PEG-ADA: An Alternative to Haploidentical Bone Marrow Transplantation and an Adjunct to Gene Therapy for Adenosine Deaminase Deficiency

Hum Mutat. 1995;5(2):107-12. doi: 10.1002/humu.1380050202.

Abstract

PEG-ADA is a long-circulating form of adenosine deaminase (ADA) that has been in use for > 8 years as replacement therapy for severe combined immunodeficiency disease due to ADA deficiency. Treatment with PEG-ADA almost completely corrects metabolic abnormalities, allowing the recovery of a variable degree of immune function. Although not normal, the level of function achieved has in most cases been sufficient to protect against opportunistic and life-threatening infections. PEG-ADA has been used as an alternative for patients who lack an HLA-identical bone marrow donor, but are judged to be at too high a risk for undergoing HLA-haploidentical marrow transplantation. To date, mortality and morbidity with PEG-ADA have been less than for the latter procedure. PEG-ADA has also been an important adjunct to attempts to develop somatic cell gene therapy for ADA deficiency, although its continued use poses a problem for evaluation of the benefit of gene therapy. As a true "orphan drug" developed to treat a very small patient population, the cost per patient of PEG-ADA is very high.

Publication types

  • Comparative Study
  • Review

MeSH terms

  • Adenosine Deaminase / chemistry
  • Adenosine Deaminase / deficiency*
  • Adenosine Deaminase / pharmacology*
  • Adolescent
  • Adult
  • Bone Marrow Transplantation*
  • Child
  • Genetic Therapy*
  • Haplotypes
  • Humans
  • Infant
  • Severe Combined Immunodeficiency / drug therapy
  • Severe Combined Immunodeficiency / genetics
  • Severe Combined Immunodeficiency / therapy*

Substances

  • Adenosine Deaminase
  • pegademase bovine