Allogeneic Stem Cell Transplantation Following Nonmyeloablative Chemotherapy in Patients With Hemoglobinopathies
Hemoglobinopathies, such as sickle cell disease and thalassemia major, are genetic diseases
associated with significant morbidity and premature death. Allogeneic bone marrow
transplantation (BMT) is the only potential cure for severe hemoglobinopathies. Typical
regimens have used high doses of chemotherapy or chemo-radiotherapy to ablate recipient
hematopoiesis and to prevent graft rejection. The widespread use of this treatment has been
limited by toxicity, risk of end-organ damage, and donor availability. This study will use
a nonmyeloablative regimen of fludarabine and busulfan to attempt to generate consistent
engraftment with donor hematopoietic stem cells in patients with severe hemoglobinopathy.
G-CSF mobilization of the donor's peripheral blood white blood cells will precede donor
apheresis. A nonmyeloablative conditioning regimen of fludarabine and busulfan will be
administered to patients prior to allogeneic peripheral blood stem cell infusions. FK506
and prednisone will be administered for graft versus host disease (GVHD) prophylaxis.
Patients will be evaluated for engraftment, donor: host hematopoietic chimerism, toxicity,
and hemoglobinopathy.
Interventional
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Evidence of engraftment of donor hematopoietic cells following administration of low doses of busulfan and fludarabine
Throughout study
No
Catherine J. Wu, MD
Principal Investigator
Dana Farber Cancer Institute/Harvard Medical School
United States: Federal Government
DAIT DF/HCC 01-098
NCT00034528
September 2001
November 2003
Name | Location |
---|---|
Dana-Farber Cancer Institute/Harvard Cancer Center, Brigham and Women's Hospital and Massachusetts General Hospital | Boston, Massachusetts 02115 |