In this installment of the "How I Treat" series in the journal Blood, Dr. H. Bobby Gaspar and colleagues describe their treatment algorithms for pediatric patients with severe combined immunodeficiency (SCID). They note that previous research has identified the factors determining successful outcome and survival for SCID, which has improved survival in these patients. They discuss advances in understanding the genetic basis of SCID, and how that knowledge permits tailoring transplant protocols to specific SCID forms and the integration of gene therapies when appropriate, especially in the X-linked and adenosine deaminase (ADA) deficient forms of SCID.
Review: How I Treat SCID
Oct 2013