Trial Outcomes & Findings for Azacytidine Prior to in Vivo T-cell Depleted Allo Stem Cell Transplant for Patients With Myeloid Malignancies in CR (NCT NCT02497404)
NCT ID: NCT02497404
Last Updated: 2021-09-22
Results Overview
Number of participants without evidence of progression of underlying malignancy for which the transplant was performed, assessed at 1 year post-transplant.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
40 participants
Primary outcome timeframe
1 year post-transplant
Results posted on
2021-09-22
Participant Flow
Participant milestones
| Measure |
5 Azacytidine
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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Overall Study
STARTED
|
40
|
|
Overall Study
COMPLETED
|
39
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
5 Azacytidine
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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Overall Study
Adverse Event
|
1
|
Baseline Characteristics
One participant was removed from the study before stem cell transplantation and is therefore unevaluable for this baseline measure
Baseline characteristics by cohort
| Measure |
5 Azacytidine
n=40 Participants
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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Age, Categorical
<=18 years
|
0 Participants
n=40 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
28 Participants
n=40 Participants
|
|
Age, Categorical
>=65 years
|
12 Participants
n=40 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=40 Participants
|
|
Sex: Female, Male
Male
|
19 Participants
n=40 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=40 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
36 Participants
n=40 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=40 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=40 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=40 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=40 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=40 Participants
|
|
Race (NIH/OMB)
White
|
35 Participants
n=40 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=40 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=40 Participants
|
|
Region of Enrollment
United States
|
40 participants
n=40 Participants
|
|
Disease
Acute Myeloid Leukemia
|
34 Participants
n=40 Participants
|
|
Disease
Myelodysplastic Syndrome/Myeloproliferative Disease
|
6 Participants
n=40 Participants
|
|
Donor Relationship
Matched Related Donor
|
17 Participants
n=39 Participants • One participant was removed from the study before stem cell transplantation and is therefore unevaluable for this baseline measure
|
|
Donor Relationship
Matched Unrelated Donor
|
22 Participants
n=39 Participants • One participant was removed from the study before stem cell transplantation and is therefore unevaluable for this baseline measure
|
PRIMARY outcome
Timeframe: 1 year post-transplantPopulation: 1 participant was unevaluable as they were removed from study before undergoing hematopoietic stem cell transplant.
Number of participants without evidence of progression of underlying malignancy for which the transplant was performed, assessed at 1 year post-transplant.
Outcome measures
| Measure |
5 Azacytidine
n=39 Participants
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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Disease Free Survival at 1 Year Post-transplant
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18 Participants
|
SECONDARY outcome
Timeframe: 6 months post-transplantPopulation: 1 participant was unevaluable as they were removed from study before undergoing hematopoietic stem cell transplant.
Number of participants without evidence of progression of underlying malignancy for which the transplant was performed, assessed at 6 months post-transplant
Outcome measures
| Measure |
5 Azacytidine
n=39 Participants
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
|
Disease Free Survival at 6 Months Post-transplant
|
25 Participants
|
SECONDARY outcome
Timeframe: 2 years post-transplantPopulation: 1 participant was unevaluable as they were removed from study before undergoing hematopoietic stem cell transplant.
Number of participants without evidence of progression of underlying malignancy for which the transplant was performed, assessed at 2 years post-transplant.
Outcome measures
| Measure |
5 Azacytidine
n=39 Participants
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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Disease Free Survival at 2 Years Post-transplant
|
13 Participants
|
SECONDARY outcome
Timeframe: 6 months post-transplantPopulation: 1 participant was unevaluable as they were removed from study before undergoing hematopoietic stem cell transplant.
Number of participants alive at 6 months post-transplant
Outcome measures
| Measure |
5 Azacytidine
n=39 Participants
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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Overall Survival at 6 Months Post-transplant
|
35 Participants
|
SECONDARY outcome
Timeframe: 1 year post-transplantPopulation: 1 participant was unevaluable as they were removed from study before undergoing hematopoietic stem cell transplant
Number of participants alive at 1 year post-transplant
Outcome measures
| Measure |
5 Azacytidine
n=39 Participants
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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Overall Survival at 1 Year Post-Transplant
|
25 Participants
|
SECONDARY outcome
Timeframe: 2 years post-transplantPopulation: 1 participant was unevaluable as they were removed from study before undergoing hematopoietic stem cell transplant.
