Trial Outcomes & Findings for HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide (NCT NCT02793544)
NCT ID: NCT02793544
Last Updated: 2025-08-06
Results Overview
Death from any cause. The time to this event is the time from HCT to death, loss to follow-up, or end of study (whichever comes first). The overall survival probability and 90% CI will be calculated by the Kaplan Meier product limit estimator.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
80 participants
Primary outcome timeframe
365 days post transplant
Results posted on
2025-08-06
Participant Flow
Participant milestones
| Measure |
Myeloablative Conditioning (MAC)
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
|---|---|---|
|
Overall Study
STARTED
|
40
|
40
|
|
Overall Study
COMPLETED
|
27
|
32
|
|
Overall Study
NOT COMPLETED
|
13
|
8
|
Reasons for withdrawal
| Measure |
Myeloablative Conditioning (MAC)
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
|---|---|---|
|
Overall Study
Death
|
10
|
8
|
|
Overall Study
Lost to Follow-up
|
2
|
0
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide
Baseline characteristics by cohort
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
n=80 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
48.5 years
n=93 Participants
|
59.5 years
n=4 Participants
|
51.5 years
n=27 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=93 Participants
|
21 Participants
n=4 Participants
|
38 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=93 Participants
|
19 Participants
n=4 Participants
|
42 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
12 Participants
n=93 Participants
|
7 Participants
n=4 Participants
|
19 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
28 Participants
n=93 Participants
|
33 Participants
n=4 Participants
|
61 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Black or African American
|
9 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
15 Participants
n=27 Participants
|
|
Race (NIH/OMB)
White
|
29 Participants
n=93 Participants
|
31 Participants
n=4 Participants
|
60 Participants
n=27 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Region of Enrollment
United States
|
40 Participants
n=93 Participants
|
40 Participants
n=4 Participants
|
80 Participants
n=27 Participants
|
|
Karnofsky/Lansky performance score
70
|
2 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Karnofsky/Lansky performance score
80
|
12 Participants
n=93 Participants
|
12 Participants
n=4 Participants
|
24 Participants
n=27 Participants
|
|
Karnofsky/Lansky performance score
90
|
21 Participants
n=93 Participants
|
17 Participants
n=4 Participants
|
38 Participants
n=27 Participants
|
|
Karnofsky/Lansky performance score
100
|
5 Participants
n=93 Participants
|
10 Participants
n=4 Participants
|
15 Participants
n=27 Participants
|
|
HCT Comorbidity Index score
0
|
4 Participants
n=93 Participants
|
9 Participants
n=4 Participants
|
13 Participants
n=27 Participants
|
|
HCT Comorbidity Index score
1
|
2 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
10 Participants
n=27 Participants
|
|
HCT Comorbidity Index score
2
|
10 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
14 Participants
n=27 Participants
|
|
HCT Comorbidity Index score
3+
|
24 Participants
n=93 Participants
|
19 Participants
n=4 Participants
|
43 Participants
n=27 Participants
|
|
Primary Disease
Acute leukemia
|
37 Participants
n=93 Participants
|
23 Participants
n=4 Participants
|
60 Participants
n=27 Participants
|
|
Primary Disease
MDS
|
2 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Primary Disease
CLL
|
0 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Primary Disease
Lymphoma
|
1 Participants
n=93 Participants
|
14 Participants
n=4 Participants
|
15 Participants
n=27 Participants
|
|
Disease status pre-HCT
CR1
|
32 Participants
n=93 Participants
|
23 Participants
n=4 Participants
|
55 Participants
n=27 Participants
|
|
Disease status pre-HCT
CR2+
|
6 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
12 Participants
n=27 Participants
|
|
Disease status pre-HCT
CR
|
1 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
|
Disease status pre-HCT
Progression/Relapse
|
0 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Disease status pre-HCT
PIF
|
0 Participants
n=93 Participants
|
5 Participants
n=4 Participants
|
5 Participants
n=27 Participants
|
|
Disease status pre-HCT
HI
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Refined disease risk index
Low
|
3 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
9 Participants
n=27 Participants
|
|
Refined disease risk index
Intermediate
|
29 Participants
n=93 Participants
|
21 Participants
n=4 Participants
|
50 Participants
