Stem Cell Transplant for Sickle Cell Disease or Thalassemia Major

The study will enroll male and femalepatients between 2 years to 25 years of age with sickle cell disease or beta thalassemiamajor, who do not have a matched related donor, and their doctor feels theircondition should be treated with hematopoietic stem cell transplantation (HSCT).        

·       -For Sickle Cell Disease, individuals must have significant disease. We would consider you for transplantif you have had severe neurologic symptoms including stroke, frequentvaso-occlusive events (examples include pain episodes requiring pain managementor acute chest syndrome), or administration of regular and frequent red bloodcell transfusions to prevent disease complications. 

·       -For Beta-Thalassemia Major, you would be considered acandidate for transplant if you have genetic test confirming Beta Thalassemiaand have required 8 or more transfusions per year in the past two years.

The purpose of this research study isto test a new method of cell processing for stem cell transplants with aclosely matched unrelated donor. The new method is called alpha/beta T cell andB cell depletion using a device called CliniMACs. We want to see if this methodcan reduce some of the complications of the transplant and decrease the time ittakes for the new stem cells to establish a new immune system. The use of theCliniMACs device for alpha/beta T and B cell depletion is experimental and isconsidered research.