Trial Outcomes & Findings for Chronic Graft-versus-Host Disease Treatment (BMT CTN 0801) (NCT NCT01106833)
NCT ID: NCT01106833
Last Updated: 2018-12-05
Results Overview
Treatment success was evaluated at 6 months in Phase II and is defined as a complete or partial response without secondary systemic immunosuppressive therapy and no recurrent malignancy or death. In Phase III, treatment success was evaluated at 24 months and is defined as a complete response without secondary systemic immunosuppressive therapy and no recurrent malignancy or death.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
151 participants
Primary outcome timeframe
6 months and 24 months post-randomization
Results posted on
2018-12-05
Participant Flow
Participant milestones
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Overall Study
STARTED
|
74
|
77
|
|
Overall Study
COMPLETED
|
74
|
77
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Donor type is described only for bone marrow and peripheral blood cell transplants.
Baseline characteristics by cohort
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Total
n=138 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
50.9 years
STANDARD_DEVIATION 14.2 • n=66 Participants
|
47.4 years
STANDARD_DEVIATION 16.1 • n=72 Participants
|
49.1 years
STANDARD_DEVIATION 15.2 • n=138 Participants
|
|
Age, Customized
Less Than 20
|
2 Participants
n=66 Participants
|
5 Participants
n=72 Participants
|
7 Participants
n=138 Participants
|
|
Age, Customized
20 - 40
|
12 Participants
n=66 Participants
|
18 Participants
n=72 Participants
|
30 Participants
n=138 Participants
|
|
Age, Customized
41 - 60
|
36 Participants
n=66 Participants
|
30 Participants
n=72 Participants
|
66 Participants
n=138 Participants
|
|
Age, Customized
Over 60
|
16 Participants
n=66 Participants
|
19 Participants
n=72 Participants
|
35 Participants
n=138 Participants
|
|
Sex: Female, Male
Female
|
33 Participants
n=66 Participants
|
24 Participants
n=72 Participants
|
57 Participants
n=138 Participants
|
|
Sex: Female, Male
Male
|
33 Participants
n=66 Participants
|
48 Participants
n=72 Participants
|
81 Participants
n=138 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=66 Participants
|
8 Participants
n=72 Participants
|
8 Participants
n=138 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
59 Participants
n=66 Participants
|
63 Participants
n=72 Participants
|
122 Participants
n=138 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
7 Participants
n=66 Participants
|
1 Participants
n=72 Participants
|
8 Participants
n=138 Participants
|
|
Race/Ethnicity, Customized
White
|
57 Participants
n=66 Participants
|
54 Participants
n=72 Participants
|
111 Participants
n=138 Participants
|
|
Race/Ethnicity, Customized
Non-white
|
9 Participants
n=66 Participants
|
13 Participants
n=72 Participants
|
22 Participants
n=138 Participants
|
|
Race/Ethnicity, Customized
Unknown / Not Answered
|
0 Participants
n=66 Participants
|
5 Participants
n=72 Participants
|
5 Participants
n=138 Participants
|
|
Primary Disease
Acute Leukemia
|
25 Participants
n=66 Participants
|
47 Participants
n=72 Participants
|
72 Participants
n=138 Participants
|
|
Primary Disease
Chronic Leukemia
|
8 Participants
n=66 Participants
|
7 Participants
n=72 Participants
|
15 Participants
n=138 Participants
|
|
Primary Disease
MDS / MPS
|
9 Participants
n=66 Participants
|
12 Participants
n=72 Participants
|
21 Participants
n=138 Participants
|
|
Primary Disease
Lymphoma
|
17 Participants
n=66 Participants
|
5 Participants
n=72 Participants
|
22 Participants
n=138 Participants
|
|
Primary Disease
Other
|
7 Participants
n=66 Participants
|
1 Participants
n=72 Participants
|
8 Participants
n=138 Participants
|
|
Graft Type
Bone Marrow
|
5 Participants
n=66 Participants
|
5 Participants
n=72 Participants
|
10 Participants
n=138 Participants
|
|
Graft Type
Peripheral Blood Stem Cells
|
60 Participants
n=66 Participants
|
64 Participants
n=72 Participants
|
124 Participants
n=138 Participants
|
|
Graft Type
Single Cord Blood
|
1 Participants
n=66 Participants
|
0 Participants
n=72 Participants
|
1 Participants
n=138 Participants
|
|
Graft Type
Double Cord Blood
|
0 Participants
n=66 Participants
|
3 Participants
n=72 Participants
|
3 Participants
n=138 Participants
|
|
Donor Type
Related
|
38 Participants
n=65 Participants • Donor type is described only for bone marrow and peripheral blood cell transplants.
