Multi-Center Study Using Allogeneic Stem Cell Transplantation Following Reduced Intensity Chemotherapy in Patients With Hemoglobinopathies
- In order to undergo transplant procedure, patients will be admitted to the hospital for
approximately 10-14 days.
- To prepare patient's bone marrow to accept donor stem cells, they will receive
fludarabine and busulfex. Fludarabine will be given intravenously once daily for 4
days. Busulfex will be given once daily for the same 4 days.
- One day before patients receive busulfex and fludarabine, they will also be given
alemtuzumab intravenously once daily for 5 days.
- Three days after the end of chemotherapy, patients will receive the infusion of donor
- If patients have thalassemia, they will receive subcutaneous injections of filgrastim
starting on day one after the donor stem cell transfusion and will continue receiving
filgrastim every day until it appears that the donor stem cells have been accepted. If
the patient has sickle cell disease, filgrastim will not be given,
- Additional drugs will be given to help prevent infection (i.e. antibiotics).
- After stem cell infusion patients will be examined and have blood tests weekly for 1
month. Bone marrow biopsies, and blood work will also be performed 1 month, 3 months,
6 months and 1 year after stem cell infusion.
- Patients will be on the study for about 12 months. After study is completed progress
will be monitored on an annual basis.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.
Outcome was measured by ANC >500 for three consecutive days prior to day 30 after PBSC infusion, >25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods prior to day 45 after PBSC infusion and >25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods after day 180 after PBSC infusion.
Catherine J. Wu, MD
Dana-Farber Cancer Institute
United States: Institutional Review Board
|Beth Israel Deaconess Medical Center||Boston, Massachusetts 02215|
|Dana-Farber Cancer Institute||Boston, Massachusetts 02115|
|Massachusetts General Hospital||Boston, Massachusetts 02114-2617|
|Winship Cancer Institute-Emory University||Atlanta, Georgia 30322|
|Feist-Weiller Cancer Center-LSU||Shreveport, Louisiana 71130|
|Ohio State University College of Medicine||Columbus, Ohio 43210|