title: [Polyethylene glycol-adenosine deaminase: a new adenosine deaminase deficiency therapy. Value of deoxyadenosine triphosphate determination for therapeutic monitoring].
abstract: The effect of polyethylene glycol-adenosine deaminase (PEG-ADA) therapy on biochemical, immunological and clinical abnormalities in an ADA-deficit child with severe combined immunodeficiency has been studied. Following PEG-ADA therapy, total lymphocytes, lymphocyte subsets (CD3, CD4 and CD8) and the response of lymphocytes to non specific mitogens increase significantly. The improvement of immunological functions is closely related to a decrease of erythrocyte deoxyadenosine triphosphate (dATP) concentrations. This study shows that PEG-ADA therapy is sufficiently effective to reduce and to maintain erythrocyte dATP levels at values compatible with normal immune functions. PEG-ADA represents an important progress for the treatment of ADA deficiency associated with severe combined immunodeficiency disease.
authors.affiliation: Laboratoire de Pharmacie Clinique, Institut des Sciences Pharmaceutiques et Biologiques, Lyon.
authors.forename: C
authors.initials: C
authors.lastname: Bory
authors.forename: R
authors.initials: R
authors.lastname: Boulieu
authors.forename: G
authors.initials: G
authors.lastname: Souillet
authors.forename: M S
authors.initials: MS
authors.lastname: Hershfield
mesh_terms: D000243:Adenosine Deaminase / Q000008:administration & dosage / Q000172:deficiency* / Q000494:pharmacology / Q000627:therapeutic use*; D002675:Child, Preschool; D003838:Deoxyadenine Nucleotides / Q000032:analysis* / Q000378:metabolism; D004912:Erythrocytes / Q000187:drug effects / Q000378:metabolism; D005260:Female; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000188:drug therapy* / Q000276:immunology; D007273:Injections, Intramuscular; D008991:Monitoring, Physiologic
chemical_list: D003838:Deoxyadenine Nucleotides; D000243:Adenosine Deaminase; C051409:pegademase bovine
vernacular_title: Le polyéthylène glycol-adénosine désaminase: nouveau traitement du déficit en adénosine désaminase. Intérêt de la détermination du désoxyadénosine triphosphate dans la surveillance thérapeutique.