Titre du document / Document title
Low-dose enzyme replacement therapy for Gaucher's disease : effects of age, sex, genotype, and clinical features on response to treatment
Auteur(s) / Author(s)
ZIMRAN A.
(1) ;
ELSTEIN D.
(1) ;
KANNAI R.
(1) ;
ZEVIN S.
(1) ;
HADAS-HALPERN I. ;
LEVY-LAHAD E.
(1) ;
COHEN Y.
(1) ;
HOROWITZ M.
(1) ;
ABRAHAMOV A. ;
Affiliation(s) du ou des auteurs / Author(s) Affiliation(s)
(1) Shaare-Zedek medical cent., Gaucher clin., Jerusalem 91031, ISRAEL
Résumé / Abstract
Although alglucerase therapy has become the treatment of choice for symptomatic patients with Gaucher's disease, the low-dose/high-frequency regimen introduced as a means to reduce the high cost of treatment has raised major controversy. We evaluated the efficacy and safety of low-dose alglucerase in 29 patients with Gaucher's disease who completed 6 to 28 months of therapy. All received intravenous alglucerase at a monthly dose of 30 units/kg, given usually in equal doses 3 times a week. All patients responded well to treatment. The hematological improvement and the reduction in organomegaly were satisfactory. No correlation was found between age, sex, genotype, previous splenectomy, or severity score index and the response to treatment
Revue / Journal Title
The American journal of medicine
ISSN 0002-9343
CODEN AJMEAZ
Source / Source
1994, vol. 97, n
o1, pp. 3-13 (29 ref.)
Langue / Language
Anglais
Editeur / Publisher
Elsevier, New York, NY, ETATS-UNIS
(1946)
(Revue)
Mots-clés anglais / English Keywords
Gaucher disease ;
Enzyme ;
Age ;
Treatment ;
Human ;
Sex ;
Genotype ;
Metabolic diseases ;
Enzymopathy ;
Genetic disease ;
Chemotherapy ;
Intravenous administration ;
Mots-clés français / French Keywords
Sphingolipidose héréditaire Gaucher ;
Enzyme ;
Age ;
Traitement ;
Homme ;
Sexe ;
Génotype ;
Alglucerase ;
Métabolisme pathologie ;
Enzymopathie ;
Maladie héréditaire ;
Chimiothérapie ;
Voie intraveineuse ;
Mots-clés espagnols / Spanish Keywords
Esfingolipidosis hereditaria Gaucher ;
Enzima ;
Edad ;
Tratamiento ;
Hombre ;
Sexo ;
Genotipo ;
Metabolismo patología ;
Enzimopatía ;
Enfermedad hereditaria ;
Quimioterapia ;
Vía intravenosa ;
Localisation / Location
INIST-CNRS, Cote INIST : 4562, 35400004057577.0020
Nº notice refdoc (ud4) : 4206470
