Stem Cell Transplant for Sickle Cell Disease or Thalassemia Major
Contact
Description
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.
Eligibility and criteria
What to expect
Patients will receive a blood stem cell
transplant. Blood stem cells will be taken from an unrelated donor and processed
with the CliniMACs device for alpha/beta T cell and CD19+ B cell depletions.
For the first 2-3 months after the transplant, patients will be seen about once weekly. This is considered standard of care for anyone
who has had a stem cell transplant. After this period, we will see patients yearly as part of regular clinical care, to monitor for
late effects following transplant. Patients will be given a schedule of
tests at that time. The
maximum duration of the study for each patient will be approximately 2 year
from day of transplant. Patients' medical
records will be reviewed throughout the study to collect information about their
health.
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