[Cysteamine in the treatment of cystinosis in children. In vitro and in vivo studies].
The effect of cysteamine was studied in 6 children with nephropathic cystinosis. In 3 of them an in vitro study on fibroblasts was performed. The cystine content of fibroblasts was immediately diminished (about 90% of total cystine content) as soon as the concentration of cysteamine in the medium was greater than or equal to 0,1 mmole/l. In vivo, 50 to 89 mg/kg/day of cysteamine was administered for 9 to 37 months (mean 21,3). There was no adverse reaction. In all cases a dramatic decline in leukocyte cystine level was observed (in 5 cases the level was within the range seen in clinically unaffected heterozygotes). Growth was not improved. The renal function was stabilised in 3 cases. Photophobia which was present in 4 children decreased in 2 cases or disappeared in 2 cases.
D002478:Cells, Cultured; D002648:Child; D002675:Child, Preschool; D003543:Cysteamine / Q000008:administration & dosage / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D003553:Cystine / Q000032:analysis / Q000097:blood; D003554:Cystinosis / Q000188:drug therapy* / Q000503:physiopathology; D005128:Eye Diseases / Q000628:therapy; D005260:Female; D005347:Fibroblasts / Q000032:analysis / Q000187:drug effects; D006128:Growth / Q000187:drug effects; D006801:Humans; D007223:Infant; D007668:Kidney / Q000503:physiopathology; D007962:Leukocytes / Q000032:analysis; D008297:Male
D003553:Cystine; D003543:Cysteamine
La cystéamine dans le traitement de la cystinose infantile. Etudes in vitro et in vivo.