Our primary objective is to determine if it is feasible for previously untreated severe aplastic anemia (SAA) patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide.
This is a clinical trial of upfront bone marrow transplantation for patients with SAA who do not have a fully human leukocyte antigen (HLA) matched donor. The trial uses a conditioning regimen which has been successful in the refractory and relapsed setting to maximize engraftment and post transplant therapy to minimize graft versus host disease (GVHD). This would be used here in patients who have not yet undergone immunosuppressive therapy for their SAA or are thought to be unlikely to respond to immunosuppressive therapy for SAA.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
21
Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily
Days -6 through -2: 30 mg/m\^2 IV daily
Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States
Overall Survival and Engraftment at One Year
Number of enrolled participants who receive BMT, achieve engraftment, and are alive at one year post bone marrow transplant (BMT).
Time frame: 1 year
Overall Survival at One Year
Number of participants alive at one year after BMT.
Time frame: 1 year
Probability of Neutrophil Recovery as Assessed by the Number of Participants Who Have Recovered Neutrophil Counts
Probability of neutrophil recovery will be assessed by the number of participants who have recovered neutrophil counts at 1 year (\>500 ANC).
Time frame: 1 year
Probability of Platelet Recovery as Assessed by Number of Participants Who Have Recovered Platelet Counts
Probability of platelet recovery will be assessed by the number of participants who have recovered platelet counts at 1 year.
Time frame: 1 year
Number of Participants Who Experience Primary Graft Failure
Number of participants who experience primary graft failure by one year after BMT.
Time frame: 1 year
Number of Participants Who Experience Secondary Graft Failure
Number of participants who experience secondary graft failure by one year after BMT.
Time frame: 1 year
Number of Participants Who Experience Grades II-IV Acute GVHD
Number of participants who experience grade II, III, or IV acute GVHD by Day 100.
Time frame: Day 100
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Day -1: 200 centigray (cGy) in a single fraction
Start on Day 5 through Day 365
Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
Number of Participants Who Experience Grades III-IV Acute GVHD
Number of participants who experience grade III or IV acute GVHD by Day 100.
Time frame: Day 100
Number of Participants Who Experience Chronic GVHD
Number of participants who experience chronic GVHD by two years after BMT.
Time frame: 2 years
Number of Participants With Full Donor Chimerism
Number of participants with full donor chimerism at Day 60.
Time frame: Day 60
GVHD-free Relapse-free Survival (GRFS)
Number of participants alive, without relapse, and without GVHD at 1 year.
Time frame: 1 year
Transplant-related Mortality
Number of participants deceased for reasons related to BMT at 1 year.
Time frame: 1 year