participants
Primary Disease Acute Biphenotypic Leukemia	6 Participants n=113 Participants	2 Participants n=111 Participants	8 Participants n=224 Participants
Primary Disease Acute Undifferentiated Leukemia	1 Participants n=113 Participants	0 Participants n=111 Participants	1 Participants n=224 Participants
Primary Disease Myelodysplastic Syndrome (MDS)	5 Participants n=113 Participants	13 Participants n=111 Participants	18 Participants n=224 Participants
Primary Disease Chronic Myelogenous Leukemia (CML)	1 Participants n=113 Participants	0 Participants n=111 Participants	1 Participants n=224 Participants
AML Disease Status First Complete Remission (CR)	17 Participants n=39 Participants • Participants with AML	14 Participants n=38 Participants • Participants with AML	31 Participants n=77 Participants • Participants with AML
AML Disease Status Second or Later CR	16 Participants n=39 Participants • Participants with AML	19 Participants n=38 Participants • Participants with AML	35 Participants n=77 Participants • Participants with AML
AML Disease Status First Relapse	1 Participants n=39 Participants • Participants with AML	3 Participants n=38 Participants • Participants with AML	4 Participants n=77 Participants • Participants with AML
AML Disease Status Morphologic CR before Complete-Blood-Count Recover	2 Participants n=39 Participants • Participants with AML	2 Participants n=38 Participants • Participants with AML	4 Participants n=77 Participants • Participants with AML
AML Disease Status Secondary or Therapy-related	3 Participants n=39 Participants • Participants with AML	0 Participants n=38 Participants • Participants with AML	3 Participants n=77 Participants • Participants with AML
ALL Disease Status First Complete Remission (CR)	19 Participants n=61 Participants • Participants with ALL	19 Participants n=58 Participants • Participants with ALL	38 Participants n=119 Participants • Participants with ALL
ALL Disease Status Second CR	29 Participants n=61 Participants • Participants with ALL	28 Participants n=58 Participants • Participants with ALL	57 Participants n=119 Participants • Participants with ALL
ALL Disease Status Subsequent CR	13 Participants n=61 Participants • Participants with ALL	10 Participants n=58 Participants • Participants with ALL	23 Participants n=119 Participants • Participants with ALL
ALL Disease Status Morphologic CR before Complete-Blood-Count Recover	0 Participants n=61 Participants • Participants with ALL	1 Participants n=58 Participants • Participants with ALL	1 Participants n=119 Participants • Participants with ALL
Acute Biphenotypic Leukemia Disease Status First Complete Remission (CR)	5 Participants n=6 Participants • Participants with Acute Biphenotypic Leukemia	1 Participants n=2 Participants • Participants with Acute Biphenotypic Leukemia	6 Participants n=8 Participants • Participants with Acute Biphenotypic Leukemia
Acute Biphenotypic Leukemia Disease Status Second CR	1 Participants n=6 Participants • Participants with Acute Biphenotypic Leukemia	1 Participants n=2 Participants • Participants with Acute Biphenotypic Leukemia	2 Participants n=8 Participants • Participants with Acute Biphenotypic Leukemia
MDS Disease Status Refractory Anemia	2 Participants n=5 Participants • Participants with MDS	0 Participants n=13 Participants • Participants with MDS	2 Participants n=18 Participants • Participants with MDS
MDS Disease Status Refractory Cytopenia with Multilineage Dysplasia	2 Participants n=5 Participants • Participants with MDS	3 Participants n=13 Participants • Participants with MDS	5 Participants n=18 Participants • Participants with MDS
MDS Disease Status Refractory Anemia with Excess Blasts 1 (RAEB1)	0 Participants n=5 Participants • Participants with MDS	3 Participants n=13 Participants • Participants with MDS	3 Participants n=18 Participants • Participants with MDS
MDS Disease Status Refractory Anemia with Excess Blasts 2 (RAEB2)	0 Participants n=5 Participants • Participants with MDS	4 Participants n=13 Participants • Participants with MDS	4 Participants n=18 Participants • Participants with MDS
MDS Disease Status Unclassified	1 Participants n=5 Participants • Participants with MDS	3 Participants n=13 Participants • Participants with MDS	4 Participants n=18 Participants • Participants with MDS
Karnofsky Performance Score 100%	58 Participants n=113 Participants	57 Participants n=111 Participants	115 Participants n=224 Participants
Karnofsky Performance Score 90%	38 Participants n=113 Participants	43 Participants n=111 Participants	81 Participants n=224 Participants
Karnofsky Performance Score 80%	13 Participants n=113 Participants	9 Participants n=111 Participants	22 Participants n=224 Participants
Karnofsky Performance Score 70%	4 Participants n=113 Participants	2 Participants n=111 Participants	6 Participants n=224 Participants
