Efficacy of valproic acid for retinitis pigmentosa patients: a pilot study

Satoshi Iraha,1–5 Yasuhiko Hirami,1,2 Sachiko Ota,1,2 Genshiro A Sunagawa,3 Michiko Mandai,1–3 Hidenobu Tanihara,5 Masayo Takahashi,1–4 Yasuo Kurimoto1,2 1Department of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, 2Departme...

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Autores principales: Iraha S, Hirami Y, Ota S, Sunagawa GA, Mandai M, Tanihara H, Takahashi M, Kurimoto Y
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Lenguaje:EN
Publicado: Dove Medical Press 2016
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spelling oai:doaj.org-article:b6dd3044a84d4574a3445b30818c6d632021-12-02T04:59:09ZEfficacy of valproic acid for retinitis pigmentosa patients: a pilot study1177-5483https://doaj.org/article/b6dd3044a84d4574a3445b30818c6d632016-07-01T00:00:00Zhttps://www.dovepress.com/efficacy-of-valproic-acid--for-retinitis-pigmentosa-patients-a-pilot-s-peer-reviewed-article-OPTHhttps://doaj.org/toc/1177-5483Satoshi Iraha,1–5 Yasuhiko Hirami,1,2 Sachiko Ota,1,2 Genshiro A Sunagawa,3 Michiko Mandai,1–3 Hidenobu Tanihara,5 Masayo Takahashi,1–4 Yasuo Kurimoto1,2 1Department of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, 2Department of Ophthalmology, Kobe City Medical Center General Hospital, 3Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology, Kobe, 4Application Biology and Regenerative Medicine, Graduate School of Medicine, Kyoto University, Kyoto, 5Department of Ophthalmology, Faculty of Life Sciences, Kumamoto University, Kumamoto, Japan Purpose: The purpose of this study was to examine the efficacy and safety of valproic acid (VPA) use in patients with retinitis pigmentosa (RP).Patients and methods: This was a prospective, interventional, noncomparative case study. In total, 29 eyes from 29 patients with RP whose best-corrected visual acuities (BCVAs) in logarithm of the minimum angle of resolution (logMAR) ranged from 1.0 to 0.16 with visual fields (VFs) of ≤10° (measured using Goldmann perimeter with I4) were recruited. The patients received oral supplementation with 400 mg of VPA daily for 6 months and were followed for an additional 6 months. BCVAs, VFs (measured with the Humphrey field analyzer central 10-2 program), and subjective questionnaires were examined before, during, and after the cessation of VPA supplementation.Results: The changes in BCVA and VF showed statistically significant differences during the internal use of VPA, compared with after cessation (P=0.001). With VPA intake, BCVA in logMAR significantly improved from baseline to 6 months (P=0.006). The mean deviation value of the VF significantly improved from baseline to 1 month (P=0.001), 3 months (P=0.004), and 6 months (P=0.004). These efficacies, however, were reversed to the baseline levels after the cessation of VPA intake. There were no significant relations between the mean blood VPA concentrations of each patient and the changes in BCVA and VF. During the internal use of VPA, 15 of 29 patients answered “easier to see”, whereas blurred vision was registered in 21 of 29 patients on cessation. No systemic drug-related adverse events were observed.Conclusion: While in use, oral intake of VPA indicated a short-term benefit to patients with RP. It is necessary to examine the effect of a longer VPA supplementation in a controlled study design. Keywords: retinitis pigmentosa, valproic acid, clinical trial, retinaIraha SHirami YOta SSunagawa GAMandai MTanihara HTakahashi MKurimoto YDove Medical Pressarticleretinitis pigmentosavalproic acidclinical trialretinaOphthalmologyRE1-994ENClinical Ophthalmology, Vol 2016, Iss Issue 1, Pp 1375-1384 (2016)
institution DOAJ
collection DOAJ
language EN
topic retinitis pigmentosa
valproic acid
clinical trial
retina
Ophthalmology
RE1-994
spellingShingle retinitis pigmentosa
valproic acid
clinical trial
retina
Ophthalmology
RE1-994
Iraha S
Hirami Y
Ota S
Sunagawa GA
Mandai M
Tanihara H
Takahashi M
Kurimoto Y
Efficacy of valproic acid for retinitis pigmentosa patients: a pilot study
description Satoshi Iraha,1–5 Yasuhiko Hirami,1,2 Sachiko Ota,1,2 Genshiro A Sunagawa,3 Michiko Mandai,1–3 Hidenobu Tanihara,5 Masayo Takahashi,1–4 Yasuo Kurimoto1,2 1Department of Ophthalmology, Institute of Biomedical Research and Innovation Hospital, 2Department of Ophthalmology, Kobe City Medical Center General Hospital, 3Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology, Kobe, 4Application Biology and Regenerative Medicine, Graduate School of Medicine, Kyoto University, Kyoto, 5Department of Ophthalmology, Faculty of Life Sciences, Kumamoto University, Kumamoto, Japan Purpose: The purpose of this study was to examine the efficacy and safety of valproic acid (VPA) use in patients with retinitis pigmentosa (RP).Patients and methods: This was a prospective, interventional, noncomparative case study. In total, 29 eyes from 29 patients with RP whose best-corrected visual acuities (BCVAs) in logarithm of the minimum angle of resolution (logMAR) ranged from 1.0 to 0.16 with visual fields (VFs) of ≤10° (measured using Goldmann perimeter with I4) were recruited. The patients received oral supplementation with 400 mg of VPA daily for 6 months and were followed for an additional 6 months. BCVAs, VFs (measured with the Humphrey field analyzer central 10-2 program), and subjective questionnaires were examined before, during, and after the cessation of VPA supplementation.Results: The changes in BCVA and VF showed statistically significant differences during the internal use of VPA, compared with after cessation (P=0.001). With VPA intake, BCVA in logMAR significantly improved from baseline to 6 months (P=0.006). The mean deviation value of the VF significantly improved from baseline to 1 month (P=0.001), 3 months (P=0.004), and 6 months (P=0.004). These efficacies, however, were reversed to the baseline levels after the cessation of VPA intake. There were no significant relations between the mean blood VPA concentrations of each patient and the changes in BCVA and VF. During the internal use of VPA, 15 of 29 patients answered “easier to see”, whereas blurred vision was registered in 21 of 29 patients on cessation. No systemic drug-related adverse events were observed.Conclusion: While in use, oral intake of VPA indicated a short-term benefit to patients with RP. It is necessary to examine the effect of a longer VPA supplementation in a controlled study design. Keywords: retinitis pigmentosa, valproic acid, clinical trial, retina
format article
author Iraha S
Hirami Y
Ota S
Sunagawa GA
Mandai M
Tanihara H
Takahashi M
Kurimoto Y
author_facet Iraha S
Hirami Y
Ota S
Sunagawa GA
Mandai M
Tanihara H
Takahashi M
Kurimoto Y
author_sort Iraha S
title Efficacy of valproic acid for retinitis pigmentosa patients: a pilot study
title_short Efficacy of valproic acid for retinitis pigmentosa patients: a pilot study
title_full Efficacy of valproic acid for retinitis pigmentosa patients: a pilot study
title_fullStr Efficacy of valproic acid for retinitis pigmentosa patients: a pilot study
title_full_unstemmed Efficacy of valproic acid for retinitis pigmentosa patients: a pilot study
title_sort efficacy of valproic acid for retinitis pigmentosa patients: a pilot study
publisher Dove Medical Press
publishDate 2016
url https://doaj.org/article/b6dd3044a84d4574a3445b30818c6d63
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