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

Abstract

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.

Keywords:

• Nephropathic cystinosis
• immediate-release cysteamine bitartrate
• delayed-release cysteamine bitartrate

Notes

Transparency

Declaration of funding

This study was conducted by Mapi on behalf of Horizon Pharma Europe BV, that funded the study and the writing of this manuscript.

Author contributions: All authors participated in the design and conduct of the study, as well as drafting and revising the manuscript.

Declaration of financial/other relationships

G.M. and M.H. have disclosed that they are employees of Horizon Pharma. M.v.d.W. and A.K. have disclosed that they are employees of Mapi and served as paid consultants to Horizon Pharma during the conduct of this study and the preparation of this manuscript.

CMRO peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Acknowledgements

The authors thank Michel Peters for assisting with study selection, and Jason Foo for contributing to the preparation of the draft manuscript.

Notes

1 Procysbi is a registered trade name of Horizon Pharma

2 Cystagon is a registered trade name of Mylan Pharma, USA

1 Procysbi is a registered trade name of Horizon Pharma

2 Cystagon is a registered trade name of Mylan Pharma, USA