Optimizing outcomes of hematopoietic stem cell transplantation for severe combined immunodeficiency

Clin Immunol. 2009 May;131(2):179-88. doi: 10.1016/j.clim.2009.01.003. Epub 2009 Feb 13.


This FOCIS Centers of Excellence Short Analytical Review is based on the clinical vignette of two boys from the same family with very different outcomes following hematopoietic stem cell transplantation (HSCT) for X-linked severe combined immunodeficiency (SCID). We review the kinetics of immune reconstitution following HSCT in SCID and emphasize the latest information regarding optimizing transplant outcomes for this disorder. The cases illustrate the difficulties and controversies surrounding the optimal strategies for planning SCID transplants. Specifically, we will focus on 3 areas of current debate and investigation: (i) factors involved in donor selection; (ii) the role of pretransplant conditioning; and (iii) benefits of early HSCT for SCID.

Publication types

  • Case Reports
  • Review

MeSH terms

  • Fatal Outcome
  • Female
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Infant, Newborn
  • Interleukin Receptor Common gamma Subunit / genetics
  • Male
  • Mutation
  • Pregnancy
  • Treatment Outcome
  • X-Linked Combined Immunodeficiency Diseases / therapy*


  • IL2RG protein, human
  • Interleukin Receptor Common gamma Subunit