A SYSTEMATIC LITERATURE REVIEW OF CYSTEAMINE BITARTRATE PREPARATIONS IN PATIENTS WITH CYSTINOSIS
Author(s)
Van Der Weijden M, Peters M, Karabis A
Mapi Group, Houten, The Netherlands
OBJECTIVES: To summarize all available clinical evidence for the different cysteamine bitartrate preparations in patients with cystinosis as identified through a systematic literature review (SLR). METHODS: We included studies reporting on the use of PROCYSBI® (cysteamine bitartrate) or Cystagon® (cysteamine bitartrate) in patients with cystinosis. All study designs and outcomes were included with no language restrictions. We searched MEDLINE, MEDLINE In-Process and EMBASE using Ovid® with a predefined search strategy through 19th January 2016. RESULTS: We included a total of 107 publications and 10 trial records, describing 107 studies, for data extraction. Of these, 13 studies describe PROCYSBI (n=286 patients), 49 describe Cystagon (n=1,587 patients) and in 53 studies the exact preparation was unclear (n=1,020 patients). The vast majority of the studies used a non-randomized study design, with RCTs being scarce (1 study comparing PROCYSBI with Cystagon) and case reports being the most common study design (50 studies). Whilst there were more studies for Cystagon than PROCYSBI, a large proportion of the Cystagon studies consisted of case reports (41%). CONCLUSIONS: A substantial evidence base for cysteamine bitartrate in nephropathic cystinosis was identified. However, the majority of the evidence was of relatively low quality.
Conference/Value in Health Info
2016-10, ISPOR Europe 2016, Vienna, Austria
Value in Health, Vol. 19, No. 7 (November 2016)
Code
PSY134
Topic
Health Service Delivery & Process of Care
Topic Subcategory
Health Care Research, Prescribing Behavior
Disease
Rare and Orphan Diseases