n=27 Participants
|
|
Refined disease risk index
High
|
3 Participants
n=93 Participants
|
7 Participants
n=4 Participants
|
10 Participants
n=27 Participants
|
|
Refined disease risk index
Very high
|
0 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Refined disease risk index
N/A
|
5 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
8 Participants
n=27 Participants
|
|
CMV serostatus
Negative
|
16 Participants
n=93 Participants
|
18 Participants
n=4 Participants
|
34 Participants
n=27 Participants
|
|
CMV serostatus
Positive
|
24 Participants
n=93 Participants
|
22 Participants
n=4 Participants
|
46 Participants
n=27 Participants
|
|
Conditioning Regimen
TBI and Cy
|
6 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
6 Participants
n=27 Participants
|
|
Conditioning Regimen
TBI, CY, and Flu
|
0 Participants
n=93 Participants
|
40 Participants
n=4 Participants
|
40 Participants
n=27 Participants
|
|
Conditioning Regimen
Bu and Cy
|
3 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Conditioning Regimen
Bu and Flu
|
31 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
31 Participants
n=27 Participants
|
|
HLA match, categorical
7/8
|
26 Participants
n=93 Participants
|
23 Participants
n=4 Participants
|
49 Participants
n=27 Participants
|
|
HLA match, categorical
6/8
|
8 Participants
n=93 Participants
|
11 Participants
n=4 Participants
|
19 Participants
n=27 Participants
|
|
HLA match, categorical
5/8
|
5 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
7 Participants
n=27 Participants
|
|
HLA match, categorical
4/8
|
1 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
5 Participants
n=27 Participants
|
|
Infused total nucleated cells x10^8/kg
|
2.81 cells x10^8/kg
n=93 Participants
|
2.8 cells x10^8/kg
n=4 Participants
|
2.8 cells x10^8/kg
n=27 Participants
|
|
Infused CD34 cells, x10^6/kg
|
2.72 cells x10^6/kg
n=93 Participants
|
2.2 cells x10^6/kg
n=4 Participants
|
2.66 cells x10^6/kg
n=27 Participants
|
|
Donor age
18-29 years of age
|
24 Participants
n=93 Participants
|
23 Participants
n=4 Participants
|
47 Participants
n=27 Participants
|
|
Donor age
30-39 years of age
|
9 Participants
n=93 Participants
|
11 Participants
n=4 Participants
|
20 Participants
n=27 Participants
|
|
Donor age
40-49 years of age
|
4 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
10 Participants
n=27 Participants
|
|
Donor age
50-59 years of age
|
3 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Donor Sex
Male
|
20 Participants
n=93 Participants
|
24 Participants
n=4 Participants
|
44 Participants
n=27 Participants
|
|
Donor Sex
Female
|
20 Participants
n=93 Participants
|
16 Participants
n=4 Participants
|
36 Participants
n=27 Participants
|
|
Donor/recipient sex
M-M
|
12 Participants
n=93 Participants
|
12 Participants
n=4 Participants
|
24 Participants
n=27 Participants
|
|
Donor/recipient sex
M-F
|
8 Participants
n=93 Participants
|
12 Participants
n=4 Participants
|
20 Participants
n=27 Participants
|
|
Donor/recipient sex
F-M
|
11 Participants
n=93 Participants
|
7 Participants
n=4 Participants
|
18 Participants
n=27 Participants
|
|
Donor/recipient sex
F-F
|
9 Participants
n=93 Participants
|
9 Participants
n=4 Participants
|
18 Participants
n=27 Participants
|
|
Donor/recipient CMV serostatus
+/+
|
18 Participants
n=93 Participants
|
13 Participants
n=4 Participants
|
31 Participants
n=27 Participants
|
|
Donor/recipient CMV serostatus
+/-
|
6 Participants
n=93 Participants
|
5 Participants
n=4 Participants
|
11 Participants
n=27 Participants
|
|
Donor/recipient CMV serostatus
-/+
|
9 Participants
n=93 Participants
|
10 Participants
n=4 Participants
|
19 Participants
n=27 Participants
|
|
Donor/recipient CMV serostatus
-/-
|
7 Participants
n=93 Participants
|
12 Participants
n=4 Participants
|
19 Participants
n=27 Participants
|
|
Donor/recipient ABO match
Matched
|
20 Participants
n=93 Participants
|
24 Participants
n=4 Participants
|
44 Participants
n=27 Participants
|
|
Donor/recipient ABO match
Minor mismatch
|
12 Participants
n=93 Participants
|
5 Participants
n=4 Participants
|
17 Participants
n=27 Participants
|
|
Donor/recipient ABO match
Major mismatch
|
8 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
16 Participants
n=27 Participants
|
|
Donor/recipient ABO match
Bi-directional
|
0 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
HIV infection pre-HCT
No
|
40 Participants
n=93 Participants
|
36 Participants
n=4 Participants
|
76 Participants
n=27 Participants
|
|
HIV infection pre-HCT
Yes
|
0 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
|
Disease and disease status at HCT
AML (CR1)
|
22 Participants
n=93 Participants
|
10 Participants
n=4 Participants
