Twice-Daily Cysteamine Bitartrate Therapy for Children with Cystinosis

Ranjan Dohil, Meredith Fidler, Jon A. Gangoiti, Frederick Kaskel, Jerry A. Schneider, Bruce A. Barshop

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Abstract

Objective: Cystinosis causes renal and other organ failure. Regular 6-hourly cysteamine bitartrate (Cystagon; Mylan, Morgantown, West Virginia) reduces intracellular cystine and the rate of organ deterioration. A formulation of cysteamine requiring less frequent dosing may improve compliance and possibly patient outcome. Methods: Enteric-release cysteamine was prepared. For a period of 1 month, patients received their regular cysteamine dose every 6 hours (stage I). The patients then underwent pharmacokinetic and pharmacodynamic studies following washout periods using single-doses of cysteamine and enteric-release cysteamine (stage II). Finally, the patients commenced regular enteric-release cysteamine therapy (stage III). Weekly trough white blood cell (WBC) cystine levels were recorded. Results: Seven children with cystinosis (mean age, 11.8 years; range, 8-17 years) who received cysteamine and enteric-release cysteamine (mean dose, 45 and 28.8 mg/kg body weight/day, respectively) had mean WBC cystine levels of 0.7 ± 0.3 and 0.41 ± 0.22 nmol half-cystine/mg protein in study stages I and III, respectively. Study stage II showed that the mean time (Tmax) to reach the maximum plasma cysteamine level (Cmax) was longer for enteric-release cysteamine than for cysteamine (176 minutes vs 60 minutes; P = .001), but the mean Cmax at the same dose was similar. Mean serum gastrin levels were similar after ingestion of cysteamine and enteric-release cysteamine. Conclusions: Twelve-hour enteric-release cysteamine, given at approximately 60% of the previous daily dose of cysteamine, was effective in maintaining trough WBC cystine levels within a satisfactory range.

Original languageEnglish (US)
Pages (from-to)71-75.e3
JournalJournal of Pediatrics
Volume156
Issue number1
DOIs
StatePublished - Jan 2010

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ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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Dohil, R., Fidler, M., Gangoiti, J. A., Kaskel, F., Schneider, J. A., & Barshop, B. A. (2010). Twice-Daily Cysteamine Bitartrate Therapy for Children with Cystinosis. Journal of Pediatrics, 156(1), 71-75.e3. https://doi.org/10.1016/j.jpeds.2009.07.016