[Polyethylene glycol-adenosine deaminase: a new adenosine deaminase deficiency therapy. Value of deoxyadenosine triphosphate determination for therapeutic monitoring].
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.
Bory
C
C
Laboratoire de Pharmacie Clinique, Institut des Sciences Pharmaceutiques et Biologiques, Lyon.
Boulieu
R
R
Souillet
G
G
Hershfield
M S
MS
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
D003838:Deoxyadenine Nucleotides; D000243:Adenosine Deaminase; C051409:pegademase bovine
Le polyéthylène glycol-adénosine désaminase: nouveau traitement du déficit en adénosine désaminase. Intérêt de la détermination du désoxyadénosine triphosphate dans la surveillance thérapeutique.