|
29 Participants
n=69 Participants • Donor type is described only for bone marrow and peripheral blood cell transplants.
|
67 Participants
n=134 Participants • Donor type is described only for bone marrow and peripheral blood cell transplants.
|
|
Donor Type
Unrelated
|
27 Participants
n=65 Participants • Donor type is described only for bone marrow and peripheral blood cell transplants.
|
40 Participants
n=69 Participants • Donor type is described only for bone marrow and peripheral blood cell transplants.
|
67 Participants
n=134 Participants • Donor type is described only for bone marrow and peripheral blood cell transplants.
|
|
Conditioning Regimen Intensity
Myeloablative
|
29 Participants
n=66 Participants
|
47 Participants
n=72 Participants
|
76 Participants
n=138 Participants
|
|
Conditioning Regimen Intensity
Non-myeloablative or Reduced Intensity
|
37 Participants
n=66 Participants
|
25 Participants
n=72 Participants
|
62 Participants
n=138 Participants
|
|
Donor Age
|
44.4 years
STANDARD_DEVIATION 15.2 • n=66 Participants
|
38.5 years
STANDARD_DEVIATION 12.9 • n=72 Participants
|
41.3 years
STANDARD_DEVIATION 14.0 • n=138 Participants
|
|
Donor Gender
Female
|
26 Participants
n=66 Participants
|
35 Participants
n=72 Participants
|
61 Participants
n=138 Participants
|
|
Donor Gender
Male
|
38 Participants
n=66 Participants
|
37 Participants
n=72 Participants
|
75 Participants
n=138 Participants
|
|
Donor Gender
Unknown
|
2 Participants
n=66 Participants
|
0 Participants
n=72 Participants
|
2 Participants
n=138 Participants
|
|
Donor/Recipient Gender Matching
Female Donor, Female Recipient
|
10 Participants
n=66 Participants
|
14 Participants
n=72 Participants
|
24 Participants
n=138 Participants
|
|
Donor/Recipient Gender Matching
Female Donor, Male Recipient
|
16 Participants
n=66 Participants
|
21 Participants
n=72 Participants
|
37 Participants
n=138 Participants
|
|
Donor/Recipient Gender Matching
Male Donor, Female Recipient
|
21 Participants
n=66 Participants
|
10 Participants
n=72 Participants
|
31 Participants
n=138 Participants
|
|
Donor/Recipient Gender Matching
Male Donor, Male Recipient
|
17 Participants
n=66 Participants
|
27 Participants
n=72 Participants
|
44 Participants
n=138 Participants
|
|
Donor/Recipient Gender Matching
Unknown Donor Gender
|
2 Participants
n=66 Participants
|
0 Participants
n=72 Participants
|
2 Participants
n=138 Participants
|
PRIMARY outcome
Timeframe: 6 months and 24 months post-randomizationPopulation: Twelve participants withdrew study consent between 6 and 24 months post-randomization, so are excluded from the analysis of treatment success at 24 months.
