Clinical Trial
Study Details
Alemtuzumab (Campath IH) is given daily over first 4 days, Day -20 to Day -17
Fludarabine 35/m2 is given daily over 4 days on Day -7 to Day -4.
Melphalan 70mg/m2 is given daily over 2 days on Day -3 to Day -2.
Immunosuppressant to prevent graft vs host disease is given on Day -1 prior to stem cell infusion
Immunosuppressant to prevent graft vs host disease is given on Day -1.
Immunosuppressant to prevent graft vs host disease is given Day -1 prior to stem cell infusion
Human Leukocyte Antigen (HLA) matched or mismatched; related or unrelated hematopoietic stem cells to be transplanted on Day 0.
This study will evaluate the use of reduced intensity conditioning regimen in patients with high risk hemoglobinopathy Sickle Cell and B-Thalassemia Major in combination with standard immunosuppressive medications, followed by a routine stem cell transplant in order to assess whether or not it is as effective as myeloablative high dose chemotherapy and transplant.
Study Overview
Standard myeloablative regimens are toxic to non-hematopoietic tissue and are associated with treatment related mortality and morbidity (TRM). Preparative regimens that are not myeloablative are associated with a greatly decreased incidence of TRM. In addition to providing a less toxic regimen, the reduced intensity chemotherapy preparative regimen also remains immunosuppressive enough to allow donor engraftment. Recent report of non-myeloablative regimens which resulted in engraftment of allogeneic stem cell in hematological malignancies raises the possibility that this conditioning regimen might be useful in achieving engraftment in non hematological disorder.
In an effort to achieve stable engraftment with any suitable donor stem cell source and to minimize toxicity the investigators have developed a new reduced intensity conditioning regimen for high risk hemoglobinopathies with the main aim of significantly suppressing the recipient's immune system and facilitate engraftment.
Non-myeloablative or reduced-intensity immunosuppressive preparative regimens have achieved a stable, mixed chimerism engraftment and successful allogeneic bone marrow transplants.
Contacts and Locations
Cohen Children's Medical Center of New York
New Hyde Park, New York 11040
United States