title: [Treatment of cystinosis using cysteamine].
abstract: Eighteen pediatric patients with cystinosis were treated with cysteamine. Treatment was started at a variable age, between 10 months and 7 years, and was continued for 6 months to eight years. Results were evaluated on renal function changes and growth. Whereas 65% of patients with cystinosis develop terminal renal failure before the age of ten years, three (16%) of our 18 patients given cysteamine started dialysis before the age of ten and all three had first received cysteamine only after 4 1/2 years of age. The plasma creatinine level was also lower in treated patients as compared with a control multicenter group. Growth was also significantly improved by treatment, especially in those children treated before the age of 26 months who were taller by 2 SD at age five and 2.5 SD at age eight than untreated controls. Some children who complied strictly with the treatment regimen had an normal stature (- 1 SD) at ten years of age. In conclusion, the effectiveness of cysteamine seems obvious in this group of patients if rigorous compliance with the drug dosing schedule is achieved.
authors.affiliation: Service de Néphrologie Pédiatrique, Hôpital des Enfants-Malades, Paris.
authors.forename: M
authors.initials: M
authors.lastname: Broyer
authors.forename: M J
authors.initials: MJ
authors.lastname: Tete
mesh_terms: D002648:Child; D002675:Child, Preschool; D003543:Cysteamine / Q000008:administration & dosage / Q000627:therapeutic use*; D003553:Cystine / Q000032:analysis; D003554:Cystinosis / Q000188:drug therapy* / Q000503:physiopathology; D005260:Female; D006128:Growth; D006801:Humans; D007223:Infant; D007668:Kidney / Q000503:physiopathology; D007962:Leukocytes / Q000032:analysis; D008297:Male; D013997:Time Factors
chemical_list: D003553:Cystine; D003543:Cysteamine
vernacular_title: Traitement de la cystinose par la cystéamine.