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Enteric-Coated Cysteamine Bitartrate in Cystinosis Patients

Authors: Klank, S. (Sabrina); Stein, C. (Christina) van; Grüneberg, M. (Marianne); Ottolenghi, C. (Chris); Rauwolf, K.K. (Kerstin); Grebe, J. (Jürgen); Reunert, J. (Janine); +2 Authors

Enteric-Coated Cysteamine Bitartrate in Cystinosis Patients

Abstract

Cystinosis is a severe inherited metabolic storage disease caused by the lysosomal accumulation of cystine. Lifelong therapy with the drug cysteamine bitartrate is necessary. Cysteamine cleaves intralysosomal cystine, and thereafter, it can exit from the organelle. The need for frequent dosing every 6 h and the high prevalence of gastrointestinal side effects lead to poor therapy adherence. The purpose of our study was to improve cysteamine treatment by comparing the efficacy of two cysteamine formulas. This is highly relevant for the long-term outcome of cystinosis patients. The cystine and cysteamine levels of 17 patients taking immediate-release cysteamine (IR-cysteamine/Cystagon®) and 6 patients taking encapsulated delayed-release cysteamine (EC-cysteamine) were analyzed. The EC-cysteamine levels showed a near-ideal pharmacokinetic profile indicative of delayed release (longer Tmax and Tmin), and the corresponding cystine levels showed few fluctuations. In addition, the Cmax of IR-cysteamine was greater, which was responsible for unbearable side effects (e.g., nausea, vomiting, halitosis, lethargy). Treatment with EC-cysteamine improves the quality of life of cystinosis patients because the frequency of intake can be reduced to 2–3 times daily and it has a more favorable pharmacokinetic profile than IR-cysteamine. In particular, cystinosis patients with no access to the only approved delayed-release cysteamine Procysbi® could benefit from a cost-effective alternative.

Keywords

ddc:610, 570, immediate-release cysteamine (Cystagon®), 150, cystine-levels, 610, cystinosis; enteric-coated cysteamine; immediate-release cysteamine (Cystagon<sup>®</sup>); cystine-levels; pharmacokinetics, Article, cystinosis, RS1-441, Pharmacy and materia medica, Medicine and health, immediate-release cysteamine (Cystagon<sup>®</sup>), cystinosis; enteric-coated cysteamine; immediate-release cysteamine (Cystagon®); cystine-levels; pharmacokinetics, pharmacokinetics, enteric-coated cysteamine, info:eu-repo/classification/ddc/610, ddc: ddc:610

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    This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
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    popularity
    This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
    Top 10%
    influence
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citations
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
4
Top 10%
Average
Average
Green
gold