Treatment success was evaluated at 6 months in Phase II and is defined as a complete or partial response without secondary systemic immunosuppressive therapy and no recurrent malignancy or death. In Phase III, treatment success was evaluated at 24 months and is defined as a complete response without secondary systemic immunosuppressive therapy and no recurrent malignancy or death.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Proportion of Participants With Treatment Success
Phase II: 6 Months · Yes
|
33 Participants
|
35 Participants
|
|
Proportion of Participants With Treatment Success
Phase II: 6 Months · No
|
33 Participants
|
37 Participants
|
|
Proportion of Participants With Treatment Success
Phase III: 24 Months · Yes
|
9 Participants
|
10 Participants
|
|
Proportion of Participants With Treatment Success
Phase III: 24 Months · No
|
49 Participants
|
58 Participants
|
SECONDARY outcome
Timeframe: 6 months and 24 months post-randomizationOverall survival is defined as survival of death from any cause.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Percentage of Participants With Overall Survival
6 Months
|
85.5 percentage of participants
Interval 74.0 to 92.2
|
91.7 percentage of participants
Interval 82.4 to 96.2
|
|
Percentage of Participants With Overall Survival
24 Months
|
74.0 percentage of participants
Interval 61.1 to 83.2
|
81.5 percentage of participants
Interval 70.3 to 88.8
|
SECONDARY outcome
Timeframe: 6 months and 24 months post-randomizationProgression-free Survival is defined as survival without malignancy relapse. Relapse and death are considered failures for this endpoint.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Percentage of Participants With Progression-free Survival
6 Months
|
84.0 percentage of participants
Interval 72.2 to 91.0
|
90.3 percentage of participants
Interval 80.7 to 95.2
|
|
Percentage of Participants With Progression-free Survival
24 Months
|
67.3 percentage of participants
Interval 53.9 to 77.5
|
78.6 percentage of participants
Interval 67.1 to 86.5
|
SECONDARY outcome
Timeframe: 6 months and 24 months post-randomizationFailure-free Survival is defined as survival without malignancy progression or initiation of secondary therapy for chronic GVHD. Progression, initiation of secondary therapy for chronic GVHD, and death are considered failures for this endpoint.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Percentage of Participants With Failure-free Survival
6 Months
|
74.7 percentage of participants
Interval 62.3 to 81.4
|
73.6 percentage of participants
Interval 61.5 to 79.9
|
|
Percentage of Participants With Failure-free Survival
24 Months
|
46.2 percentage of participants
Interval 33.4 to 58.1
|
48.6 percentage of participants
Interval 36.5 to 59.7
|
SECONDARY outcome
Timeframe: 6 months and 24 months post-randomizationRelapse is defined as recurrence of the primary malignancy. Death is considered a competing risk for this endpoint.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Percentage of Participants With Relapse
6 Months
|
4.8 percentage of participants
Interval 1.3 to 12.2
|
1.4 percentage of participants
Interval 0.1 to 6.7
|
|
Percentage of Participants With Relapse
24 Months
|
14.9 percentage of participants
Interval 7.2 to 25.1
|
10.1 percentage of participants
Interval 4.4 to 18.6
|
SECONDARY outcome
Timeframe: 6 months and 24 months post-randomizationThe percentage of participants initiating secondary immunosuppressive therapy for chronic GVHD is described. Death is considered a competing risk for this endpoint.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Percentage of Participants With Secondary Immunosuppressive Therapy Initiated
6 Months
|
12.9 percentage of participants
Interval 6.0 to 22.5
|
13.9 percentage of participants
Interval 7.1 to 23.0
|
|
Percentage of Participants With Secondary Immunosuppressive Therapy Initiated
24 months
|
29.4 percentage of participants
Interval 18.5 to 41.2
|
38.5 percentage of participants
Interval 27.1 to 49.8
|
SECONDARY outcome
Timeframe: 2 years post-randomizationThe percentage of participants discontinuing all systemic immunosuppressive therapy by two years post-randomization is described. Death is considered a competing risk for this endpoint.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Percentage of Participants With Discontinuation of Systemic Immunosuppressive Therapy at Two Years
|
20 percentage of participants
Interval 10.9 to 31.0
|
23.2 percentage of participants
Interval 14.0 to 33.8
|
SECONDARY outcome
Timeframe: Baseline, 6 months, and 1 year post-randomizationPopulation: Outcomes are analyzed in participants that were alive and completed assessments.
