Trial Outcomes & Findings for Umbilical Cord Blood Transplant for Children With Myeloid Hematological Malignancies (NCT NCT01247701)
NCT ID: NCT01247701
Last Updated: 2022-06-08
Results Overview
To determine the overall survival rate at 1 year after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
16 participants
Primary outcome timeframe
100 days, 1 year, and 3 years
Results posted on
2022-06-08
Participant Flow
Participant milestones
| Measure |
Umbilical Cord Blood Transplant
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
|
Overall Study
STARTED
|
16
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
10
|
Reasons for withdrawal
| Measure |
Umbilical Cord Blood Transplant
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
|
Overall Study
Death
|
2
|
|
Overall Study
Relapsed
|
8
|
Baseline Characteristics
Umbilical Cord Blood Transplant for Children With Myeloid Hematological Malignancies
Baseline characteristics by cohort
| Measure |
Umbilical Cord Blood Transplant
n=16 Participants
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
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Age, Continuous
|
1.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 100 days, 1 year, and 3 yearsPopulation: The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT) except the one participant who died 4 days after transplant.
To determine the overall survival rate at 1 year after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Outcome measures
| Measure |
Umbilical Cord Blood Transplant
n=15 Participants
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
|
Overall Survival at 100 Days, 1 Year, and 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
100 days
|
0.923 probability of overall survival
Interval 0.566 to 0.989
|
|
Overall Survival at 100 Days, 1 Year, and 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
1-Year
|
0.923 probability of overall survival
Interval 0.566 to 0.989
|
|
Overall Survival at 100 Days, 1 Year, and 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
3-Year
|
0.923 probability of overall survival
Interval 0.566 to 0.989
|
SECONDARY outcome
Timeframe: Day 100Population: The analysis included all participants who underwent Umbilical Cord Blood Transplant (UCBT) and were evaluable for acute GVHD. A participant was evaluable for acute GVHD if he/she engrafted and either completed 100 days observation after transplant or experienced acute GVHD.
Number of participants with acute GVHD graded by the method of Przepiorka et al, which evaluates skin involvement, lower and upper GI, and liver function (bilirubin), each being graded in stages from 0 to 4, where 0 means no acute GVHD, and 4 is the highest stage of acute GVHD.
Outcome measures
| Measure |
Umbilical Cord Blood Transplant
n=15 Participants
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
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Number of Participants With Severe Acute GVHD Grade III-IV
|
2 Participants
|
SECONDARY outcome
Timeframe: 1 yearPopulation: The analysis included all enrolled participants who underwent umbilical cord blood transplant (UCBT) and were evaluable for chronic GVHD. A participant was evaluable for chronic GVHD if he/she engrafted and survived or remained in the study for more than 100 days after transplant. However, if the participant(s) relapsed within or around 100 days, they would not be evaluable.
Number of participants with chronic GVHD graded by the method of Przepiorka et al, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3.
Outcome measures
| Measure |
Umbilical Cord Blood Transplant
n=10 Participants
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
|
Number of Participants With Chronic GvHD
|
1 Participants
|
SECONDARY outcome
Timeframe: 1 and 3 yearsPopulation: The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT) except the one participant who died 4 days after transplant.
To assess relapse rate at 1 and 3 years after transplant. Cumulative incidence of relapse was calculated from the date of umbilical cord blood transplant using the competing risk method as described in Gray(1988) with death prior to relapse as the competing risk. Participants still alive without a date of relapse were censored at the time of the last follow-up.
Outcome measures
| Measure |
Umbilical Cord Blood Transplant
n=15 Participants
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
|
Number of Participants With Relapse Rate After Transplant
1 year
|
53.3 percentage of participants
Interval 24.8 to 75.3
|
|
Number of Participants With Relapse Rate After Transplant
3 year
|
53.3 percentage of participants
Interval 24.8 to 75.3
|
SECONDARY outcome
Timeframe: 100 days, 6, 9, 12, 24 and 36 monthsPopulation: The analysis included all participants who underwent umbilical cord blood transplant (UCBT) except the one participant who died 4 days after transplant. If the participant(s) relapsed, they would not be evaluable for donor engraftment.
To evaluate donor engraftment at 100 days, 6, 9, 12, 24, and 36 months after transplant.
Outcome measures
| Measure |
Umbilical Cord Blood Transplant
n=12 Participants
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
|
Number of Participants With Donor Engraftment After Transplant.
100 days
|
11 Participants
|
|
Number of Participants With Donor Engraftment After Transplant.
6 months
|
6 Participants
|
|
Number of Participants With Donor Engraftment After Transplant.
9 months
|
6 Participants
|
|
Number of Participants With Donor Engraftment After Transplant.
12 months
|
6 Participants
|
|
Number of Participants With Donor Engraftment After Transplant.
24 months
|
6 Participants
|
|
Number of Participants With Donor Engraftment After Transplant.
