Enteric-Coated Cysteamine Bitartrate in Cystinosis Patients

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...

Verfasser: Klank, Sabrina
Stein, Christina van
Grüneberg, Marianne
Ottolenghi, Chris
Rauwolf, Kerstin Katharina
Grebe, Jürgen
Reunert, Janine
Harms, Erik
Marquardt, Thorsten
FB/Einrichtung:FB 05: Medizinische Fakultät
Dokumenttypen:Artikel
Medientypen:Text
Erscheinungsdatum:2023
Publikation in MIAMI:09.01.2024
Datum der letzten Änderung:09.01.2024
Angaben zur Ausgabe:[Electronic ed.]
Quelle:Pharmaceutics 15 (2023) 7, 1851, 1-13
Schlagwörter:cystinosis; enteric-coated cysteamine; immediate-release cysteamine (Cystagon®); cystine-levels; pharmacokinetics
Fachgebiet (DDC):610: Medizin und Gesundheit
Lizenz:CC BY 4.0
Sprache:Englisch
Förderung:Finanziert durch den Open-Access-Publikationsfonds der Universität Münster.
Format:PDF-Dokument
URN:urn:nbn:de:hbz:6-57998675421
Weitere Identifikatoren:DOI: 10.17879/97998581790
Permalink:https://nbn-resolving.de/urn:nbn:de:hbz:6-57998675421
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Onlinezugriff:10.3390_pharmaceutics15071851.pdf

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.