Recipient CMV Status Positive	58 Participants n=113 Participants	66 Participants n=111 Participants	124 Participants n=224 Participants
Recipient CMV Status Negative	51 Participants n=113 Participants	39 Participants n=111 Participants	90 Participants n=224 Participants
Recipient CMV Status Inconclusive	1 Participants n=113 Participants	1 Participants n=111 Participants	2 Participants n=224 Participants
Recipient CMV Status Unknown	3 Participants n=113 Participants	5 Participants n=111 Participants	8 Participants n=224 Participants
Weight at Infusion	39.6 kilograms STANDARD_DEVIATION 20.2 • n=113 Participants	38.7 kilograms STANDARD_DEVIATION 19.2 • n=111 Participants	39.15 kilograms STANDARD_DEVIATION 19.7 • n=224 Participants
Recipient to First Cord Blood Unit HLA Match 3/6	1 Participants n=112 Participants • Transplanted participants	2 Participants n=108 Participants • Transplanted participants	3 Participants n=220 Participants • Transplanted participants
Recipient to First Cord Blood Unit HLA Match 4/6	44 Participants n=112 Participants • Transplanted participants	33 Participants n=108 Participants • Transplanted participants	77 Participants n=220 Participants • Transplanted participants
Recipient to First Cord Blood Unit HLA Match 5/6	51 Participants n=112 Participants • Transplanted participants	59 Participants n=108 Participants • Transplanted participants	110 Participants n=220 Participants • Transplanted participants
Recipient to First Cord Blood Unit HLA Match 6/6	16 Participants n=112 Participants • Transplanted participants	14 Participants n=108 Participants • Transplanted participants	30 Participants n=220 Participants • Transplanted participants
Recipient to Second Cord Blood Unit HLA Match 3/6	—	1 Participants n=108 Participants • Double UCB transplant participants	1 Participants n=108 Participants • Double UCB transplant participants
Recipient to Second Cord Blood Unit HLA Match 4/6	—	43 Participants n=108 Participants • Double UCB transplant participants	43 Participants n=108 Participants • Double UCB transplant participants
Recipient to Second Cord Blood Unit HLA Match 5/6	—	43 Participants n=108 Participants • Double UCB transplant participants	43 Participants n=108 Participants • Double UCB transplant participants
Recipient to Second Cord Blood Unit HLA Match 6/6	—	21 Participants n=108 Participants • Double UCB transplant participants	21 Participants n=108 Participants • Double UCB transplant participants
Recipient to First Cord Blood Unit ABO Match Major Mismatch	30 Participants n=112 Participants • Transplanted participants	25 Participants n=108 Participants • Transplanted participants	55 Participants n=220 Participants • Transplanted participants
Recipient to First Cord Blood Unit ABO Match Minor Mismatch	24 Participants n=112 Participants • Transplanted participants	35 Participants n=108 Participants • Transplanted participants	59 Participants n=220 Participants • Transplanted participants
Recipient to First Cord Blood Unit ABO Match Bidirectional Mismatch	10 Participants n=112 Participants • Transplanted participants	8 Participants n=108 Participants • Transplanted participants	18 Participants n=220 Participants • Transplanted participants
Recipient to First Cord Blood Unit ABO Match No Mismatch	44 Participants n=112 Participants • Transplanted participants	36 Participants n=108 Participants • Transplanted participants	80 Participants n=220 Participants • Transplanted participants
Recipient to First Cord Blood Unit ABO Match Unknown	4 Participants n=112 Participants • Transplanted participants	4 Participants n=108 Participants • Transplanted participants	8 Participants n=220 Participants • Transplanted participants
Recipient to Second Cord Blood Unit ABO Match Major mismatch	—	31 Participants n=108 Participants • Double UCB transplant participants	31 Participants n=108 Participants • Double UCB transplant participants
Recipient to Second Cord Blood Unit ABO Match Minor mismatch	—	29 Participants n=108 Participants • Double UCB transplant participants	29 Participants n=108 Participants • Double UCB transplant participants
Recipient to Second Cord Blood Unit ABO Match Bidirectional mismatch	—	4 Participants n=108 Participants • Double UCB transplant participants	4 Participants n=108 Participants • Double UCB transplant participants
Recipient to Second Cord Blood Unit ABO Match No mismatch	—	38 Participants n=108 Participants • Double UCB transplant participants	38 Participants n=108 Participants • Double UCB transplant participants
Recipient to Second Cord Blood Unit ABO Match Unknown	—	6 Participants n=108 Participants • Double UCB transplant participants	6 Participants n=108 Participants • Double UCB transplant participants