Daily dose of prednisone is described by treatment arm at baseline, 6 months, and 1 year post-randomization.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Prednisone Dose
Baseline
|
0.9 mg/kg/day
Interval 0.2 to 1.2
|
0.9 mg/kg/day
Interval 0.2 to 2.1
|
|
Prednisone Dose
6 Months
|
0.2 mg/kg/day
Interval 0.0 to 1.1
|
0.2 mg/kg/day
Interval 0.0 to 0.8
|
|
Prednisone Dose
1 Year
|
0.2 mg/kg/day
Interval 0.0 to 1.8
|
0.1 mg/kg/day
Interval 0.0 to 0.9
|
SECONDARY outcome
Timeframe: 6 months and 1 year post-randomizationPopulation: Outcomes are analyzed in participants that were alive and completed assessments.
Change in the daily dose of prednisone from baseline, the time of randomization, is described by treatment arm at 6 months and 1 year post-randomization.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Change in Prednisone Dose From Baseline
6 Months
|
-0.6 mg/kg/day
Interval -1.1 to 0.3
|
-0.6 mg/kg/day
Interval -1.7 to 0.2
|
|
Change in Prednisone Dose From Baseline
1 Year
|
-0.6 mg/kg/day
Interval -1.0 to 0.8
|
-0.7 mg/kg/day
Interval -1.9 to -0.1
|
SECONDARY outcome
Timeframe: Baseline, 6 months, and 1 year post-randomizationPopulation: Outcomes are analyzed in participants that were alive and completed assessments.
Creatinine level is described by treatment arm at baseline, 6 months, and 1 year post-randomization.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Serum Creatinine Level
Baseline
|
0.9 mg/dL
Interval 0.3 to 1.8
|
0.9 mg/dL
Interval 0.4 to 1.6
|
|
Serum Creatinine Level
6 Months
|
1.0 mg/dL
Interval 0.4 to 2.6
|
1.0 mg/dL
Interval 0.5 to 1.6
|
|
Serum Creatinine Level
1 Year
|
0.9 mg/dL
Interval 0.4 to 2.4
|
0.9 mg/dL
Interval 0.4 to 1.7
|
SECONDARY outcome
Timeframe: 6 months and 1 year post-randomizationPopulation: Outcomes are analyzed in participants that were alive and completed assessments.
Change in creatinine level from baseline, the time of randomization, is described by treatment arm at 6 months and 1 year post-randomization.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Change in Serum Creatinine Level From Baseline
6 Months
|
0.1 mg/dL
Interval -0.8 to 1.8
|
0.1 mg/dL
Interval -0.6 to 1.1
|
|
Change in Serum Creatinine Level From Baseline
1 Year
|
0.0 mg/dL
Interval -0.5 to 1.2
|
0.0 mg/dL
Interval -0.5 to 0.6
|
SECONDARY outcome
Timeframe: Baseline, 6 months, 1 year, and 2 years post-randomizationPopulation: Outcomes are analyzed in participants that were alive and completed assessments.
Each patient's perception of the severity of the chronic GVHD was collected at baseline and at 6 months, 1 year, and 2 years post-randomization. Severity is categorized as none, mild, moderate, and severe.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Patient-reported Chronic GVHD Severity
Baseline · None
|
0 Participants
|
0 Participants
|
|
Patient-reported Chronic GVHD Severity
Baseline · Mild
|
21 Participants
|
20 Participants
|
|
Patient-reported Chronic GVHD Severity
Baseline · Moderate
|
23 Participants
|
32 Participants
|
|
Patient-reported Chronic GVHD Severity
Baseline · Severe
|
10 Participants
|
10 Participants
|
|
Patient-reported Chronic GVHD Severity
Baseline · Relapsed
|
0 Participants
|
0 Participants
|
|
Patient-reported Chronic GVHD Severity
Baseline · Initiated Secondary Therapy
|
0 Participants
|
0 Participants
|
|
Patient-reported Chronic GVHD Severity
Baseline · Dead
|
0 Participants
|
0 Participants
|
|
Patient-reported Chronic GVHD Severity
6 Months · None
|
4 Participants
|
6 Participants
|
|
Patient-reported Chronic GVHD Severity
6 Months · Mild
|
13 Participants
|
25 Participants
|
|
Patient-reported Chronic GVHD Severity
6 Months · Moderate
|
15 Participants
|
10 Participants
|
|
Patient-reported Chronic GVHD Severity
6 Months · Severe
|
2 Participants
|
3 Participants
|
|
Patient-reported Chronic GVHD Severity
6 Months · Relapsed
|
3 Participants
|
1 Participants
|
|
Patient-reported Chronic GVHD Severity
6 Months · Initiated Secondary Therapy
|
7 Participants
|