|
32 Participants
n=27 Participants
|
|
Disease and disease status at HCT
AML (CR2+)
|
1 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Disease and disease status at HCT
AML (PIF)
|
0 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Disease and disease status at HCT
ALL (CR1)
|
7 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
13 Participants
n=27 Participants
|
|
Disease and disease status at HCT
ALL (CR2+)
|
3 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
|
Disease and disease status at HCT
CLL (CR)
|
0 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Disease and disease status at HCT
MDS (CR)
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Disease and disease status at HCT
MDS (HI)
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Disease and disease status at HCT
Other acute leukemia (CR1)
|
3 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Disease and disease status at HCT
Other acute leukemia (CR2+)
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Disease and disease status at HCT
NHL (CR1)
|
0 Participants
n=93 Participants
|
5 Participants
n=4 Participants
|
5 Participants
n=27 Participants
|
|
Disease and disease status at HCT
NHL (CR2+)
|
1 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
|
Disease and disease status at HCT
NHL (Relapse)
|
0 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Disease and disease status at HCT
NHL (PIF)
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Disease and disease status at HCT
HL (CR1)
|
0 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Disease and disease status at HCT
HL (Relapse)
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Disease and disease status at HCT
HL (PIF)
|
0 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Time between diagnosis to HCT
< 6 months
|
14 Participants
n=93 Participants
|
10 Participants
n=4 Participants
|
24 Participants
n=27 Participants
|
|
Time between diagnosis to HCT
≥ 6 months
|
26 Participants
n=93 Participants
|
30 Participants
n=4 Participants
|
56 Participants
n=27 Participants
|
|
Number of prior autoHCTs
0
|
38 Participants
n=93 Participants
|
37 Participants
n=4 Participants
|
75 Participants
n=27 Participants
|
|
Number of prior autoHCTs
1
|
2 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
5 Participants
n=27 Participants
|
PRIMARY outcome
Timeframe: 365 days post transplantDeath from any cause. The time to this event is the time from HCT to death, loss to follow-up, or end of study (whichever comes first). The overall survival probability and 90% CI will be calculated by the Kaplan Meier product limit estimator.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Overall Survival
|
72.3 percentage of participants
Interval 59.9 to 83.1
|
78.9 percentage of participants
Interval 66.9 to 88.8
|
—
|
SECONDARY outcome
Timeframe: 180 days and 365 days post-transplantTime from HCT until the documentation of disease progression / relapse or death due to any cause, whichever occurs first. Progression-free survival probability and 90% CI will be calculated by the Kaplan Meier product limit estimator.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Progression-free Survival
180 days
|
69.9 percentage of participants
Interval 57.4 to 81.1
|
72.5 percentage of participants
Interval 60.3 to 83.2
|
—
|
|
Progression-free Survival
365 days
|
62.1 percentage of participants
Interval 49.2 to 74.3
|
67.5 percentage of participants
Interval 54.9 to 79.0
|
—
|
SECONDARY outcome
Timeframe: 100 days, 180 days, and 365 days post-transplantDeath without evidence of disease progression or recurrence. A cumulative incidence will be computed along with a 90% CI.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Transplant-related Mortality
100 days
|
5 percentage of participants
Interval 0.9 to 12.2
|
7.5 percentage of participants
Interval 2.1 to 15.8
|
—
|
|
Transplant-related Mortality
180 days
|
7.5 percentage of participants
Interval 2.1 to 15.8
|
7.5 percentage of participants
Interval 2.1 to 15.8
|
—
|
|
Transplant-related Mortality
365 days
|
7.5 percentage of participants
Interval 2.1 to 15.8
|
10 percentage of participants
Interval 3.6 to 19.2
|
—
|
SECONDARY outcome
Timeframe: 100 days and 365 days post transplantAchieving a donor derived ANC ≥ 500/mm3 for 3 consecutive laboratory values on different days. A cumulative incidence will be computed along with a 90% CI.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidence of Neutrophil Recovery
365 days
|
97.5 percentage of participants
Interval 89.7 to 100.0
|
97.5 percentage of participants
Interval 89.7 to 100.0
|
—
|
|
Cumulative Incidence of Neutrophil Recovery
100 days
|
97.5 percentage of participants
