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Systematic Review of Randomized Clinical Trials on Safety and Efficacy of Pharmacological and Nonpharmacological Treatments for Retinitis Pigmentosa.

Sacchetti M, Mantelli F, Merlo D, Lambiase A - J Ophthalmol (2015)

Bottom Line: Aims.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.

View Article: PubMed Central - PubMed

Affiliation: Cornea and Ocular Surface Unit, San Raffaele Hospital IRCCS, Via Olgettina, No. 60, 20132 Milan, Italy.

ABSTRACT

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.

No MeSH data available.


Related in: MedlinePlus

Risk of bias graph (a) showing authors' judgements about each risk of bias item presented as percentages across all included studies. Risk of bias summary (b) reviewing authors' judgements about each risk of bias item for each included study.
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fig2: Risk of bias graph (a) showing authors' judgements about each risk of bias item presented as percentages across all included studies. Risk of bias summary (b) reviewing authors' judgements about each risk of bias item for each included study.

Mentions: All studies included in the review were randomised controlled trials. The main quality attributes were scored as “low risk,” “high risk,” and “unclear risk” for the following areas: (i) whether or not the randomisation is properly concealed (random sequence generation and allocation concealment), (ii) whether or not the participants are masked (blinding), (iii) whether or not the outcome assessment is masked (incomplete outcome data), and (iv) for selective reporting bias. Other biases include the presence of commercial support, potential source of bias related to the specific study design used, or we are not sure whether an important risk of bias existed (Figure 2).


Systematic Review of Randomized Clinical Trials on Safety and Efficacy of Pharmacological and Nonpharmacological Treatments for Retinitis Pigmentosa.

Sacchetti M, Mantelli F, Merlo D, Lambiase A - J Ophthalmol (2015)

Risk of bias graph (a) showing authors' judgements about each risk of bias item presented as percentages across all included studies. Risk of bias summary (b) reviewing authors' judgements about each risk of bias item for each included study.
© Copyright Policy - open-access
Related In: Results  -  Collection

Show All Figures
getmorefigures.php?uid=PMC4539114&req=5

fig2: Risk of bias graph (a) showing authors' judgements about each risk of bias item presented as percentages across all included studies. Risk of bias summary (b) reviewing authors' judgements about each risk of bias item for each included study.
Mentions: All studies included in the review were randomised controlled trials. The main quality attributes were scored as “low risk,” “high risk,” and “unclear risk” for the following areas: (i) whether or not the randomisation is properly concealed (random sequence generation and allocation concealment), (ii) whether or not the participants are masked (blinding), (iii) whether or not the outcome assessment is masked (incomplete outcome data), and (iv) for selective reporting bias. Other biases include the presence of commercial support, potential source of bias related to the specific study design used, or we are not sure whether an important risk of bias existed (Figure 2).

Bottom Line: Aims.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.

View Article: PubMed Central - PubMed

Affiliation: Cornea and Ocular Surface Unit, San Raffaele Hospital IRCCS, Via Olgettina, No. 60, 20132 Milan, Italy.

ABSTRACT

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.

No MeSH data available.


Related in: MedlinePlus