14 Participants
|
|
Patient-reported Chronic GVHD Severity
6 Months · Dead
|
6 Participants
|
4 Participants
|
|
Patient-reported Chronic GVHD Severity
1 Year · None
|
9 Participants
|
2 Participants
|
|
Patient-reported Chronic GVHD Severity
1 Year · Mild
|
13 Participants
|
23 Participants
|
|
Patient-reported Chronic GVHD Severity
1 Year · Moderate
|
4 Participants
|
3 Participants
|
|
Patient-reported Chronic GVHD Severity
1 Year · Severe
|
0 Participants
|
4 Participants
|
|
Patient-reported Chronic GVHD Severity
1 Year · Relapsed
|
5 Participants
|
3 Participants
|
|
Patient-reported Chronic GVHD Severity
1 Year · Initiated Secondary Therapy
|
15 Participants
|
21 Participants
|
|
Patient-reported Chronic GVHD Severity
1 Year · Dead
|
7 Participants
|
4 Participants
|
|
Patient-reported Chronic GVHD Severity
2 Years · None
|
2 Participants
|
6 Participants
|
|
Patient-reported Chronic GVHD Severity
2 Years · Mild
|
10 Participants
|
13 Participants
|
|
Patient-reported Chronic GVHD Severity
2 Years · Moderate
|
2 Participants
|
1 Participants
|
|
Patient-reported Chronic GVHD Severity
2 Years · Severe
|
0 Participants
|
0 Participants
|
|
Patient-reported Chronic GVHD Severity
2 Years · Relapsed
|
8 Participants
|
5 Participants
|
|
Patient-reported Chronic GVHD Severity
2 Years · Initiated Secondary Therapy
|
18 Participants
|
28 Participants
|
|
Patient-reported Chronic GVHD Severity
2 Years · Dead
|
7 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Baseline, 6 months, 1 year, and 2 years post-randomizationPopulation: Outcomes are analyzed in participants that were alive and whose providers completed assessments.
Each patient's care provider's perception of the severity of the chronic GVHD was collected at baseline and at 6 months, 1 year, and 2 years post-randomization. Severity is categorized as none, mild, moderate, and severe.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Provider-reported Chronic GVHD Severity
1 Year · None
|
8 Participants
|
7 Participants
|
|
Provider-reported Chronic GVHD Severity
1 Year · Mild
|
15 Participants
|
19 Participants
|
|
Provider-reported Chronic GVHD Severity
Baseline · None
|
1 Participants
|
0 Participants
|
|
Provider-reported Chronic GVHD Severity
Baseline · Mild
|
13 Participants
|
17 Participants
|
|
Provider-reported Chronic GVHD Severity
Baseline · Moderate
|
46 Participants
|
48 Participants
|
|
Provider-reported Chronic GVHD Severity
Baseline · Severe
|
3 Participants
|
7 Participants
|
|
Provider-reported Chronic GVHD Severity
Baseline · Relapsed
|
0 Participants
|
0 Participants
|
|
Provider-reported Chronic GVHD Severity
Baseline · Initiated Secondary Therapy
|
0 Participants
|
0 Participants
|
|
Provider-reported Chronic GVHD Severity
Baseline · Dead
|
0 Participants
|
0 Participants
|
|
Provider-reported Chronic GVHD Severity
6 Months · None
|
6 Participants
|
2 Participants
|
|
Provider-reported Chronic GVHD Severity
6 Months · Mild
|
27 Participants
|
33 Participants
|
|
Provider-reported Chronic GVHD Severity
6 Months · Moderate
|
10 Participants
|
13 Participants
|
|
Provider-reported Chronic GVHD Severity
6 Months · Severe
|
0 Participants
|
0 Participants
|
|
Provider-reported Chronic GVHD Severity
6 Months · Relapsed
|
3 Participants
|
1 Participants
|
|
Provider-reported Chronic GVHD Severity
6 Months · Initiated Secondary Therapy
|
7 Participants
|
14 Participants
|
|
Provider-reported Chronic GVHD Severity
6 Months · Dead
|
6 Participants
|
4 Participants
|
|
Provider-reported Chronic GVHD Severity
1 Year · Moderate
|
5 Participants
|
9 Participants
|
|
Provider-reported Chronic GVHD Severity
1 Year · Severe
|
1 Participants
|
1 Participants
|
|
Provider-reported Chronic GVHD Severity
1 Year · Relapsed
|
5 Participants
|
3 Participants
|
|
Provider-reported Chronic GVHD Severity
1 Year · Initiated Secondary Therapy
|
15 Participants
|
21 Participants
|
|
Provider-reported Chronic GVHD Severity
1 Year · Dead
|
7 Participants
|
4 Participants
|
|
