Enzyme replacement therapy with polyethylene glycol-adenosine deaminase in adenosine deaminase deficiency: overview and case reports of three patients, including two now receiving gene therapy.
Journal Article (Journal Article;Review)
During the past 6 y, 29 adenosine deaminase (ADA)-deficient patients with combined immunodeficiency have been treated with polyethylene glycol (PEG)-modified bovine ADA (PEG-ADA). We have monitored plasma ADA activity, metabolic effects of treatment, and the evolution of antibody to PEG-ADA in these patients, in collaboration with immunologists and clinicians in North America, Europe, and Australia, who have monitored immune function and clinical response to treatment. This article summarizes the current status of PEG-ADA therapy and provides recommendations for its use. Recovery of specific immune function during treatment with PEG-ADA is illustrated for three patients, who represent early, delayed, these patients have entered a trial of gene therapy, but continue to receive enzyme replacement.
Full Text
Duke Authors
Cited Authors
- Hershfield, MS; Chaffee, S; Sorensen, RU
Published Date
- January 1, 1993
Published In
Volume / Issue
- 33 / 1 Suppl
Start / End Page
- S42 - S47
PubMed ID
- 8433874
International Standard Serial Number (ISSN)
- 0031-3998
Digital Object Identifier (DOI)
- 10.1203/00006450-199305001-00236
Language
- eng
Conference Location
- United States