How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID) View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2017-05

AUTHORS

Donald B. Kohn, H. Bobby Gaspar

ABSTRACT

Adenosine deaminase-deficient severe combined immune deficiency (ADA SCID) accounts for 10-15% of cases of human SCID. From what was once a uniformly fatal disease, the prognosis for infants with ADA SCID has improved greatly based on the development of multiple therapeutic options, coupled with more frequent early diagnosis due to implementation of newborn screening for SCID. We review the various treatment approaches for ADA SCID including allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen-matched sibling or family member or from a matched unrelated donor or a haplo-identical donor, autologous HSCT with gene correction of the hematopoietic stem cells (gene therapy-GT), and enzyme replacement therapy (ERT) with polyethylene glycol-conjugated adenosine deaminase. Based on growing evidence of safety and efficacy from GT, we propose a treatment algorithm for patients with ADA SCID that recommends HSCT from a matched family donor, when available, as a first choice, followed by GT as the next option, with allogeneic HSCT from an unrelated or haplo-identical donor or long-term ERT as other options. More... »

PAGES

351-356

Identifiers

URI

http://scigraph.springernature.com/pub.10.1007/s10875-017-0373-y

DOI

http://dx.doi.org/10.1007/s10875-017-0373-y

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1083805833

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/28194615


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