Interval 89.7 to 100.0
|
97.5 percentage of participants
Interval 89.8 to 100.0
|
—
|
SECONDARY outcome
Timeframe: 100 days and 365 days post transplantAchieving a platelet count ≥ 20,000/μL for 3 consecutive laboratory values on different days (with no platelet transfusions in the preceding seven days). A cumulative incidence will be computed along with a 90% CI.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidence of Platelet Recovery
100 days
|
87.5 percentage of participants
Interval 77.0 to 95.1
|
95 percentage of participants
Interval 86.2 to 99.5
|
—
|
|
Cumulative Incidence of Platelet Recovery
365 days
|
90 percentage of participants
Interval 79.7 to 96.9
|
95 percentage of participants
Interval 86.2 to 99.5
|
—
|
SECONDARY outcome
Timeframe: 100 days post-transplantAny skin, gastrointestinal or liver abnormalities fulfilling the BMT CTN Manual of Operations (2013) criteria of grades II-IV acute GVHD. A cumulative incidence will be computed along with a 90% CI. Any skin, gastrointestinal or liver abnormalities fulfilling the BMT CTN Manual of Operations (2013) criteria of grades III-IV acute GVHD. A cumulative incidence will be computed along with a 90% CI.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidence of Acute GVHD
Grades II-IV acute GVHD
|
42.5 percentage of participants
Interval 29.8 to 55.7
|
32.5 percentage of participants
Interval 20.9 to 45.3
|
—
|
|
Cumulative Incidence of Acute GVHD
Grades III-IV acute GVHD
|
17.5 percentage of participants
Interval 8.7 to 28.5
|
0 percentage of participants
Interval 0.0 to 0.0
|
—
|
SECONDARY outcome
Timeframe: 100 daysAny skin, gastrointestinal or liver abnormalities fulfilling the BMT CTN Manual of Operations (2013) criteria of grades II-IV acute GVHD. A cumulative incidence will be computed along with a 90% CI.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Grades II-IV Acute GVHD
|
42.5 percentage of participants
Interval 29.8 to 55.7
|
32.5 percentage of participants
Interval 20.9 to 45.3
|
—
|
SECONDARY outcome
Timeframe: 100 daysAny skin, gastrointestinal or liver abnormalities fulfilling the BMT CTN Manual of Operations (2013) criteria of grades III-IV acute GVHD. A cumulative incidence will be computed along with a 90% CI.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Grades III-IV Acute GVHD
|
17.5 percentage of participants
Interval 8.7 to 28.5
|
0 percentage of participants
Interval 0.0 to 0.0
|
—
|
SECONDARY outcome
Timeframe: 180 days and 365 days post-transplantPer National Institutes of Health (NIH) Consensus Criteria and includes organ involvement and severity, and overall global composite score (mild/moderate/severe). A cumulative incidence will be computed along with a 90% CI. Death from any cause. The time to this event is the time from HCT to death, loss to follow-up, or end of study (whichever comes first). The overall survival probability and 90% CI will be calculated by the Kaplan Meier product limit estimator.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidence of Chronic GVHD
180 days
|
27.6 percentage of participants
Interval 16.7 to 40.2
|
10 percentage of participants
Interval 3.6 to 19.2
|
—
|
|
Cumulative Incidence of Chronic GVHD
365 days
|
35.5 percentage of participants
Interval 23.3 to 48.7
|
17.5 percentage of participants
Interval 8.7 to 28.5
|
—
|
SECONDARY outcome
Timeframe: 100 days, 180 days, and 365 daysGrade 2: Clinically active CMV infection (e.g. symptoms, cytopenias) or CMV Viremia not decreasing by at least 2/3 of the baseline value after 2 weeks of therapy; Grade 3: CMV end-organ involvement (pneumonitis, enteritis, retinitis). A cumulative incidence will be computed along with a 90% CI. Death from any cause. The time to this event is the time from HCT to death, loss to follow-up, or end of study (whichever comes first). The overall survival probability and 90% CI will be calculated by the Kaplan Meier product limit estimator.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2-3 CMV Reactivation or Infection
100 days
|
10.4 percentage of participants
Interval 3.8 to 19.8
|
7.6 percentage of participants
Interval 2.2 to 16.0
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2-3 CMV Reactivation or Infection
180 days
|
10.4 percentage of participants
Interval 3.8 to 19.8
|
7.6 percentage of participants
Interval 2.2 to 16.0
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2-3 CMV Reactivation or Infection
365 days
|
10.4 percentage of participants
Interval 3.8 to 19.8
|
7.6 percentage of participants
Interval 2.2 to 16.0
|
—
|
SECONDARY outcome
