Trial Outcomes & Findings for Combined Haploidentical-Cord Blood Transplantation for Adults and Children (NCT NCT00943800)
NCT ID: NCT00943800
Last Updated: 2021-01-27
Results Overview
Cumulative incidence of graft failure (neutrophil) by day 28 was reported. Patients who did not have neutrophil engraftment before death was considered as a competing risk. Failure to engraft was defined as lack of evidence of hematopoietic recovery (ANC \<500/mm3 and platelet count \< 20,000/mm3) by day +35, confirmed by a biopsy revealing a marrow cellularity \< 5%. Graft failure was also defined as initial myeloid engraftment by day +35, documented to be of donor origin, followed by a drop in the ANC to \< 500/mm3 for more than three days, independent of any myelosuppressive drugs, severe GVHD, CMV, or other infection.
COMPLETED
NA
87 participants
Transplant (Day 0) through Day +28
2021-01-27
Participant Flow
Participant milestones
| Measure |
Treatment Group
Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients.
Subjects received 30mg/m\^2 of Fludarabine from Day-7 until Day-3, 140mg/m\^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."
|
|---|---|
|
Overall Study
STARTED
|
87
|
|
Overall Study
COMPLETED
|
87
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Combined Haploidentical-Cord Blood Transplantation for Adults and Children
Baseline characteristics by cohort
| Measure |
Treatment Group
n=87 Participants
Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients. Subjects received 30mg/m\^2 of Fludarabine from Day-7 until Day-3, 140mg/m\^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."
|
|---|---|
|
Age, Continuous
|
45.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
36 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
51 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
3 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
|
21 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
53 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
9 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Transplant (Day 0) through Day +28Cumulative incidence of graft failure (neutrophil) by day 28 was reported. Patients who did not have neutrophil engraftment before death was considered as a competing risk. Failure to engraft was defined as lack of evidence of hematopoietic recovery (ANC \<500/mm3 and platelet count \< 20,000/mm3) by day +35, confirmed by a biopsy revealing a marrow cellularity \< 5%. Graft failure was also defined as initial myeloid engraftment by day +35, documented to be of donor origin, followed by a drop in the ANC to \< 500/mm3 for more than three days, independent of any myelosuppressive drugs, severe GVHD, CMV, or other infection.
Outcome measures
| Measure |
Treatment Group
n=87 Participants
Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients. Subjects received 30mg/m\^2 of Fludarabine from Day-7 until Day-3, 140mg/m\^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."
|
|---|---|
|
Percentage of Participants With Neutrophil Engraftment
|
85.1 percentage of patients
Interval 75.4 to 91.1
|
SECONDARY outcome
Timeframe: Up to 2 yearsAcute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+). Chronic GVHD is assessed by NIH consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005; 11:945-56., for grading criteria. (See Citation: Filipovich AH et al) The incidence of patients with acute GVHD (Grade II-IV) was determined at 180 days. The incidence of Chronic GVHD by 2 years was reported
Outcome measures
| Measure |
Treatment Group
n=87 Participants
Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients. Subjects received 30mg/m\^2 of Fludarabine from Day-7 until Day-3, 140mg/m\^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."
|
|---|---|
|
Percentage of Participants With Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)
Acute GVHD (grade II-IV)
|
16.1 percentage of patients
Interval 9.3 to 24.6
|
|
Percentage of Participants With Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)
Chronic GVHD
|
3.4 percentage of patients
Interval 0.9 to 9.0
|
SECONDARY outcome
Timeframe: up to 5 yearsWe reported overall survival at 2 years and 5 years after transplant
Outcome measures
| Measure |
Treatment Group
n=87 Participants
Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients. Subjects received 30mg/m\^2 of Fludarabine from Day-7 until Day-3, 140mg/m\^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."
|
|---|---|
|
Overall Survival- Percentage of Participants Who Survived at 2 Years and 5 Years
Two-year overall survival
|
43.7 percentage of patients
Interval 34.4 to 55.4
|
|
Overall Survival- Percentage of Participants Who Survived at 2 Years and 5 Years
Five-year overall survival
|
32.9 percentage of patients
Interval 24.3 to 44.5
|
Adverse Events
Treatment Group
Serious adverse events
| Measure |
Treatment Group
n=87 participants at risk
Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients. Subjects received 30mg/m\^2 of Fludarabine from Day-7 until Day-3, 140mg/m\^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."
|
|---|---|
|
Infections and infestations
Abdominal infection
|
2.3%
2/87 • 100 days
|
|
Blood and lymphatic system disorders
Anemia
|
1.1%
1/87 • 100 days
|
|
Immune system disorders
Cytokine release syndrome
|
1.1%
1/87 • 100 days
|
|
Gastrointestinal disorders
Diarrhea
|
1.1%
1/87 • 100 days
|
|
Infections and infestations
Epstein-barr virus infection
|
3.4%
3/87 • 100 days
|
|
Infections and infestations
Esophageal infection
|
1.1%
1/87 • 100 days
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
1.1%
1/87 • 100 days
|
|
Gastrointestinal disorders
Fever
|
1.1%
1/87 • 100 days
|
|
General disorders
Gait disturbance
|
1.1%
1/87 • 100 days
|
|
Cardiac disorders
Heart failure
|
1.1%
1/87 • 100 days
|
|
Metabolism and nutrition disorders
Hypernatremia
|
1.1%
1/87 • 100 days
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
3.4%
3/87 • 100 days
|
|
Infections and infestations
Lung infection
|
2.3%
2/87 • 100 days
|
|
Investigations
Neutrophil count decreased
|
1.1%
1/87 • 100 days
|
|
Cardiac disorders
Pericardial tamponade
|
1.1%
1/87 • 100 days
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
4.6%
4/87 • 100 days
|
|
Infections and infestations
Sepsis
|
12.6%
11/87 • 100 days
|
|
Infections and infestations
Skin infection
|
1.1%
1/87 • 100 days
|
|
Investigations
White blood cell decreased
|
1.1%
1/87 • 100 days
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place