Number of participants alive at 2 years post-transplant
Outcome measures
| Measure |
5 Azacytidine
n=39 Participants
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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Overall Survival at 2 Years Post-Transplant
|
15 Participants
|
SECONDARY outcome
Timeframe: 21 days post-transplantPopulation: 1 participant was unevaluable as they were removed from study before undergoing hematopoietic stem cell transplant.
Number of patients who experience graft failure, defined as the absence of neutrophil engraftment by Day +21 or a drop in the absolute neutrophil count to \<0.3 cells/microL for five consecutive days occurring after initial neutrophil engraftment within the first 3 weeks post-transplantation.
Outcome measures
| Measure |
5 Azacytidine
n=39 Participants
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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Graft Failure
|
1 Participants
|
SECONDARY outcome
Timeframe: 2 years post-transplantPopulation: 1 participant was unevaluable as they were removed from study before undergoing hematopoietic stem cell transplant.
Number of patients who develop acute graft-versus-host disease of any grade.
Outcome measures
| Measure |
5 Azacytidine
n=39 Participants
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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Acute Graft-versus-Host Disease (GVHD)
|
13 Participants
|
SECONDARY outcome
Timeframe: 2 years post-transplantPopulation: 1 participant was unevaluable as they were removed from study before undergoing hematopoietic stem cell transplant.
Number of patients who develop high-risk extensive chronic graft-versus-host disease. Extensive chronic GVHD is defined as generalized skin or multiple organ involvement. High risk chronic GVHD is defined as platelet count of less than 100k/microL
Outcome measures
| Measure |
5 Azacytidine
n=39 Participants
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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High-Risk Extensive Chronic Graft-versus-Host-Disease
|
2 Participants
|
Adverse Events
5 Azacytidine
Serious events: 28 serious events
Other events: 38 other events
Deaths: 28 deaths
Serious adverse events
| Measure |
5 Azacytidine
n=40 participants at risk
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
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|---|---|
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Blood and lymphatic system disorders
Atypical Hemolytic Uremic Syndrome
|
10.0%
4/40 • Number of events 4 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Cardiac disorders
Pericardial Effusion
|
10.0%
4/40 • Number of events 4 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Diarrhea
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Small Bowel Obstruction
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Gastroenteritis
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Lower Gastrointestinal Hemorrhage
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Abdominal Pain
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Intra-Abdominal Abscess
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
General disorders
Failure to Thrive
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
General disorders
Hypervolemia
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
General disorders
Fever
|
7.5%
3/40 • Number of events 3 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
General disorders
Infusion-Related Reaction
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Hepatobiliary disorders
Cholecystitis
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Bacteremia
|
7.5%
3/40 • Number of events 3 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Human Herpesvirus 6 Infection
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Cytomegalovirus Infection
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Pneumonia
|
7.5%
3/40 • Number of events 3 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Norovirus Infection
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Upper Respiratory Infection - Influenza B
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Upper Respiratory Infection - Rhinovirus
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Fungal Pneumonia
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Sepsis
|
12.5%
5/40 • Number of events 5 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Bronchial Infection
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Appendicitis
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Fungal Infection
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Conjunctivitis
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Herpes Simplex Virus Infection
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Investigations
Acute Graft-versus-Host Disease
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Investigations
Platelet Count Decreased
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Investigations
Chronic Graft-versus-Host Disease
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Investigations
Post-Transplant Lymphoproliferative Disorder
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous Cell Carcinoma
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Nervous system disorders
Intracranial Hemorrhage
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Nervous system disorders
Stroke
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Nervous system disorders
Posterior Reversible Encephalopathy Syndrome
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Nervous system disorders
Seizure
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Renal and urinary disorders
Acute Kidney Injury
|
15.0%
6/40 • Number of events 6 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Renal and urinary disorders
Renal Failure
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
10.0%
4/40 • Number of events 4 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Distress
|
7.5%
3/40 • Number of events 3 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Edema
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Skin and subcutaneous tissue disorders
Rash Maculo-papular
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Vascular disorders
Hypotension
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Vascular disorders
Hypertension
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Vascular disorders
Thromboembolic Event
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Injury, poisoning and procedural complications
Fall
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
Other adverse events
| Measure |
5 Azacytidine
n=40 participants at risk
Patients will be given a five day course of subcutaneous 5-azacytidine, followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation (TBI) prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor.