Provider-reported Chronic GVHD Severity
2 Years · None
|
7 Participants
|
7 Participants
|
|
Provider-reported Chronic GVHD Severity
2 Years · Mild
|
10 Participants
|
11 Participants
|
|
Provider-reported Chronic GVHD Severity
2 Years · Moderate
|
1 Participants
|
4 Participants
|
|
Provider-reported Chronic GVHD Severity
2 Years · Severe
|
0 Participants
|
0 Participants
|
|
Provider-reported Chronic GVHD Severity
2 Years · Relapsed
|
8 Participants
|
5 Participants
|
|
Provider-reported Chronic GVHD Severity
2 Years · Initiated Secondary Therapy
|
18 Participants
|
28 Participants
|
|
Provider-reported Chronic GVHD Severity
2 Years · Dead
|
7 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Baseline, 6 months, 1 year, and 2 years post-randomizationPopulation: Outcomes are analyzed in participants that were alive and whose clinical assessments were completed.
Chronic GVHD severity was determined at baseline and at 6 months, 1 year, and 2 years post-randomization per the 2005 NIH Consensus Criteria (Filipovich et al. 2005). Severity is categorized as none, mild, moderate, and severe.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
NIH Consensus Criteria Chronic GVHD Severity
Baseline · None
|
0 Participants
|
1 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
Baseline · Mild
|
1 Participants
|
1 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
Baseline · Moderate
|
29 Participants
|
23 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
Baseline · Severe
|
33 Participants
|
46 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
Baseline · Relapsed
|
0 Participants
|
0 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
Baseline · Initiated Secondary Therapy
|
0 Participants
|
0 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
Baseline · Dead
|
0 Participants
|
0 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
6 Months · None
|
3 Participants
|
1 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
6 Months · Mild
|
3 Participants
|
3 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
6 Months · Moderate
|
25 Participants
|
27 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
6 Months · Severe
|
12 Participants
|
17 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
6 Months · Relapsed
|
3 Participants
|
1 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
6 Months · Initiated Secondary Therapy
|
7 Participants
|
14 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
6 Months · Dead
|
6 Participants
|
4 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
1 Year · None
|
2 Participants
|
3 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
1 Year · Mild
|
2 Participants
|
5 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
1 Year · Moderate
|
16 Participants
|
16 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
1 Year · Severe
|
9 Participants
|
10 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
1 Year · Relapsed
|
5 Participants
|
3 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
1 Year · Initiated Secondary Therapy
|
15 Participants
|
21 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
1 Year · Dead
|
7 Participants
|
4 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
2 Years · None
|
9 Participants
|
9 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
2 Years · Mild
|
6 Participants
|
12 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
2 Years · Moderate
|
8 Participants
|
8 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
2 Years · Severe
|
2 Participants
|
2 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
2 Years · Relapsed
|
8 Participants
|
5 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
2 Years · Initiated Secondary Therapy
|
18 Participants
|
28 Participants
|
|
NIH Consensus Criteria Chronic GVHD Severity
2 Years · Dead
|
7 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Baseline, 2 months, 6 months, 1 year, and 2 years post-randomizationPopulation: Outcomes are analyzed in participants that were alive and completed assessments.
