Adverse Reactions to Oral Cysteamine Use in Nephropathie Cystinosis

Abstract
Cysteamine (2-aminoethanethiol) was given orally to 19 patients with nephropathic cystinosis for 8-24 mo. in doses ranging from 50-70 mg base/kg per day. Adverse reactions were noted in 3 patients early in the study when a rapidly increasing dosage schedule was followed. Reactions included hyperthermia, lethargy and rash. These reactions were not seen when patients were started on a low dosage which was increased gradually at 3-wk intervals to a level which depleted leukocytes of .apprx. 90% of their free Cys. All 3 reactions resolved within 24 h of cessation of therapy and in these cases successful readministration of drug was achieved. Chronic cysteamine administration to pediatric patients with cystinosis is feasible. The efficacy of this therapy is still being evaluated.

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