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Therapeutic potential of valproic acid for retinitis pigmentosa
C M Clemson1, R Tzekov, M Krebs
1Department of Ophthalmology, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA.
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.