The Medical Outcome Study SF-36 Physical Component Summary (PCS) is a subscale of the SF-36 intended to measure physical well-being. It is scored on a scale of 0-100, with higher scores indicating higher levels of well-being.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
SF-36 Physical Component Summary
Baseline
|
38 score on a scale
Standard Error 1.3
|
38 score on a scale
Standard Error 1.2
|
|
SF-36 Physical Component Summary
2 Months
|
39 score on a scale
Standard Error 1.6
|
42 score on a scale
Standard Error 1.6
|
|
SF-36 Physical Component Summary
6 Months
|
38 score on a scale
Standard Error 2.1
|
43 score on a scale
Standard Error 1.5
|
|
SF-36 Physical Component Summary
1 Year
|
40 score on a scale
Standard Error 1.8
|
43 score on a scale
Standard Error 1.7
|
|
SF-36 Physical Component Summary
2 Years
|
44 score on a scale
Standard Error 2.4
|
43 score on a scale
Standard Error 2.0
|
SECONDARY outcome
Timeframe: Baseline, 2 months, 6 months, 1 year, and 2 years post-randomizationPopulation: Outcomes are analyzed in participants that were alive and completed assessments.
The Medical Outcome Study SF-36 Mental Component Summary (MCS) is a subscale of the SF-36 intended to measure mental well-being. It is scored on a scale of 0-100, with higher scores indicating higher levels of well-being.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
SF-36 Mental Component Summary
Baseline
|
47 score on a scale
Standard Error 1.3
|
48 score on a scale
Standard Error 1.4
|
|
SF-36 Mental Component Summary
2 Months
|
49 score on a scale
Standard Error 1.4
|
50 score on a scale
Standard Error 1.5
|
|
SF-36 Mental Component Summary
6 Months
|
50 score on a scale
Standard Error 1.5
|
48 score on a scale
Standard Error 1.6
|
|
SF-36 Mental Component Summary
1 Year
|
50 score on a scale
Standard Error 1.7
|
46 score on a scale
Standard Error 2.1
|
|
SF-36 Mental Component Summary
2 Years
|
52 score on a scale
Standard Error 1.7
|
50 score on a scale
Standard Error 1.6
|
SECONDARY outcome
Timeframe: Baseline, 2 months, 6 months, 1 year, and 2 years post-randomizationPopulation: Outcomes are analyzed in participants that were alive and completed assessments.
The Functional Assessment of Cancer Therapy-Bone Marrow Transplant scale (FACT-BMT) is a quality of life instrument that assesses the effects of bone marrow transplantation (BMT) on a patient's physical, social/family, emotional, and functional well-being while taking into consideration BMT-specific concerns. The assessment has 37 questions, each scored on a Likert scale from 0-4. The overall score is computed by adding scores of the questions and falls in the range 0-148, with higher scores indicating higher levels of overall well-being.
Outcome measures
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 Participants
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 Participants
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
FACT-BMT Score
Baseline
|
103 score on a scale
Standard Error 2.6
|
104 score on a scale
Standard Error 2.5
|
|
FACT-BMT Score
2 Months
|
105 score on a scale
Standard Error 2.8
|
110 score on a scale
Standard Error 2.4
|
|
FACT-BMT Score
6 Months
|
109 score on a scale
Standard Error 3.2
|
109 score on a scale
Standard Error 3.4
|
|
FACT-BMT Score
1 Year
|
114 score on a scale
Standard Error 2.6
|
110 score on a scale
Standard Error 3.6
|
|
FACT-BMT Score
2 Years
|
119 score on a scale
Standard Error 3.8
|
113 score on a scale
Standard Error 3.7
|
Adverse Events
Sirolimus, Calcineurin Inhibitor, and Prednisone
Serious events: 13 serious events
Other events: 0 other events
Deaths: 16 deaths
Sirolimus and Prednisone
Serious events: 12 serious events
Other events: 0 other events
Deaths: 13 deaths
Serious adverse events
| Measure |
Sirolimus, Calcineurin Inhibitor, and Prednisone
n=66 participants at risk
Sirolimus + calcineurin inhibitor + prednisone
Sirolimus + calcineurin inhibitor + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
The target serum level for tacrolimus is 5-10 ng/mL. The target serum level for cyclosporine is 120-200 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
Sirolimus and Prednisone
n=72 participants at risk
Sirolimus + prednisone
Sirolimus + prednisone: The target serum level for sirolimus is 3-12 ng/mL.
