Trial Outcomes & Findings for Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders (NCT NCT01043640)
NCT ID: NCT01043640
Last Updated: 2018-02-05
Results Overview
Donor derived engraftment is defined as 80 percent or greater donor cells in the recipient's bone marrow and blood cells.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
46 participants
Primary outcome timeframe
Day 100 Post Transplant
Results posted on
2018-02-05
Participant Flow
Participant milestones
| Measure |
Transplant Patients
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Overall Study
STARTED
|
46
|
|
Overall Study
COMPLETED
|
46
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Baseline characteristics by cohort
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Age, Categorical
<=18 years
|
46 Participants
n=93 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=93 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
35 Participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Day 100 Post TransplantDonor derived engraftment is defined as 80 percent or greater donor cells in the recipient's bone marrow and blood cells.
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Number of Patients With Donor Derived Engraftment
|
42 Participants
|
SECONDARY outcome
Timeframe: Day 100 Post TransplantGVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)
|
32 Participants
|
SECONDARY outcome
Timeframe: Day 100 Post TransplantGVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD)
|
2 Participants
|
SECONDARY outcome
Timeframe: Day 100 Post TransplantGVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD)
|
5 Participants
|
SECONDARY outcome
Timeframe: Day 100 Post TransplantGVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD)
|
2 Participants
|
SECONDARY outcome
Timeframe: Day 100 Post TransplantGVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD)
|
5 Participants
|
SECONDARY outcome
Timeframe: By Day 100 Post TransplantPeri-transplant is defined as within 100 days of transplant.
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Number of Patients Who Died Peri-Transplant
|
2 Participants
|
SECONDARY outcome
Timeframe: Day 28Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Donor Cell Chimerism Following Transplant
|
95 percentage of donor cells
Standard Deviation 9
|
SECONDARY outcome
Timeframe: Day 42Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Donor Cell Chimerism Following Transplant
|
93 percentage of donor cells
Standard Deviation 15
|
SECONDARY outcome
Timeframe: Day 100Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Donor Cell Chimerism Following Transplant
|
90 percentage of donor cells
Standard Deviation 26
|
SECONDARY outcome
Timeframe: 6 monthsDonor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Donor Cell Chimerism Following Transplant
|
94 percentage of donor cells
Standard Deviation 22
|
SECONDARY outcome
Timeframe: One yearDonor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
Outcome measures
| Measure |
Transplant Patients
n=46 Participants
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Donor Cell Chimerism Following Transplant
|
99 percentage of donor cells
Standard Deviation 4
|
Adverse Events
Transplant Patients
Serious events: 8 serious events
Other events: 40 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Transplant Patients
n=46 participants at risk
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Cardiac disorders
Pericardial Effusion
|
4.3%
2/46
|
|
Renal and urinary disorders
Renal Failure
|
2.2%
1/46
|
|
Gastrointestinal disorders
Diarrhea
|
4.3%
2/46
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
2.2%
1/46
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Veno-Occlusive Disease
|
2.2%
1/46
|
|
Cardiac disorders
Cardiac Arrest
|
2.2%
1/46
|
|
Nervous system disorders
Intracerebral Hemorrhage
|
2.2%
1/46
|
Other adverse events
| Measure |
Transplant Patients
n=46 participants at risk
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if \> 12 kg Allogeneic stem cell transplantation: \> 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day -3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is \> 40 kg
Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:
Stop MMF at Day +42 or 7 days after engraftment achieved (ANC\>500 x 10\^6 neutrophils/L x 3 days and chimerism \>90%), whichever is later.
|
|---|---|
|
Metabolism and nutrition disorders
Anorexia
|
15.2%
7/46
|
|
Investigations
Bilirubin Increased
|
34.8%
16/46
|
|
Gastrointestinal disorders
Diarrhea
|
8.7%
4/46
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
30.4%
14/46
|
|
General disorders
Fever
|
23.9%
11/46
|
|
General disorders
Graft Versus Host Disease
|
19.6%
9/46
|
|
Nervous system disorders
Headache
|
6.5%
3/46
|
|
Gastrointestinal disorders
Hemorrhage, Gastrointestinal
|
17.4%
8/46
|
|
Renal and urinary disorders
Hemorrhage, Genitourinary
|
21.7%
10/46
|
|
Skin and subcutaneous tissue disorders
Hives
|
6.5%
3/46
|
|
Vascular disorders
Hypertension
|
45.7%
21/46
|
|
Vascular disorders
Hypotension
|
6.5%
3/46
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
30.4%
14/46
|
|
Infections and infestations
Infection, NOS
|
76.1%
35/46
|
|
Infections and infestations
Infection, Respiratory
|
19.6%
9/46
|
|
Gastrointestinal disorders
Mucositis
|
23.9%
11/46
|
|
Gastrointestinal disorders
Nausea
|
13.0%
6/46
|
|
Cardiac disorders
Pericardial Effusion
|
15.2%
7/46
|
|
Skin and subcutaneous tissue disorders
Rash
|
21.7%
10/46
|
|
Cardiac disorders
Tachycardia
|
6.5%
3/46
|
|
Gastrointestinal disorders
Vomiting
|
8.7%
4/46
|
|
Infections and infestations
Infection, Gastrointestinal
|
15.2%
7/46
|
Additional Information
Dr. Paul Orchard
Masonic Cancer Center, University of Minnesota
Phone: 612-626-2313
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place