Optimizing outcomes of hematopoietic stem cell transplantation for severe combined immunodeficiency.
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.
Cuvelier
Geoff D E
GD
University of British Columbia Federation of Clinical Immunology Societies Center of Excellence, Department of Pediatrics, University of British Columbia, BC, Canada. geoff.cuvelier@cancercare.mb.ca
Schultz
Kirk R
KR
Davis
Jeff
J
Hirschfeld
Aaron F
AF
Junker
Anne K
AK
Tan
Rusung
R
Turvey
Stuart E
SE
D017809:Fatal Outcome; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007231:Infant, Newborn; D053631:Interleukin Receptor Common gamma Subunit / Q000235:genetics; D008297:Male; D009154:Mutation; D011247:Pregnancy; D016896:Treatment Outcome; D053632:X-Linked Combined Immunodeficiency Diseases / Q000628:therapy*
C507941:IL2RG protein, human; D053631:Interleukin Receptor Common gamma Subunit