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JACI Highlights - November 2009
Helping SCID infants survive and thrive after bone marrow transplantation
A study performed at Baylor College of Medicine and Texas Children’s Hospital in Houston, Tex, is the first to directly address whether preconditioning of infants with severe combined immunodeficiency (SCID) with immunosuppressant drugs prior to bone marrow transplantation improves their long-term outcomes. Patel et al recently assessed preconditioned SCID patients and compared their outcomes to nonconditioned SCID transplanted patients. Eighteen patients, 17 of whom received pretransplant conditioning followed by haploidentical mismatched related donor, matched unrelated donor, or mismatched unrelated donor transplants; 13 (72%) engrafted and have survived a median of 3.8 years, 5 of 13 (38%) require IVIG, and 6 of 6 age-eligible children attend school. Five of 5 transplants with matched sibling bone marrow survive. Early outcomes and quality of life of the previous nonconditioned versus the present preconditioned cohorts appear to be equal (except for less graft-versus-host disease with preconditioning), but longer-term follow-up will be necessary. The study group (William T. Shearer, MD, PhD, Director) concluded that bone marrow transplantation with or without preconditioning in SCID patients results in engraftment, long-term survival, and a good quality of life for the majority of patients.
“Outcomes of Severe Combined Immunodeficiency Patients Treated with Hematopoietic Stem Cell Transplantation with and without Pre-conditioning” by Patel et al. (JACI November 2009 Volume 124 No. 5)
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