Severe Combined Immunodeficiency Clinical Trial
Official title:
Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil
This pilot clinical trial studies total-body irradiation followed by cyclosporine and mycophenolate mofetil in treating patients with severe combined immunodeficiency (SCID) undergoing donor bone marrow transplant. Giving total-body irradiation (TBI) before a donor bone marrow transplant using stem cells that closely match the patient's stem cells, helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may mix with the patient's immune cells and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening.
PRIMARY OBJECTIVES:
I. To safely establish partial lymphoid chimerism (1-95% donor cluster of differentiation
[CD]3+ cells) using a non-lethal conditioning regimen in patients with severe combined
immunodeficiency syndrome.
II. To define the kinetics of immune reconstitution following a non-lethal conditioning
regimen in patients with immunodeficiency diseases.
OUTLINE:
Patients receive cyclosporine orally (PO) or intravenously (IV) on days -3 to 100 followed by
a taper until day 180 and mycophenolate mofetil PO or IV on days 0-40 with a taper until day
96 in the absence of unacceptable toxicity. Unrelated donor recipients also undergo TBI on
day 0. Patients undergo bone marrow transplant on day 0.
After completion of study treatment, patients are followed up at 6 months and then yearly for
5 years.
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