PRIMARY outcome

Timeframe: 1 year post-randomization

Overall survival is defined as survival of death from any cause.

Outcome measures

Measure	Single UCB Transplant n=113 Participants Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=111 Participants Double Umbilical Cord Blood Unit Transplantation
Percentage of Participants With Overall Survival	73 percentage of participants Interval 63.0 to 80.0	65 percentage of participants Interval 56.0 to 74.0

SECONDARY outcome

Timeframe: 1 year post-randomization

Disease-free survival is defined as survival without relapse of the primary disease.

Outcome measures

Measure	Single UCB Transplant n=113 Participants Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=111 Participants Double Umbilical Cord Blood Unit Transplantation
Percentage of Participants With Disease-free Survival	70 percentage of participants Interval 60.0 to 77.0	64 percentage of participants Interval 54.0 to 72.0

SECONDARY outcome

Timeframe: Days 42 and 100

Population: Transplanted participants

Neutrophil engraftment is defined as achieving an absolute neutrophil count greater than 500x10^6/liter for three consecutive measurements on different days. The first of the three days will be designated the day of neutrophil engraftment. Platelet engraftment is defined as achieving platelet counts greater than 50,000/microliter for consecutive measurements over 7 days without requiring platelet transfusions. The first of the 7 days will be designated the day of platelet engraftment. Subjects must not have had platelet transfusions during the preceding 7 days.

Outcome measures

Measure	Single UCB Transplant n=112 Participants Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=108 Participants Double Umbilical Cord Blood Unit Transplantation
Percentage of Participants With Neutrophil and Platelet Engraftment Neutrophil Engraftment at Day 42	89 percentage of participants Interval 83.0 to 95.0	88 percentage of participants Interval 82.0 to 94.0
Percentage of Participants With Neutrophil and Platelet Engraftment Platelet Engraftment at Day 100	76 percentage of participants Interval 68.0 to 85.0	65 percentage of participants Interval 56.0 to 74.0

SECONDARY outcome

Timeframe: 2 years post-transplant

Population: Transplanted participants

Platelet engraftment is defined as achieving platelet counts greater than 50,000/microliter for consecutive measurements over 7 days without requiring platelet transfusions. The first of the 7 days will be designated the day of platelet engraftment. Subjects must not have had platelet transfusions during the preceding 7 days.

Outcome measures

Measure	Single UCB Transplant n=112 Participants Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=108 Participants Double Umbilical Cord Blood Unit Transplantation
Time to Neutrophil and Platelet Engraftment Neutrophil Engraftment	21 days Interval 11.0 to 62.0	23 days Interval 11.0 to 133.0
Time to Neutrophil and Platelet Engraftment Platelet Engraftment	58 days Interval 28.0 to 295.0	84 days Interval 22.0 to 716.0

SECONDARY outcome

Timeframe: Day 100 post-randomization

Population: Transplanted participants

Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995:

Skin stage:

0: No rash

1. Rash <25% of body surface area

2. Rash on 25-50% of body surface area

3. Rash on > 50% of body surface area

4. Generalized erythroderma with bullous formation

Liver stage (based on bilirubin level)*:

0: <2 mg/dL

1. 2-3 mg/dL

2. 3.01-6 mg/dL

3. 6.01-15.0 mg/dL

4. >15 mg/dL

GI stage*:

0: No diarrhea or diarrhea <500 mL/day

1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD

2. Diarrhea 1000-1499 mL/day

3. Diarrhea >1500 mL/day

4. Severe abdominal pain with or without ileus * If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1.

GVHD grade:

0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4

Outcome measures

Measure	Single UCB Transplant n=112 Participants Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=108 Participants Double Umbilical Cord Blood Unit Transplantation
Percentage of Participants With Acute Graft-versus-host Disease (GVHD) Acute GVHD Grade II-IV	57 percentage of participants Interval 48.0 to 67.0	56 percentage of participants Interval 47.0 to 65.0
Percentage of Participants With Acute Graft-versus-host Disease (GVHD) Acute GVHD Grade III-IV	13 percentage of participants Interval 7.0 to 20.0	23 percentage of participants Interval 15.0 to 31.0

SECONDARY outcome

Timeframe: 1 year post-randomization

Population: Transplanted participants

Incidences of chronic GVHD will be graded per Shulman et al. 1980. This reference categorizes chronic GVHD as either limited or extensive. For this outcome, participants developing either type are considered to have a chronic GVHD event.

