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Twice-daily cysteamine bitartrate therapy for children with cystinosis

Ranjan Dohil1, Meredith Fidler, Jon A Gangoiti

  • 1Department of Pediatrics, University of California San Diego, La Jolla, CA 92103-8450, USA. rdohil@ucsd.edu

The Journal of Pediatrics
|September 25, 2009
PubMed

Insights

New enteric-release cysteamine, taken every 12 hours, effectively lowers cystine levels in children with cystinosis. This improved formulation may enhance treatment compliance and patient outcomes for this rare genetic disorder.

Area of Science:

  • Biochemistry
  • Pharmacology
  • Pediatric Nephrology

Background:

  • Cystinosis is a rare genetic disorder leading to renal and organ failure due to intracellular cystine accumulation.
  • Current treatment involves frequent dosing of cysteamine bitartrate, impacting patient compliance.
  • A less frequent dosing formulation could improve outcomes in cystinosis management.

Purpose of the Study:

  • To evaluate the efficacy of an enteric-release cysteamine formulation for treating cystinosis.
  • To compare pharmacokinetic and pharmacodynamic profiles of standard vs. enteric-release cysteamine.
  • To assess the impact of enteric-release cysteamine on white blood cell cystine levels.

Main Methods:

  • Preparation of an enteric-release cysteamine formulation.
  • A 3-stage study involving regular cysteamine, pharmacokinetic analysis, and regular enteric-release cysteamine therapy.
  • Weekly monitoring of trough white blood cell (WBC) cystine levels.

Main Results:

  • Enteric-release cysteamine significantly reduced mean WBC cystine levels compared to standard cysteamine.
  • Pharmacokinetic studies showed a longer time to maximum plasma concentration (Tmax) for enteric-release cysteamine, with similar Cmax.
  • Serum gastrin levels remained comparable between the two formulations.

Conclusions:

  • Twelve-hour enteric-release cysteamine is effective in maintaining satisfactory trough WBC cystine levels in children with cystinosis.
  • A reduced daily dose of enteric-release cysteamine (approx. 60%) achieved therapeutic efficacy.
  • This formulation offers a promising alternative for improved cystinosis management and compliance.
Abstract

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