Timeframe: 100 days, 180 days, and 365 daysCMV end-organ involvement (pneumonitis, enteritis, retinitis) ). A cumulative incidence will be computed along with a 90% CI. Death from any cause. The time to this event is the time from HCT to death, loss to follow-up, or end of study (whichever comes first). The overall survival probability and 90% CI will be calculated by the Kaplan Meier product limit estimator.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 3 CMV Infection
100 days
|
0 percentage of participants
Interval 0.0 to 0.0
|
2.6 percentage of participants
Interval 0.1 to 8.5
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 3 CMV Infection
180 days
|
0 percentage of participants
Interval 0.0 to 0.0
|
2.6 percentage of participants
Interval 0.1 to 8.5
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 3 CMV Infection
365 days
|
0 percentage of participants
Interval 0.0 to 0.0
|
2.6 percentage of participants
Interval 0.1 to 8.5
|
—
|
SECONDARY outcome
Timeframe: 100 days, 180 days, and 365 daysGrade 2: EBV reactivation requiring institution of therapy with rituximab; Grade 3: EBV post-transplant lymphoproliferative disorder). A cumulative incidence will be computed along with a 90% CI. Death from any cause. The time to this event is the time from HCT to death, loss to follow-up, or end of study (whichever comes first). The overall survival probability and 90% CI will be calculated by the Kaplan Meier product limit estimator.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2-3 EBV Infection
100 days
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2-3 EBV Infection
180 days
|
2.9 percentage of participants
Interval 0.1 to 9.5
|
0 percentage of participants
Interval 0.0 to 0.0
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2-3 EBV Infection
365 days
|
2.9 percentage of participants
Interval 0.1 to 9.5
|
0 percentage of participants
Interval 0.0 to 0.0
|
—
|
SECONDARY outcome
Timeframe: 100 days, 180 days, 365 daysBK viremia or viruria with clinical consequence requiring prolonged therapy and/or surgical intervention). A cumulative incidence will be computed along with a 90% CI.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=38 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=37 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2 BK Virus Infection
100 days
|
26.7 percentage of participants
Interval 15.7 to 39.2
|
29.7 percentage of participants
Interval 18.3 to 42.7
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2 BK Virus Infection
180 days
|
26.7 percentage of participants
Interval 15.7 to 39.2
|
29.7 percentage of participants
Interval 18.3 to 42.7
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2 BK Virus Infection
365 days
|
26.7 percentage of participants
Interval 15.7 to 39.2
|
29.7 percentage of participants
Interval 18.3 to 42.7
|
—
|
SECONDARY outcome
Timeframe: 100 days, 180 days, and 365 daysGrade 2: Adenoviral URI, viremia, or symptomatic viruria requiring treatment; Grade 3: ADV with end-organ involvement (except conjunctivitis and upper respiratory tract) ). A cumulative incidence will be computed along with a 90% CI. Death from any cause. The time to this event is the time from HCT to death, loss to follow-up, or end of study (whichever comes first). The overall survival probability and 90% CI will be calculated by the Kaplan Meier product limit estimator.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=39 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=36 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2-3 Adenovirus Infection
100 days
|
2.6 percentage of participants
Interval 0.1 to 8.5
|
0 percentage of participants
Interval 0.0 to 0.0
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2-3 Adenovirus Infection
180 days
|
9 percentage of participants
Interval 2.6 to 18.9
|
9.4 percentage of participants
Interval 2.7 to 19.4
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2-3 Adenovirus Infection
365 days
|
9 percentage of participants
Interval 2.6 to 18.9
|
12.6 percentage of participants
Interval 4.6 to 23.8
|
—
|
SECONDARY outcome
Timeframe: 100 days, 180 days, 365 daysClinically active HHV-6 infection (e.g. symptoms, cytopenias) or HHV-6 viremia without viral load decline 0.5 log after 2 weeks of therapy). A cumulative incidence will be computed along with a 90% CI.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=39 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2 HHV-6 Infection
100 days
|
12.5 percentage of participants
Interval 5.3 to 22.3
|
5.1 percentage of participants
Interval 0.9 to 12.4
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2 HHV-6 Infection
180 days
|
12.5 percentage of participants
Interval 5.3 to 22.3
|
5.1 percentage of participants
Interval 0.9 to 12.4
|
—
|
|
