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Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency

  • Immunology (HB Gaspar, Section Editor)
  • Published:
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Abstract

Hematopoietic stem cell transplantation 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.

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Disclosure

Mortan J. Cowan reports personal fees from Bluebird Bio, Inc. and served on the Scientific Advisory board for Exogen Bio, Inc. Christopher C. Dvorak reports personal fees from Bluebird Bio, Inc.  Justin T. Wahlstrom declares no conflict of interest.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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  1. 505 Parnassus Ave, Room M-659, San Francisco, CA, 94143, USA

    Justin T. Wahlstrom, Christopher C. Dvorak & Morton J. Cowan

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  1. Justin T. Wahlstrom
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  2. Christopher C. Dvorak
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  3. Morton J. Cowan
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Correspondence to Morton J. Cowan.

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This article is part of the Topical Collection on Immunology.

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Wahlstrom, J.T., Dvorak, C.C. & Cowan, M.J. Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency. Curr Pediatr Rep 3, 1–10 (2015). https://doi.org/10.1007/s40124-014-0071-7

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  • DOI: https://doi.org/10.1007/s40124-014-0071-7

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