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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.

Publication ,  Journal Article
Hershfield, MS; Chaffee, S; Sorensen, RU
Published in: Pediatr Res
January 1993

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.

Duke Scholars

Published In

Pediatr Res

DOI

ISSN

0031-3998

Publication Date

January 1993

Volume

33

Issue

1 Suppl

Start / End Page

S42 / S47

Location

United States

Related Subject Headings

  • Severe Combined Immunodeficiency
  • Pediatrics
  • Lymphocyte Activation
  • Leukocyte Count
  • Immune System
  • Humans
  • Genetic Therapy
  • Female
  • Child, Preschool
  • Child
 

Citation

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ICMJE
MLA
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Hershfield, M. S., Chaffee, S., & Sorensen, R. U. (1993). 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. Pediatr Res, 33(1 Suppl), S42–S47. https://doi.org/10.1203/00006450-199305001-00236
Hershfield, M. S., S. Chaffee, and R. U. Sorensen. “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.Pediatr Res 33, no. 1 Suppl (January 1993): S42–47. https://doi.org/10.1203/00006450-199305001-00236.
Hershfield, M. S., et al. “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.Pediatr Res, vol. 33, no. 1 Suppl, Jan. 1993, pp. S42–47. Pubmed, doi:10.1203/00006450-199305001-00236.

Published In

Pediatr Res

DOI

ISSN

0031-3998

Publication Date

January 1993

Volume

33

Issue

1 Suppl

Start / End Page

S42 / S47

Location

United States

Related Subject Headings

  • Severe Combined Immunodeficiency
  • Pediatrics
  • Lymphocyte Activation
  • Leukocyte Count
  • Immune System
  • Humans
  • Genetic Therapy
  • Female
  • Child, Preschool
  • Child