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. doi: 10.1016/s0090-1229(95)90306-2.


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.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adenosine Deaminase / administration & dosage
  • Adenosine Deaminase / deficiency*
  • Adenosine Deaminase / therapeutic use*
  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Dose-Response Relationship, Drug
  • Humans
  • Immunologic Deficiency Syndromes / drug therapy*
  • Immunotherapy / trends


  • Adenosine Deaminase
  • pegademase bovine