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Therapeutic potential of valproic acid for retinitis pigmentosa
  1. C M Clemson1,
  2. R Tzekov1,
  3. M Krebs2,
  4. J M Checchi1,
  5. C Bigelow3,
  6. S Kaushal1
  1. 1Department of Ophthalmology, University of Massachusetts Medical School, Worcester, Massachusetts, USA
  2. 2Ophthalmology Department, University of Florida, Gainesville, Florida, USA
  3. 3Department of Statistics, University of Massachusetts School of Public Health and Health Sciences, Amherst, Massachusetts, USA
  1. Correspondence to Dr Shalesh Kaushal, Department of Ophthalmology, University of Massachusetts Medical School, 381 Plantation St., Biotech 5, Suite 250, Worcester, MA 01605; shalesh.kaushal{at}


Background/aim To examine the efficacy and safety of valproic acid (VPA) in patients with retinitis pigmentosa (RP).

Methods Thirteen eyes were examined before and after brief treatment (average 4 months) with VPA. Visual fields (VF) for each eye were defined using digitised Goldmann Kinetic Perimetry tracings. VF areas were log-transformed and VF loss/gain relative to baseline was calculated. Visual acuity was measured using a Snellen chart at a distance of 20 feet (6.1 m). Values were converted to the logarithm of the minimum angle of resolution (logMAR) score.

Results Nine eyes had improved VF with treatment, two eyes had decreased VF and two eyes experienced no change, with an overall average increase of 11%. Assuming typical loss in VF area without treatment, this increase in VF was statistically significant (p<0.02). An average decrease (0.172) in the logMAR scores was seen in these 13 eyes, which translates to a positive change in Snellen score of approximately 20/47 to 20/32, which was significant (p<0.02) assuming no loss in acuity without treatment. Side effects were mild and well tolerated.

Conclusion Treatment with VPA is suggestive of a therapeutic benefit to patients with RP. A placebo-controlled clinical trial will be necessary to assess the efficacy and safety of VPA for RP rigorously.

  • Retinitis pigmentosa
  • valproic acid
  • visual fields
  • visual acuity
  • retina
  • apotosis
  • degeneration
  • drugs
  • field of vision

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  • Linked article 175364.

  • Funding Vision Research Fund at the University of Massachusetts Medical School.

  • Competing interests None.

  • Ethics approval This study was conducted with the approval of the University of Massachusetts Medical School Institutional Review Board.

  • Provenance and peer review Not commissioned; externally peer reviewed.

Linked Articles

  • PostScript
    Mary J van Schooneveld L Ingeborgh van den Born Mies van Genderen Jan-Geert Bollemeijer
  • PostScript
    Christine M Clemson Radouil Tzekov Jenna M Checchi Mark Krebs Shalesh Kaushal