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...

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Bibliographic Details
Published in:Pharmaceutics 2023-06, Vol.15 (7), p.1851
Main Authors: Klank, Sabrina, van Stein, Christina, Grüneberg, Marianne, Ottolenghi, Chris, Rauwolf, Kerstin K, Grebe, Jürgen, Reunert, Janine, Harms, Erik, Marquardt, Thorsten
Format: Article
Language:English
Subjects:
Blood
Care and treatment
Chromatography
Compliance
Cysteamine
Cysteine
Cystine
cystine-levels
Cystinosis
enteric-coated cysteamine
Ethylenediaminetetraacetic acid
immediate-release cysteamine (Cystagon®)
Leukocytes
Patient compliance
Patients
Pharmacists
Pharmacokinetics
Plasma
Proteins
Small intestine
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Summary: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 T and T ), and the corresponding cystine levels showed few fluctuations. In addition, the C 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.
ISSN:1999-4923
1999-4923
DOI:10.3390/pharmaceutics15071851