5-Azacytidine: Patients will be given a five day course of subcutaneous 5-Azacytidine followed by a reduced intensity conditioning regimen of fludarabine and melphalan with or without total body irradiation prior to an allogeneic hematopoietic stem cell transplantation from a related or unrelated HLA matched donor. AZA 75 mg/m2 subcutaneously daily at the same time on days -11, -10, -9, -8 and -7. This will be administered on an outpatient basis if possible.
Fludarabine (conditioning regimen): Fludarabine will be given at 40 mg/m2 intravenously daily at the same time over 30 minutes on days -6,-5,-4,-3.
Melphalan (conditioning regimen): Melphalan will be given at 140 mg/m2 IV on day -3.
Alemtuzumab (conditioning regimen): Alemtuzumab will be given at 30 mg subcutaneously on Days -4 and -2 for unrelated donors, and Day -2 for related donors.
TBI (conditioning regimen): TBI will be given at 2 doses of 200 cGy each on one day of the conditioning regimen (between days -6 and -3).
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
10.0%
4/40 • Number of events 4 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Diarrhea
|
7.5%
3/40 • Number of events 3 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Nausea
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Mucositis Oral
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Small Bowel Obstruction
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Rectal Pain
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Gastrointestinal disorders
Periapical Abscess
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Hepatobiliary disorders
Hepatosplenic Lesion
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Bacteremia
|
27.5%
11/40 • Number of events 11 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Urinary Tract Infection
|
17.5%
7/40 • Number of events 7 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Upper Respiratory Infection - Parainfluenza
|
20.0%
8/40 • Number of events 8 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Clostridium difficile Colitis
|
30.0%
12/40 • Number of events 12 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Adenovirus Infection
|
7.5%
3/40 • Number of events 3 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Human Herpesvirus 6 Infection
|
32.5%
13/40 • Number of events 13 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Upper Respiratory Infection - Respiratory Synctyial Virus
|
7.5%
3/40 • Number of events 3 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Cytomegalovirus Infection
|
27.5%
11/40 • Number of events 11 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Varicella Zoster Infection
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Upper Respiratory Infection - Influenza B
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Upper Respiratory Infection - Rhinovirus
|
25.0%
10/40 • Number of events 10 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
BK Virus Infection
|
22.5%
9/40 • Number of events 9 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Upper Respiratory Infection - Human metapneumovirus
|
10.0%
4/40 • Number of events 4 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Upper Respiratory Infection - Staphylococcus aureus
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Fungal Pneumonia
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Infectious Diarrhea
|
15.0%
6/40 • Number of events 6 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Upper Respiratory Infection - Influenza A
|
12.5%
5/40 • Number of events 5 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Upper Respiratory Infection - SARS-CoV-2
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Upper Respiratory Infection - Coronavirus
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Central Line Infection
|
10.0%
4/40 • Number of events 4 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Fungal Infection
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Infections and infestations
Epstein Barr Virus Infection
|
22.5%
9/40 • Number of events 9 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Investigations
Acute Graft-versus-Host Disease
|
30.0%
12/40 • Number of events 12 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Investigations
Platelet count decreased
|
7.5%
3/40 • Number of events 3 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Investigations
Neutrophil count decreased
|
7.5%
3/40 • Number of events 3 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Investigations
Creatinine increased
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Investigations
Chronic Graft-versus-Host Disease
|
12.5%
5/40 • Number of events 5 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Investigations
Aspartate Aminotransferase Increased
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Metabolism and nutrition disorders
Anorexia
|
7.5%
3/40 • Number of events 3 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Metabolism and nutrition disorders
Hyponatremia
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Metabolism and nutrition disorders
Acidosis
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Psychiatric disorders
Delirium
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Renal and urinary disorders
Acute Kidney Injury
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Renal and urinary disorders
Urinary Retention
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
5.0%
2/40 • Number of events 2 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Skin and subcutaneous tissue disorders
Rash Maculo-papular
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Vascular disorders
Hypotension
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
|
Vascular disorders
Hypertension
|
2.5%
1/40 • Number of events 1 • Adverse events were collected for all patients from start of 5-azacytidine therapy through 5 years post-transplant or when the participant was removed from the study, whichever was sooner.
All adverse events were first documented by the study transplant physicians and thereafter collated and graded by two independent reviewed according to CTCAE version 4.0. Per protocol, events reported were all infectious events and Grade 3 and higher events for non-infectious events
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place