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)

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, and late onset of immunodeficiency disease. Two of these patients have entered a trial of gene therapy, but continue to receive enzyme replacement. © 1992 International Pediatric Research Foundation, Inc.

Full Text

Duke Authors

Cited Authors

  • Hershfield, MS; Chaffee, S; Sorensen, RU

Published Date

  • January 1, 1993

Published In

Volume / Issue

  • 33 / 1

Start / End Page

  • S42 - S48

Electronic International Standard Serial Number (EISSN)

  • 1530-0447

International Standard Serial Number (ISSN)

  • 0031-3998

Digital Object Identifier (DOI)

  • 10.1203/00006450-199333011-00009

Citation Source

  • Scopus