Prednisone is administered initially as a single early morning dose of 1 mg/kg/day (or equivalent). If prednisone at a dose of 1 mg/kg/day (or equivalent) is contraindicated, patients may begin prednisone between 0.5-1 mg/kg/day.
|
|---|---|---|
|
Blood and lymphatic system disorders
Thrombotic thrombocytopenic purpura
|
1.5%
1/66 • Number of events 1 • Three years post-randomization
|
0.00%
0/72 • Three years post-randomization
|
|
Cardiac disorders
Bradycardia
|
0.00%
0/66 • Three years post-randomization
|
1.4%
1/72 • Number of events 1 • Three years post-randomization
|
|
Cardiac disorders
Cardiac fibrillation
|
1.5%
1/66 • Number of events 1 • Three years post-randomization
|
0.00%
0/72 • Three years post-randomization
|
|
Cardiac disorders
Coronary artery disease
|
0.00%
0/66 • Three years post-randomization
|
1.4%
1/72 • Number of events 1 • Three years post-randomization
|
|
Cardiac disorders
Myocardial infarction
|
1.5%
1/66 • Number of events 1 • Three years post-randomization
|
1.4%
1/72 • Number of events 1 • Three years post-randomization
|
|
Gastrointestinal disorders
Gastric ulcer
|
1.5%
1/66 • Number of events 1 • Three years post-randomization
|
0.00%
0/72 • Three years post-randomization
|
|
Hepatobiliary disorders
Cholecystitis
|
1.5%
1/66 • Number of events 1 • Three years post-randomization
|
0.00%
0/72 • Three years post-randomization
|
|
Infections and infestations
Sepsis
|
3.0%
2/66 • Number of events 3 • Three years post-randomization
|
1.4%
1/72 • Number of events 1 • Three years post-randomization
|
|
Injury, poisoning and procedural complications
Hip fracture
|
1.5%
1/66 • Number of events 1 • Three years post-randomization
|
0.00%
0/72 • Three years post-randomization
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma
|
1.5%
1/66 • Number of events 1 • Three years post-randomization
|
0.00%
0/72 • Three years post-randomization
|
|
Nervous system disorders
Cerebrovascular accident
|
0.00%
0/66 • Three years post-randomization
|
1.4%
1/72 • Number of events 1 • Three years post-randomization
|
|
Nervous system disorders
Loss of consciousness
|
0.00%
0/66 • Three years post-randomization
|
1.4%
1/72 • Number of events 1 • Three years post-randomization
|
|
Nervous system disorders
Neuropathy peripheral
|
0.00%
0/66 • Three years post-randomization
|
1.4%
1/72 • Number of events 1 • Three years post-randomization
|
|
Nervous system disorders
Syncope
|
1.5%
1/66 • Number of events 1 • Three years post-randomization
|
2.8%
2/72 • Number of events 2 • Three years post-randomization
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
1.5%
1/66 • Number of events 1 • Three years post-randomization
|
0.00%
0/72 • Three years post-randomization
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/66 • Three years post-randomization
|
1.4%
1/72 • Number of events 1 • Three years post-randomization
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
1.5%
1/66 • Number of events 1 • Three years post-randomization
|
0.00%
0/72 • Three years post-randomization
|
|
Surgical and medical procedures
Splenectomy
|
0.00%
0/66 • Three years post-randomization
|
1.4%
1/72 • Number of events 1 • Three years post-randomization
|
|
Vascular disorders
Deep vein thrombosis
|
1.5%
1/66 • Number of events 1 • Three years post-randomization
|
1.4%
1/72 • Number of events 1 • Three years post-randomization
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place