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

Journal Article (Clinical Trial;Journal Article)

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.

Full Text

Duke Authors

Cited Authors

  • Hershfield, MS

Published Date

  • September 1995

Published In

Volume / Issue

  • 76 / 3 Pt 2

Start / End Page

  • S228 - S232

PubMed ID

  • 7554473

International Standard Serial Number (ISSN)

  • 0090-1229

Digital Object Identifier (DOI)

  • 10.1016/s0090-1229(95)90306-2


  • eng

Conference Location

  • United States