Cumulative Incidences of Viral Reactivations and Infections: Grade 2 HHV-6 Infection
365 days
|
12.5 percentage of participants
Interval 5.3 to 22.3
|
5.1 percentage of participants
Interval 0.9 to 12.4
|
—
|
SECONDARY outcome
Timeframe: 180 days and 365 days post-transplantDefined by either morphological, cytogenetic/molecular or radiological evidence of disease consistent with pre-HCT features. A cumulative incidence will be computed along with a 90% CI. Death from any cause. The time to this event is the time from HCT to death, loss to follow-up, or end of study (whichever comes first). The overall survival probability and 90% CI will be calculated by the Kaplan Meier product limit estimator.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidence of Relapse/Progression
180 days
|
22.6 percentage of participants
Interval 12.6 to 34.5
|
20 percentage of participants
Interval 10.6 to 31.5
|
—
|
|
Cumulative Incidence of Relapse/Progression
365 days
|
30.4 percentage of participants
Interval 18.9 to 43.2
|
22.5 percentage of participants
Interval 12.6 to 34.3
|
—
|
SECONDARY outcome
Timeframe: 365 days post transplantTMA is defined as (Ho et al., 2005): 1. RBC fragmentation and \>2 schistocytes per high-power field on peripheral smear 2. Concurrent increased serum LDH above institutional baseline 3. Concurrent renal and/or neurologic dysfunction without other explanation 4. Negative direct and indirect Coombs test results A cumulative incidence will be computed along with a 90% CI. VOD/SOS is defined as: In the first 20 days after HCT, the presence of ≥2 of the following: 1. Bilirubin \>2 mg/Dl, 2. Hepatomegaly or pain in right upper quadrant, 3. Weight gain (\>2% basal weight). A cumulative incidence will be computed along with a 90% CI.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidences of Thrombotic Microangiopathy (TMA) and Hepatic Veno-occlusive Disease (VOD)/Sinusoidal Obstruction Syndrome (SOS)
Thrombotic microangiopathy (TMA)
|
5 percentage of participants
Interval 0.9 to 12.2
|
0 percentage of participants
Interval 0.0 to 0.0
|
—
|
|
Cumulative Incidences of Thrombotic Microangiopathy (TMA) and Hepatic Veno-occlusive Disease (VOD)/Sinusoidal Obstruction Syndrome (SOS)
Hepatic veno-occlusive disease (VOD)/sinusoidal obstruction syndrome (SOS)
|
10 percentage of participants
Interval 3.6 to 19.2
|
2.5 percentage of participants
Interval 0.1 to 8.2
|
—
|
SECONDARY outcome
Timeframe: 56 days post-transplantPopulation: Primary graft failure Lack of donor-derived neutrophil engraftment. The frequency of subjects experiencing primary graft failure by 56 days will be described. Transplanted participants surviving a minimum of 14 days post HCT and have complete data for both event status and event date.
Lack of donor-derived neutrophil engraftment. The frequency of subjects experiencing primary graft failure by 56 days will be described.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=39 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Cumulative Incidence of Primary Graft Failure
|
0 Participants
|
3 Participants
|
—
|
SECONDARY outcome
Timeframe: 28 days, 56 days, 100 days, 180 days, and 365 days post-transplantPeripheral blood chimerism (% of donor chimerism) in whole blood (unsorted). The degree of donor chimerism will be summarized using descriptive statistics.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=34 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=39 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Donor Chimerism
56 days
|
100 percentage of donor chimerism
Interval 64.0 to 100.0
|
100 percentage of donor chimerism
Interval 0.0 to 100.0
|
—
|
|
Donor Chimerism
28 days
|
100 percentage of donor chimerism
Interval 92.0 to 100.0
|
99.1 percentage of donor chimerism
Interval 0.0 to 100.0
|
—
|
|
Donor Chimerism
100 days
|
100 percentage of donor chimerism
Interval 43.0 to 100.0
|
100 percentage of donor chimerism
Interval 0.0 to 100.0
|
—
|
|
Donor Chimerism
180 days
|
100 percentage of donor chimerism
Interval 54.2 to 100.0
|
100 percentage of donor chimerism
Interval 43.9 to 100.0
|
—
|
|
Donor Chimerism
365 days
|
100 percentage of donor chimerism
Interval 79.4 to 100.0
|
100 percentage of donor chimerism
Interval 83.2 to 100.0
|
—
|
SECONDARY outcome
Timeframe: 56 days post-transplantThe frequency of subjects with Peripheral blood (unsorted) chimerism\>95% at 56 days will be described.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=26 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=32 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Peripheral Blood Chimerism
|
24 Participants
|
27 Participants
|
—
|
SECONDARY outcome
Timeframe: Pre-HCTPopulation: Participants who signed informed consent.