36 months
|
3 Participants
|
SECONDARY outcome
Timeframe: Day 180Population: The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT) except the one participant who died 4 days after transplant.
Achievement of untransfused platelet count \> 20 x 10\^9/L on three consecutive days
Outcome measures
| Measure |
Umbilical Cord Blood Transplant
n=15 Participants
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
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Number of Participants With Platelet Engraftment
|
13 Participants
|
SECONDARY outcome
Timeframe: Day 42Population: The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT) except the one participant who died 4 days after transplant.
Achievement of absolute neutrophil count \> 0.5 x 10\^9/L on three consecutive days
Outcome measures
| Measure |
Umbilical Cord Blood Transplant
n=15 Participants
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
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Number of Participants With Neutrophil Engraftment
|
15 Participants
|
Adverse Events
Umbilical Cord Blood Transplant
Serious events: 11 serious events
Other events: 13 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Umbilical Cord Blood Transplant
n=16 participants at risk
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
|
Cardiac disorders
Cardiac arrest
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Infections and infestations
Catheter related infection
|
25.0%
4/16 • Number of events 5 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Nervous system disorders
Encephalopathy
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Infections and infestations
Endocarditis infective
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other, specify: VOD
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Infections and infestations
Infections and infestations - Other, specify: Adenovirus in plasma and adenovirus enteritis
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Infections and infestations
Infections and infestations - Other, specify: BK virus infection with hemorrhagic cystitis
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Infections and infestations
Infections and infestations - Other, specify: C.Difficile gastroenteritis
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Infections and infestations
Infections and infestations - Other,specify:Hemorrhagic cystitis due to BK virus uremia and viremia
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Infections and infestations
Infections and infestations - Other, specify: Sepsis without identified organism
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Gastrointestinal disorders
Intra-abdominal hemorrhage
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Infections and infestations
Lung infection
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Nervous system disorders
Nervous system disorders - Other, specify: Altered mental status
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
12.5%
2/16 • Number of events 2 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify: Pulmonary hemorrhage
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory,thoracic and mediastinal disorders - Other,specify:Respiratory distress after sedation
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Infections and infestations
Sepsis
|
12.5%
2/16 • Number of events 2 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
|
Vascular disorders
Vascular disorders - Other, specify: VOD
|
6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
|
Other adverse events
| Measure |
Umbilical Cord Blood Transplant
n=16 participants at risk
Busulfan,Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Busulfan dosing will be as follows: Patients \< 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \> 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Cyclophosphamide: Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: Fludarabine will be given IV daily over 1 hour for 3 days. Dosing will be as follows: for patients ≤ 10 kg: 1.3 mg/kg; for patients \> 10 kg: 40 mg/m\^2.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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Renal and urinary disorders
Acute kidney injury
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Investigations
Aspartate aminotransferase increased
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Investigations
Blood bilirubin increased
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12.5%
2/16 • Number of events 2 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Infections and infestations
Catheter related infection
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12.5%
2/16 • Number of events 2 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Renal and urinary disorders
Cystitis noninfective
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Metabolism and nutrition disorders
Hypermagnesemia
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Metabolism and nutrition disorders
Hypertriglyceridemia
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Metabolism and nutrition disorders
Hypocalcemia
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12.5%
2/16 • Number of events 3 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Metabolism and nutrition disorders
Hyponatremia
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Metabolism and nutrition disorders
Hypophosphatemia
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12.5%
2/16 • Number of events 3 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Infections and infestations
Infections and infestations - Other, specify: Adenovirus
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Infections and infestations
Infections and infestations -Other,specify: Hemorrhagic cystitis due to BK virus uremia and viremia
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Infections and infestations
Infections and infestations - Other, specify: Norovirus viral diarrheal disese
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Infections and infestations
Infections and infestations - Other, specify: Recurrent Clostridium Difficile colitis
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Gastrointestinal disorders
Mucositis oral
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12.5%
2/16 • Number of events 2 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Gastrointestinal disorders
Nausea
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Nervous system disorders
Nervous system disorders - Other, specify: Posterior reversible encephalopathy syndrome
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify: Respiratory distress
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6.2%
1/16 • Number of events 1 • We have recorded all grade 3 and 4 toxicities, except hematological toxicities, fever, abnormal glucose, ALT, GGT, and K levels from the day conditioning started until day 30 post-transplant. SAEs were reported up to day 100. Our study primary outcome is Overall Survival (OS) up to 3 years after transplant. The use of AEs up to 100 days is the standard measurement after a stem cell transplant. OS will capture any major toxicity up to 3 years after transplant that will lead to mortality.
The analysis included all participants who enrolled in the study and underwent umbilical cord blood transplant (UCBT).
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Additional Information
Caridad A. Martinez
Baylor College of Medicine/Texas Children's Hospital
Phone: 832-824-4692
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place