title: PEG-ADA replacement therapy for adenosine deaminase deficiency: an update after 8.5 years.
abstract: 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.
authors.affiliation: Department of Medicine, Duke University Medical Center, Durham, North Carolina 27707, USA.
authors.forename: M S
authors.initials: MS
authors.lastname: Hershfield
mesh_terms: D000243:Adenosine Deaminase / Q000008:administration & dosage / Q000172:deficiency* / Q000627:therapeutic use*; D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000188:drug therapy*; D007167:Immunotherapy / Q000639:trends
chemical_list: D000243:Adenosine Deaminase; C051409:pegademase bovine