The proportion of subjects proceeding to HCT after informed consent.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=94 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Proportion of Subjects Proceeding to Transplant
|
80 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Pre-HCTTime from search to donor identification Time from preliminary search to formal donor activation.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Time From Search to Donor Identification
|
77.5 days
Interval 16.0 to 733.0
|
88.5 days
Interval 30.0 to 763.0
|
—
|
SECONDARY outcome
Timeframe: Pre-HCTNumber of matched donor and recipient HLA allele pairs. A matched donor would have 8/8 HLA allele pairs matching; mismatched donors listed below have one to four mismatched HLA allele pairs at HLA-A, -B, -C, or -DRB1.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
n=80 Participants
Total number of participants
|
|---|---|---|---|
|
Donor Selection Characteristics: HLA Match
7 out of 8 matching HLA allele pairs
|
26 Participants
|
23 Participants
|
49 Participants
|
|
Donor Selection Characteristics: HLA Match
6 out of 8 matching HLA allele pairs
|
8 Participants
|
11 Participants
|
19 Participants
|
|
Donor Selection Characteristics: HLA Match
5 out of 8 matching HLA allele pairs
|
5 Participants
|
2 Participants
|
7 Participants
|
|
Donor Selection Characteristics: HLA Match
4 out of 8 matching HLA allele pairs
|
1 Participants
|
4 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Pre-HCTDonor age
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
n=80 Participants
Total number of participants
|
|---|---|---|---|
|
Donor Selection Characteristics: Donor Age
|
27 years
Interval 18.0 to 56.0
|
29 years
Interval 21.0 to 44.0
|
29 years
Interval 18.0 to 56.0
|
SECONDARY outcome
Timeframe: Pre-HCTDonor age, categorical
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
n=80 Participants
Total number of participants
|
|---|---|---|---|
|
Donor Selection Characteristics: Donor Age, Categorical
18-29
|
24 Participants
|
23 Participants
|
47 Participants
|
|
Donor Selection Characteristics: Donor Age, Categorical
30-39
|
9 Participants
|
11 Participants
|
20 Participants
|
|
Donor Selection Characteristics: Donor Age, Categorical
40-49
|
4 Participants
|
6 Participants
|
10 Participants
|
|
Donor Selection Characteristics: Donor Age, Categorical
50-59
|
3 Participants
|
0 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Pre-HCTDonor weight
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
n=80 Participants
Total number of participants
|
|---|---|---|---|
|
Donor Selection Characteristics: Donor Weight
|
77 kg
Interval 55.0 to 103.0
|
77 kg
Interval 52.0 to 104.0
|
77 kg
Interval 52.0 to 104.0
|
SECONDARY outcome
Timeframe: Pre-HCTDonor sex
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
n=80 Participants
Total number of participants
|
|---|---|---|---|
|
Donor Selection Characteristics: Donor Sex
Male
|
20 Participants
|
24 Participants
|
44 Participants
|
|
Donor Selection Characteristics: Donor Sex
Female
|
20 Participants
|
16 Participants
|
36 Participants
|
SECONDARY outcome
Timeframe: Pre-HCTDonor-recipient sex match
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
n=80 Participants
Total number of participants
|
|---|---|---|---|
|
Donor Selection Characteristics: Donor and Recipient Sex
Male and male
|
12 Participants
|
12 Participants
|
24 Participants
|
|
Donor Selection Characteristics: Donor and Recipient Sex
Male and female
|
8 Participants
|
12 Participants
|
20 Participants
|
|
Donor Selection Characteristics: Donor and Recipient Sex
Female and male
|
11 Participants
|
7 Participants
|
18 Participants
|
|
Donor Selection Characteristics: Donor and Recipient Sex
Female and female
|
9 Participants
|
9 Participants
|
18 Participants
|
SECONDARY outcome
Timeframe: Pre-HCTDonor-recipient Cytomegalovirus (CMV) serostatus match
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
n=80 Participants
Total number of participants
|
|---|---|---|---|
|
Donor Selection Characteristics: Donor and Recipient CMV Serostatus
+/+
|
18 Participants
|
13 Participants
|
31 Participants
|
|
Donor Selection Characteristics: Donor and Recipient CMV Serostatus
+/-
|
6 Participants
|
5 Participants
|
11 Participants
|
|
Donor Selection Characteristics: Donor and Recipient CMV Serostatus
-/+
|
9 Participants
|
10 Participants
|
19 Participants
|
|
Donor Selection Characteristics: Donor and Recipient CMV Serostatus
-/-
|
7 Participants
|
12 Participants
|
19 Participants
|
SECONDARY outcome
Timeframe: Pre-HCTDonor-recipient ABO group match
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
n=80 Participants
Total number of participants
|
|---|---|---|---|
|
Donor Selection Characteristics: Donor and Recipient ABO Blood Match
Matched
|
20 Participants
|
24 Participants
|
44 Participants
|
|
Donor Selection Characteristics: Donor and Recipient ABO Blood Match
Minor mismatch
|
12 Participants
|
5 Participants
|
17 Participants
|
|
Donor Selection Characteristics: Donor and Recipient ABO Blood Match
Major mismatch
|
8 Participants
|
8 Participants
|
16 Participants
|
|
Donor Selection Characteristics: Donor and Recipient ABO Blood Match
Bidirectional
|
0 Participants
|
3 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: 100 days and 365 days post-transplantPopulation: All participants assessed for donor clonal hematopoiesis.
