Trial Outcomes & Findings for Umbilical Cord Blood Transplant for Congenital Pediatric Disorders (NCT NCT00950846)
NCT ID: NCT00950846
Last Updated: 2023-10-30
Results Overview
To determine the overall survival rate at 100 days after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
40 participants
Primary outcome timeframe
100 days
Results posted on
2023-10-30
Participant Flow
Participant milestones
| Measure |
Umbilical Cord Blood Transplant Treatment Plan
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
|
|---|---|
|
Overall Study
STARTED
|
40
|
|
Overall Study
COMPLETED
|
30
|
|
Overall Study
NOT COMPLETED
|
10
|
Reasons for withdrawal
| Measure |
Umbilical Cord Blood Transplant Treatment Plan
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
|
Overall Study
Death
|
6
|
|
Overall Study
Lost to Follow-up
|
3
|
|
Overall Study
Possible undiagnosed AML at study entry
|
1
|
Baseline Characteristics
This is age at transplant. One participant did not receive transplant.
Baseline characteristics by cohort
| Measure |
Umbilical Cord Blood Transplant Treatment Plan
n=38 Participants
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
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|---|---|
|
Age, Continuous
|
5 months
n=5 Participants • This is age at transplant. One participant did not receive transplant.
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
18 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
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
35 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 daysPopulation: The analysis included 38 evaluable participants who completed conditioning and received transplants. Two participants were excluded from the analysis. One participant died before receiving transplant and the other was diagnosed with aplastic anemia without signs of malignancy at the time of enrollment, but later showed signs of MDS and progressed to AML, which made the participant not evaluable for this study.
To determine the overall survival rate at 100 days after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Outcome measures
| Measure |
Umbilical Cord Blood Transplant Treatment Plan
n=38 Participants
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
|
|---|---|
|
Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients.
|
0.947 probability of overall survival
Interval 0.806 to 0.987
|
PRIMARY outcome
Timeframe: 1 yearPopulation: The analysis included 38 evaluable participants who completed conditioning and received transplants. Two participants were excluded from the analysis. One participant died before receiving transplant and the other was diagnosed with aplastic anemia without signs of malignancy at the time of enrollment, but later showed signs of MDS and progressed to AML, which made the participant not evaluable for this study.
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 Treatment Plan
n=38 Participants
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
|
|---|---|
|
Overall Survival at 1 Year After Umbilical Cord Blood Transplant in Pediatric Patients.
|
0.868 probability of overall survival
Interval 0.712 to 0.943
|
PRIMARY outcome
Timeframe: 3 yearsPopulation: The analysis included 38 evaluable participants who completed conditioning and received transplants. Two participants were excluded from the analysis. One participant died before receiving transplant and the other was diagnosed with aplastic anemia without signs of malignancy at the time of enrollment, but later showed signs of MDS and progressed to AML, which made the participant not evaluable for this study.
To determine the overall survival rate at 3 years after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Outcome measures
| Measure |
Umbilical Cord Blood Transplant Treatment Plan
n=38 Participants
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
|
|---|---|
|
Overall Survival at 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
|
0.868 probability of overall survival
Interval 0.712 to 0.943
|
SECONDARY outcome
Timeframe: Day 42Population: The analysis included 37 evaluable participants who completed conditioning, received transplants and were alive on day 42. Three participants were excluded from the analysis. One participant died before receiving transplant, one was not evaluable due to possible undiagnosed AML at study entry, and one died on day 31 after transplant.
Achievement of untransfused platelet count \> 20 x 10\^9/L on three consecutive days
Outcome measures
| Measure |
Umbilical Cord Blood Transplant Treatment Plan
n=37 Participants
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
|
|---|---|
|
Number of Participants With Platelet Engraftment
Not engrafted
|
20 Participants
|
|
Number of Participants With Platelet Engraftment
Engrafted
|
17 Participants
|
SECONDARY outcome
Timeframe: Day 100Population: The analysis included all participants who received transplants and were evaluable for acute GVHD. One participant was not evaluable due to possible undiagnosed AML at study entry and all other participants who received transplants were 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 Treatment Plan
n=36 Participants
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
|
|---|---|
|
Incidence of Severe Grade III-IV Acute GvHD at Day 100.
|
1 Participants
|
SECONDARY outcome
Timeframe: 1 yearPopulation: The analysis included all participants who received transplants and were evaluable for chronic GVHD. One participant was not evaluable due to possible undiagnosed AML at study entry and all other participants who received transplants were evaluable for chronic GVHD if he/she engrafted and survived or remained in the study for more than 101 days after transplant.
