June 2013
Volume 54, Issue 15
Free
ARVO Annual Meeting Abstract  |   June 2013
Long-term efficacy and safety of treatment of retinitis pigmentosa with valproic acid
Author Affiliations & Notes
  • Christine Kay
    Ophthalmology, University of Florida, Gainesville, FL
  • Sheena Bhalla
    Ophthalmology, University of Florida, Gainesville, FL
  • Footnotes
    Commercial Relationships Christine Kay, None; Sheena Bhalla, None
  • Footnotes
    Support None
Investigative Ophthalmology & Visual Science June 2013, Vol.54, 4016. doi:https://doi.org/
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      Christine Kay, Sheena Bhalla; Long-term efficacy and safety of treatment of retinitis pigmentosa with valproic acid. Invest. Ophthalmol. Vis. Sci. 2013;54(15):4016. doi: https://doi.org/.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract
 
Purpose
 

The purpose of this study was to examine the long-term safety and efficacy of valproic acid (VPA) use in patients with retinitis pigmentosa (RP) and other retinal degenerative disorders that were offered off-label use of this drug by a previous investigator at the University of Florida. More recently, any patients currently seen at the University of Florida who had not already self-terminated use of VPA were asked to stop this drug until further prospective data on safety and efficacy is released.

 
Methods
 

This study was a retrospective chart review of all patients with retinal dystrophies who had been prescribed VPA at the University of Florida Ophthalmology Department clinic between January 2009 and April 2012. Visual field (VF), visual acuity (VA), length of treatment, liver enzymes, and side effects were analyzed. Visual field (VF) areas were defined using Goldmann visual field (GVF) tracings recorded before, during, and after VPA treatment using the V4e isopter for each eye. Areas determined by isopter V4e were converted into areas of functioning retina, as shown in figure 1.

 
Results
 

Five of the patients (10 eyes) had two GVF tracings, allowing comparison between baseline and follow-up VF. After 9.8 months of VPA, VF decreased by 0.145 cm2 (26.478%) (p=0.432) as shown in Figure 2. For 22 of the patients (41 eyes), VA data was available, and logMAR score changed by 0.056 log units (representing a decline in VA) after 14.9 months on VPA (p=0.002). Twelve patients (38.7%) reported negative side effects related to VPA use and 9 (29%) patients discontinued VPA due to these side effects.

 
Conclusions
 

We found that VPA may not be an appropriate treatment for all retinal dystrophies. After an average of 9.8 months on VPA, visual field areas showed a declining trend in four out of five patients and visual acuity significantly worsened during treatment with VPA. VPA plays a complex role in patients with pigmentary retinopathy and may be associated with visual acuity and field decline as well as adverse side effects. Physicians should use caution with using VPA for pigmentary retinopathies. Effective treatment options likely depend on first identifying the disease-causing mutation and then optimizing treatment targeting genotype-specific pathology.

 
 
GVF before and after 19 months of VPA treatment.
 
GVF before and after 19 months of VPA treatment.
 
 
Percent change in visual field while on VPA treatment compared to baseline.
 
Percent change in visual field while on VPA treatment compared to baseline.
 
Keywords: 702 retinitis • 494 degenerations/dystrophies • 642 perimetry  
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