A systematic literature review of cysteamine bitartrate in the treatment of nephropathic cystinosis

• Published in: Current medical research and opinion 2017-11, Vol.33 (11), p.2065-2076
• Main Authors: Medic, Goran, van der Weijden, Miriam, Karabis, Andreas, Hemels, Michiel
• Format: Article
• Language: English
• Subjects: delayed-release cysteamine bitartrate; immediate-release cysteamine bitartrate; Nephropathic cystinosis
• ISSN: 0300-7995; 1473-4877
• DOI: 10.1080/03007995.2017.1354288

Objectives: To summarize available clinical evidence for cysteamine bitartrate preparations in the treatment of nephropathic cystinosis as identified through a systematic literature review (SLR). Methods: We searched MEDLINE, MEDLINE In-Process and Embase using Ovid with a predefined search strategy through 19 January 2016. All publicly available clinical reports on the use of delayed-release (DR) cysteamine bitartrate (Procysbi 1 ) or immediate-release (IR) cysteamine bitartrate (Cystagon 2 ) in patients with cystinosis were included. Results: We identified a total of 103 publications and 10 trial records. Of these, 9 studies describe DR cysteamine bitartrate (n = 267 patients), 42 describe IR cysteamine bitartrate (n = 1,427 patients) and in 53 studies the exact preparation was not specified (n = 906 patients). The vast majority of the studies used a non-randomized study design, with randomized clinical trials (RCTs) being scarce (1 study comparing DR and IR formulation) and case reports (n = 49) being the most common study design representing 47% of the total. Conclusion: A substantial evidence base for cysteamine bitartrate in the treatment of nephropathic cystinosis was identified. However, the majority of the evidence was of relatively low quality, with evidence levels of 3 or 4.