{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,10]],"date-time":"2026-01-10T03:02:21Z","timestamp":1768014141536,"version":"3.49.0"},"reference-count":39,"publisher":"MDPI AG","issue":"7","license":[{"start":{"date-parts":[[2023,6,29]],"date-time":"2023-06-29T00:00:00Z","timestamp":1687996800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Orphan Europe","award":["141217"],"award-info":[{"award-number":["141217"]}]},{"name":"Orphan Europe","award":["20150904"],"award-info":[{"award-number":["20150904"]}]},{"DOI":"10.13039\/100014480","name":"Horizon Pharmaceuticals","doi-asserted-by":"publisher","award":["141217"],"award-info":[{"award-number":["141217"]}],"id":[{"id":"10.13039\/100014480","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100014480","name":"Horizon Pharmaceuticals","doi-asserted-by":"publisher","award":["20150904"],"award-info":[{"award-number":["20150904"]}],"id":[{"id":"10.13039\/100014480","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pharmaceutics"],"abstract":"<jats:p>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\u00ae) 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\u20133 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\u00ae could benefit from a cost-effective alternative.<\/jats:p>","DOI":"10.3390\/pharmaceutics15071851","type":"journal-article","created":{"date-parts":[[2023,6,30]],"date-time":"2023-06-30T00:51:06Z","timestamp":1688086266000},"page":"1851","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Enteric-Coated Cysteamine Bitartrate in Cystinosis Patients"],"prefix":"10.3390","volume":"15","author":[{"given":"Sabrina","family":"Klank","sequence":"first","affiliation":[{"name":"Department of Paediatrics, Metabolic Diseases, University of M\u00fcnster, Albert-Schweitzer-Campus 1, 48149 M\u00fcnster, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christina","family":"van Stein","sequence":"additional","affiliation":[{"name":"Department of Paediatrics, Metabolic Diseases, University of M\u00fcnster, Albert-Schweitzer-Campus 1, 48149 M\u00fcnster, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marianne","family":"Gr\u00fcneberg","sequence":"additional","affiliation":[{"name":"Department of Paediatrics, Metabolic Diseases, University of M\u00fcnster, Albert-Schweitzer-Campus 1, 48149 M\u00fcnster, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chris","family":"Ottolenghi","sequence":"additional","affiliation":[{"name":"UMR 1163, Universit\u00e9 Paris Descartes, Sorbonne Paris Cit\u00e9, Institut IMAGINE, 24 Boulevard du Montparnasse, 75015 Paris, France"},{"name":"Biochimie M\u00e9tabolomique et Prot\u00e9omique, H\u00f4pital Necker\u2014Enfants Malades, 149 Rue de S\u00e8vres, 75015 Paris, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kerstin K.","family":"Rauwolf","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology and Oncology, University of M\u00fcnster, Albert-Schweitzer-Campus 1, 48149 M\u00fcnster, Germany"},{"name":"Division of Pediatric Oncology, University Children\u2019s Hospital Z\u00fcrich, Steinwiesstra\u00dfe 75, 8032 Z\u00fcrich, Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J\u00fcrgen","family":"Grebe","sequence":"additional","affiliation":[{"name":"Department of Paediatrics, Metabolic Diseases, University of M\u00fcnster, Albert-Schweitzer-Campus 1, 48149 M\u00fcnster, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Janine","family":"Reunert","sequence":"additional","affiliation":[{"name":"Department of Paediatrics, Metabolic Diseases, University of M\u00fcnster, Albert-Schweitzer-Campus 1, 48149 M\u00fcnster, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Erik","family":"Harms","sequence":"additional","affiliation":[{"name":"Department of Paediatrics, Metabolic Diseases, University of M\u00fcnster, Albert-Schweitzer-Campus 1, 48149 M\u00fcnster, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Thorsten","family":"Marquardt","sequence":"additional","affiliation":[{"name":"Department of Paediatrics, Metabolic Diseases, University of M\u00fcnster, Albert-Schweitzer-Campus 1, 48149 M\u00fcnster, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2023,6,29]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1056\/NEJMra020552","article-title":"Cystinosis","volume":"347","author":"Gahl","year":"2002","journal-title":"N. 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Nephrol."}],"container-title":["Pharmaceutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1999-4923\/15\/7\/1851\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,10]],"date-time":"2025-10-10T20:03:47Z","timestamp":1760126627000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1999-4923\/15\/7\/1851"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,6,29]]},"references-count":39,"journal-issue":{"issue":"7","published-online":{"date-parts":[[2023,7]]}},"alternative-id":["pharmaceutics15071851"],"URL":"https:\/\/doi.org\/10.3390\/pharmaceutics15071851","relation":{},"ISSN":["1999-4923"],"issn-type":[{"value":"1999-4923","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,6,29]]}}}