Stem Cell Transplant for Sickle Cell Disease or Thalassemia Major

Description

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

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

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

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

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