The proportion of subjects developing donor clonal hematopoiesis
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
n=40 Participants
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Donor Clonal Hematopoiesis
|
0 Participants
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: 365 days post transplantPopulation: Four subjects with HIV-positive, no statistical analysis for this outcome measure was completed due to limited HIV-positive rate. Descriptive analysis provided.
If CCR5delta32 homozygous donors are successfully found and used for one or more HIV-positive subjects, a descriptive analysis of baseline characteristics and outcomes for those HIV-positive subjects will be conducted, including the viral load detected over time obtained from collected samples.
Outcome measures
| Measure |
Myeloablative Conditioning (MAC)
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=4 Participants
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
Total
Total number of participants
|
|---|---|---|---|
|
Subgroup Analysis of HIV-positive Subjects
Survival Status: Alive
|
0 Participants
|
1 Participants
|
—
|
|
Subgroup Analysis of HIV-positive Subjects
Survival Status: Deceased (relapse)
|
0 Participants
|
2 Participants
|
—
|
|
Subgroup Analysis of HIV-positive Subjects
Survival Status: Deceased (fungal infection)
|
0 Participants
|
1 Participants
|
—
|
Adverse Events
Myeloablative Conditioning (MAC)
Serious events: 12 serious events
Other events: 5 other events
Deaths: 11 deaths
Reduced Intensity Conditioning (RIC)
Serious events: 13 serious events
Other events: 0 other events
Deaths: 8 deaths
Serious adverse events
| Measure |
Myeloablative Conditioning (MAC)
n=40 participants at risk
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 participants at risk
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
|---|---|---|
|
Blood and lymphatic system disorders
Other
|
5.0%
2/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Eye disorders
Other
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
General disorders
Multi-organ failure
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
5.0%
2/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Gastrointestinal disorders
Obstruction gastric
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Infections and infestations
Lung infection
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Infections and infestations
Sepsis
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
7.5%
3/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Infections and infestations
Sinusitis
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Infections and infestations
Other
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Investigations
Other
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Leukemia secondary to oncology chemotherapy
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Other
|
7.5%
3/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
5.0%
2/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Respiratory, thoracic and mediastinal disorders
Adult respiratory distress syndrome
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
5.0%
2/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
Other adverse events
| Measure |
Myeloablative Conditioning (MAC)
n=40 participants at risk
Participants received one of three regimens: cyclophosphamide and total body irradiation; busulfan and cyclophosphamide; or fludarabine and busulfan
|
Reduced Intensity Conditioning (RIC)
n=40 participants at risk
Participants received one of two regimens: fludarabine, cyclophosphamide, and low-dose total body irradiation
|
|---|---|---|
|
Cardiac disorders
Myocardial infarction
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Infections and infestations
Sepsis
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Injury, poisoning and procedural complications
Fall
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
2.5%
1/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
0.00%
0/40 • Up to 1 year post transplant
Evaluation of toxicities will be graded using National Cancer Institute Common Toxicity Criteria, specifically CTCAE version 4.03. The protocol pre-specifies that Adverse Events on this study are reported and evaluated by conditioning regimen arm (MAC or RIC).
|
Additional Information
Steve Spellman
National Marrow Donor Program/BeTheMatch
Phone: 763-406-8334
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place