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Curr Med Res Opin. 2017 Nov;33(11):2065-2076. doi: 10.1080/03007995.2017.1354288. Epub 2017 Aug 3.

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

Author information

1
a Horizon Pharma Europe BV , Utrecht , The Netherlands.
2
b Unit of Pharmacoepidemiology and Pharmacoeconomics, Department of Pharmacy , University of Groningen , Groningen , The Netherlands.
3
c Real World Strategy & Analytics , Mapi Group , Houten , The Netherlands.

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 (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.

KEYWORDS:

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

PMID:
28692321
DOI:
10.1080/03007995.2017.1354288
[Indexed for MEDLINE]

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