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 Treatment Plan
n=35 Participants
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
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: 100 days, 6 months and 12 monthsPopulation: The analysis included all participants who underwent transplant and were evaluable at the time of assessments. One participant was not evaluable due to possible undiagnosed AML at study entry.
To evaluate donor engraftment at 100 days, 6 and 12 months after transplant.
Outcome measures
| Measure |
Umbilical Cord Blood Transplant Treatment Plan
n=36 Participants
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
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
|
36 Participants
|
|
Number of Participants With Donor Engraftment After Transplant.
6 months
|
33 Participants
|
|
Number of Participants With Donor Engraftment After Transplant.
12 months
|
33 Participants
|
SECONDARY outcome
Timeframe: Day 42Population: The analysis included 37 evaluable participants who completed conditioning, received transplants and were alive on day 42. Three participants were excluded from the analysis. One participant died before receiving transplant, one was not evaluable due to possible undiagnosed AML at study entry, and one died on day 31 after transplant.
Achievement of absolute neutrophil count \> 0.5 x 10\^9/L on three consecutive days
Outcome measures
| Measure |
Umbilical Cord Blood Transplant Treatment Plan
n=37 Participants
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
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
Engrafted
|
37 Participants
|
|
Number of Participants With Neutrophil Engraftment
Not engrafted
|
0 Participants
|
Adverse Events
Umbilical Cord Blood Transplant Treatment Plan
Serious events: 27 serious events
Other events: 25 other events
Deaths: 6 deaths
Serious adverse events
| Measure |
Umbilical Cord Blood Transplant Treatment Plan
n=40 participants at risk
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
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
|
5.0%
2/40 • 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.
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, specify: Thrombotic microangiopathy
|
2.5%
1/40 • 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.
|
|
Cardiac disorders
Cardiac General - Other: Cardiopulmonary collapse of unknown etiology
|
2.5%
1/40 • 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.
|
|
Infections and infestations
Cardiac arrest
|
2.5%
1/40 • 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.
|
|
Metabolism and nutrition disorders
Catheter related infection
|
20.0%
8/40 • Number of events 8 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Dehydration
|
2.5%
1/40 • 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.
|
|
Gastrointestinal disorders
Dyspnea
|
5.0%
2/40 • 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.
|
|
Gastrointestinal disorders
Gastritis
|
2.5%
1/40 • 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.
|
|
Renal and urinary disorders
Gastrointestinal disorders - Other, specify: Gastritis, reflux esophagitis, CMV postitve
|
2.5%
1/40 • 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.
|
|
Hepatobiliary disorders
Hematuria
|
2.5%
1/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Hepatobiliary disorders - Other, specify: VOD
|
7.5%
3/40 • 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.
|
|
Infections and infestations
Hypoxia
|
2.5%
1/40 • 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.
|
|
Infections and infestations
Infections and infestations - Other, specify: CMV viremia
|
2.5%
1/40 • 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.
|
|
Infections and infestations
Infections and infestations - Other, specify: Klebsiella pneumoniae bacteremia
|
2.5%
1/40 • 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.
|
|
Infections and infestations
Infections and infestations - Other, specify: RSV pneumonia
|
2.5%
1/40 • 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.
|
|
Infections and infestations
Infections and infestations - Other, specify: Rotavirus gastroenteritis
|
2.5%
1/40 • 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.
|
|
General disorders
Infections and infestations - Other, specify: Staphylococcus Epidermidis bacteremia
|
2.5%
1/40 • 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.
|
|
Infections and infestations
Irritability
|
2.5%
1/40 • 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.
|
|
General disorders
Lung infection
|
2.5%
1/40 • 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.
|
|
Musculoskeletal and connective tissue disorders
Multi-organ failure
|
5.0%
2/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Musculoskeletal and connective tissue disorder - Other, specify: Cephalhematoma
|
2.5%
1/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
2.5%
1/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
10.0%
4/40 • Number of events 4 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify: Acute pulmonary hemorrhage
|
2.5%
1/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify: Bronchitis
|
2.5%
1/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify: Chronic lung disease
|
2.5%
1/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify: Respiratory distress
|
5.0%
2/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory,thoracic and mediastinal disorders-Other:Abnormal CT chest;rulled out fungal disease
|
2.5%
1/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory,thoracic and mediastinal disorders-Other:Bilateral pneumonia with pleural effusion
|
2.5%
1/40 • 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.