Outcome measures

Measure	Single UCB Transplant n=112 Participants Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=108 Participants Double Umbilical Cord Blood Unit Transplantation
Percentage of Participants With Chronic GVHD Chronic GVHD	30 percentage of participants Interval 22.0 to 39.0	32 percentage of participants Interval 23.0 to 40.0
Percentage of Participants With Chronic GVHD Extensive Chronic GVHD	9 percentage of participants Interval 4.0 to 14.0	15 percentage of participants Interval 8.0 to 22.0

SECONDARY outcome

Timeframe: 2 years post-randomization

Population: Transplanted participants

Outcome measures

Measure	Single UCB Transplant n=112 Participants Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=108 Participants Double Umbilical Cord Blood Unit Transplantation
Number of Infections Per Participant 0	10 Participants	9 Participants
Number of Infections Per Participant 1	15 Participants	13 Participants
Number of Infections Per Participant 2	12 Participants	15 Participants
Number of Infections Per Participant 3	19 Participants	10 Participants
Number of Infections Per Participant 4	17 Participants	15 Participants
Number of Infections Per Participant 5	7 Participants	12 Participants
Number of Infections Per Participant 6-10	19 Participants	24 Participants
Number of Infections Per Participant More than 10	13 Participants	10 Participants

SECONDARY outcome

Timeframe: 1 year post-randomization

Population: Transplanted participants

Relapse is defined by either morphological or cytogenetic evidence of AML, ALL, CML, or MDS consistent with pre-transplant features. Testing for recurrent malignancy in the blood, marrow or other sites will be used to assess relapse after transplantation.

Outcome measures

Measure	Single UCB Transplant n=112 Participants Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=108 Participants Double Umbilical Cord Blood Unit Transplantation
Percentage of Participants With Relapse	12 percentage of participants Interval 6.0 to 18.0	14 percentage of participants Interval 7.0 to 21.0

SECONDARY outcome

Timeframe: 1 year post-randomization

Population: Transplanted participants

Treatment related mortality is defined as death without relapse of the primary disease.

Outcome measures

Measure	Single UCB Transplant n=112 Participants Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=108 Participants Double Umbilical Cord Blood Unit Transplantation
Percentage of Participants With Treatment-related Mortality	19 percentage of participants Interval 11.0 to 26.0	22 percentage of participants Interval 14.0 to 30.0

SECONDARY outcome

Timeframe: Day 100 post-transplant

Population: Transplanted participants

Outcome measures

Measure	Single UCB Transplant n=112 Participants Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=108 Participants Double Umbilical Cord Blood Unit Transplantation
Number of Participants With Engraftment Syndrome	11 Participants	7 Participants

Adverse Events

Single UCB Transplant

Serious events: 5 serious events

Other events: 0 other events

Deaths: 0 deaths

Double UCB Transplant

Serious events: 14 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Single UCB Transplant n=112 participants at risk Single Umbilical Cord Blood Unit Transplantation	Double UCB Transplant n=108 participants at risk Double Umbilical Cord Blood Unit Transplantation
Blood and lymphatic system disorders Hemolytic anemia	0.00% 0/112 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	1.9% 2/108 • Number of events 2 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Cardiac disorders Congestive heart failure	0.89% 1/112 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.93% 1/108 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Gastrointestinal disorders Abdominal pain	0.00% 0/112 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.93% 1/108 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Infections and infestations Pneumonia	0.00% 0/112 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.93% 1/108 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Metabolism and nutrition disorders Hyperglycemia	1.8% 2/112 • Number of events 2 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.00% 0/108 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Nervous system disorders Acute intracranial Hemorrage	0.00% 0/112 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.93% 1/108 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Respiratory, thoracic and mediastinal disorders Hypoxia	0.00% 0/112 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	1.9% 2/108 • Number of events 2 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Vascular disorders Hypotension	0.00% 0/112 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.93% 1/108 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Infections and infestations Septic shock	0.00% 0/112 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.93% 1/108 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Nervous system disorders Seizure/aspiration Pneumonia	0.89% 1/112 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.00% 0/108 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Respiratory, thoracic and mediastinal disorders Pulmonary Hemorrage	0.00% 0/112 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.93% 1/108 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Respiratory, thoracic and mediastinal disorders Aspiration	0.00% 0/112 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.93% 1/108 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Respiratory, thoracic and mediastinal disorders Aspiration Pneumonia	0.89% 1/112 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.00% 0/108 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Vascular disorders Hypertension	0.00% 0/112 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.93% 1/108 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
Vascular disorders Hematoma	0.00% 0/112 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.	0.93% 1/108 • Number of events 1 • 2 years post-transplant Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.

Other adverse events

Adverse event data not reported

Additional Information

Adam Mendizabal

The EMMES Corporation

Phone: 301-251-1161

Email: amendizabal@EMMES.com

Results disclosure agreements

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