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PEG-ADA replacement therapy for adenosine deaminase deficiency: an update after 8.5 years.

Publication ,  Journal Article
Hershfield, MS
Published in: Clin Immunol Immunopathol
September 1995

Polyethylene glycol-modified adenosine deaminase (PEG-ADA) has now been used for 8.5 years as enzyme replacement therapy for immunodeficiency due to ADA deficiency. PEG-ADA restores a metabolic environment necessary for recovery of immune function. In most cases, the level of function achieved has been sufficient to protect against opportunistic and life-threatening infections. To date, mortality and morbidity with PEG-ADA have been less than for haploidentical bone marrow transplantation. As a true "orphan drug" used to treat a very small patient population, the cost per patient of PEG-ADA is very high, but it has been well tolerated, free of adverse reactions, and effective as an alternative for patients who lack an HLA-identical marrow donor, but are considered too ill to undergo haploidentical marrow transplantation. Concomitant treatment with PEG-ADA has also permitted investigation of gene therapy to be carried out safely.

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Published In

Clin Immunol Immunopathol

DOI

ISSN

0090-1229

Publication Date

September 1995

Volume

76

Issue

3 Pt 2

Start / End Page

S228 / S232

Location

United States

Related Subject Headings

  • Immunotherapy
  • Immunology
  • Immunologic Deficiency Syndromes
  • Humans
  • Dose-Response Relationship, Drug
  • Child, Preschool
  • Child
  • Adult
  • Adolescent
  • Adenosine Deaminase
 

Citation

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ICMJE
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Hershfield, M. S. (1995). PEG-ADA replacement therapy for adenosine deaminase deficiency: an update after 8.5 years. Clin Immunol Immunopathol, 76(3 Pt 2), S228–S232. https://doi.org/10.1016/s0090-1229(95)90306-2
Hershfield, M. S. “PEG-ADA replacement therapy for adenosine deaminase deficiency: an update after 8.5 years.Clin Immunol Immunopathol 76, no. 3 Pt 2 (September 1995): S228–32. https://doi.org/10.1016/s0090-1229(95)90306-2.
Hershfield MS. PEG-ADA replacement therapy for adenosine deaminase deficiency: an update after 8.5 years. Clin Immunol Immunopathol. 1995 Sep;76(3 Pt 2):S228–32.
Hershfield, M. S. “PEG-ADA replacement therapy for adenosine deaminase deficiency: an update after 8.5 years.Clin Immunol Immunopathol, vol. 76, no. 3 Pt 2, Sept. 1995, pp. S228–32. Pubmed, doi:10.1016/s0090-1229(95)90306-2.
Hershfield MS. PEG-ADA replacement therapy for adenosine deaminase deficiency: an update after 8.5 years. Clin Immunol Immunopathol. 1995 Sep;76(3 Pt 2):S228–S232.

Published In

Clin Immunol Immunopathol

DOI

ISSN

0090-1229

Publication Date

September 1995

Volume

76

Issue

3 Pt 2

Start / End Page

S228 / S232

Location

United States

Related Subject Headings

  • Immunotherapy
  • Immunology
  • Immunologic Deficiency Syndromes
  • Humans
  • Dose-Response Relationship, Drug
  • Child, Preschool
  • Child
  • Adult
  • Adolescent
  • Adenosine Deaminase