• Vitamin A Supplements and Retinitis Pigmentosa in Children

    Written By: Lynda Seminara
    Selected By: Neil M. Bressler, MD, and Deputy Editors

    Journal Highlights

    JAMA Ophthalmology, May 2018

    Download PDF

    Supplementation with vitamin A may slow the loss of retinal function in adults with retinitis pigmentosa (RP), but little is known about its effect in children with the disease. Berson et al. compared the disease course of RP between children using oral vitamin A palmitate and a control group. Although definitive conclusions could not be drawn from their small retrospective study, the findings suggest that vitamin A therapy may slow the loss of cone function in children with common forms of RP.

    For this nonrandomized retrospec­tive study, the researchers evaluated 80 children with RP, 55 of whom received ≤15,000 IU of vitamin A palmitate per day. The remaining 25 children served as a control group. Both cohorts were followed for several years by the Elec­troretinography Service of the Massa­chusetts Eye and Ear Infirmary (dates for patient evaluations ranged from 1976 to 2016, and data were analyzed in 2016). Sex distribution and mean age appeared similar for the study cohorts. The primary outcome was the mean exponential rate of change of full-field cone electroretinogram (ERG) am­plitude to 30-Hz flashes, estimated by repeated-measures longitudinal regres­sion, with and without adjustment for potential confounders.

    According to the unadjusted model, the estimated mean rate of change was −0.0713 loge unit/year for the vitamin cohort and −0.1419 loge unit/year for the control cohort (difference, 0.0706 loge unit/year; p = .01). The adjusted model showed that the mean rate of decline was slower for the vitamin cohort (difference, 0.0771 loge unit/year; p = .009). Ocular safety and the mean exponential change rates in visual acuity and visual field area appeared similar for the study groups.

    The authors acknowledged that their study has several limitations, but the findings appear to support consideration of age-appropriate vitamin A therapy in children with common forms of RP and normal liver function. They suggest that vitamin A supplementa­tion may be particularly beneficial for children with long cone ERG implicit time, who have a high risk for aggres­sive disease. (Also see related commen­tary by Caroline C.W. Klaver, MD, PhD, and Alberta A.H.J. Thiadens, MD, PhD, in the same issue.)

    The original article can be found here.