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Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency #MMPMID25821657
Wahlstrom JT; Dvorak CC; Cowan MJ
Curr Pediatr Rep 2015[Mar]; 3 (1): 1-10 PMID25821657show ga
Hematopoietic stem cell transplantation (HSCT) is an effective approach for the treatment of severe combined immunodeficiency (SCID). However, SCID is not a homogeneous disease, and the treatment required for successful transplantation varies significantly between SCID subtypes and the degree of HLA mismatch between the best available donor and the patient. Recent studies are beginning to more clearly define this heterogeneity and how outcomes may vary. With a more detailed understanding of SCID, new approaches can be developed to maximize immune reconstitution, while minimizing acute and long-term toxicities associated with chemotherapy conditioning.