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Systematic Review of Randomized Clinical Trials on Safety and Efficacy of
Pharmacological and Nonpharmacological Treatments for Retinitis Pigmentosa
#MMPMID26339504
Sacchetti M
; Mantelli F
; Merlo D
; Lambiase A
J Ophthalmol
2015[]; 2015
(?): 737053
PMID26339504
show ga
Aims. Several treatments have been proposed to slow down progression of Retinitis
pigmentosa (RP), a hereditary retinal degenerative condition leading to severe
visual impairment. The aim of this study is to systematically review data from
randomized clinical trials (RCTs) evaluating safety and efficacy of medical
interventions for the treatment of RP. Methods. Randomized clinical trials on
medical treatments for syndromic and nonsyndromic RP published up to December
2014 were included in the review. Visual acuity, visual field, electroretinogram,
and adverse events were used as outcome measures. Results. The 19 RCTs included
in this systematic review included trials on hyperbaric oxygen delivery, topical
brimonidine tartrate, vitamins, docosahexaenoic acid, gangliosides, lutein, oral
nilvadipine, ciliary neurotrophic factor, and valproic acid. All treatments
proved safe but did not show significant benefit on visual function. Long term
supplementation with vitamin A showed a significantly slower decline rate in
electroretinogram amplitude. Conclusions. Although all medical treatments for RP
appear safe, evidence emerging from RCTs is limited since they do not present
comparable results suitable for quantitative statistical analysis. The limited
number of RCTs, the poor clinical results, and the heterogeneity among studies
negatively influence the strength of recommendations for the long term management
of RP patients.