|
|
Nervous system disorders
Respiratory,thoracic and mediastinal disorders-Other:Respiratory distress secondary to fluid overloa
|
2.5%
1/40 • 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.
|
|
Infections and infestations
Seizure
|
5.0%
2/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Sepsis
|
2.5%
1/40 • 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.
|
|
Infections and infestations
Stridor
|
2.5%
1/40 • 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.
|
|
Vascular disorders
Upper respiratory infection
|
2.5%
1/40 • 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.
|
|
Vascular disorders
Vascular disorders - Other, specify: Veno-occlusive disease
|
2.5%
1/40 • 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.
|
Other adverse events
| Measure |
Umbilical Cord Blood Transplant Treatment Plan
n=40 participants at risk
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan: Day -9, -8, -7 and -6
Patients less than or equal to 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.
Cytoxan: (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
Fludarabine: 40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
Cord Blood Stem Cell Infusion: The cord blood stem cells will be infused on Day 0.
|
|---|---|
|
Investigations
Aspartate aminotransferase increased
|
5.0%
2/40 • 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.
|
|
Investigations
Bicarbonate, serum-low
|
2.5%
1/40 • 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.
|
|
Infections and infestations
Blood bilirubin increased
|
10.0%
4/40 • Number of events 4 • 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.
|
|
Investigations
Calcium, serum-low (hypocalcemia)
|
2.5%
1/40 • 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.
|
|
Gastrointestinal disorders
Catheter related infection
|
15.0%
6/40 • Number of events 7 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Creatinine increased
|
2.5%
1/40 • 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.
|
|
Metabolism and nutrition disorders
Diarrhea
|
5.0%
2/40 • 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.
|
|
Metabolism and nutrition disorders
Dyspnea
|
2.5%
1/40 • 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.
|
|
Vascular disorders
Fibrinogen
|
2.5%
1/40 • 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.
|
|
Metabolism and nutrition disorders
Hematuria
|
2.5%
1/40 • 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.
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
2.5%
1/40 • 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.
|
|
Metabolism and nutrition disorders
Hypernatremia
|
5.0%
2/40 • 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.
|
|
Metabolism and nutrition disorders
Hypertension
|
7.5%
3/40 • Number of events 4 • 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.
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
5.0%
2/40 • 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.
|
|
Investigations
Hypoalbuminemia
|
2.5%
1/40 • 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.
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
17.5%
7/40 • Number of events 9 • 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.
|
|
Gastrointestinal disorders
Hyponatremia
|
10.0%
4/40 • 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.
|
|
Gastrointestinal disorders
Hypophosphatemia
|
5.0%
2/40 • 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.
|
|
General disorders
INR (International Normalized Ratio of prothrombin time)
|
2.5%
1/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Lipase increased
|
2.5%
1/40 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Metabolism and nutrition disorders - Other, specify: Diabetic ketoacidosis
|
2.5%
1/40 • 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.
|
|
Skin and subcutaneous tissue disorders
Mucositis oral
|
2.5%
1/40 • 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.
|
|
Nervous system disorders
Nausea
|
2.5%
1/40 • 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.
|
|
Vascular disorders
PTT (Partial Thromboplastin Time)
|
2.5%
1/40 • 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.
|
|
Vascular disorders
Pain
|
2.5%
1/40 • 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.
|
|
Vascular disorders
Pharyngeal mucositis
|
2.5%
1/40 • 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.
|
|
Vascular disorders
Pulmonary edema
|
2.5%
1/40 • 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.
|
|
Vascular disorders
Rash maculo-papular
|
2.5%
1/40 • 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.
|
|
Vascular disorders
Seizure
|
7.5%
3/40 • 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.
|
|
Vascular disorders
Urinary tract infection
|
2.5%
1/40 • 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.
|
|
Vascular disorders
Vascular disorders - Other, specify: Thrombosis of left femoral vein
|
2.5%
1/40 • 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.
|
Additional Information
Dr. 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