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

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Published:1st Nov 2017
Author: Medic G, van der Weijden M, Karabis A, Hemels M.
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Ref.:Curr Med Res Opin. 2017;33(11):2065-2076.
DOI:10.1080/03007995.2017.1354288
A systematic literature review of cysteamine bitartrate in the treatment of nephropathic cystinosis


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 (Procysbi1) or immediate-release (IR) cysteamine bitartrate (Cystagon2) 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.

 

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