Association Of Vitamin A Supplementation With Disease Course In Children With Retinitis Pigmentosa

IMPORTANCE While oral vitamin A supplementation is considered to potentially slow loss of retinal function in adults with retinitis pigmentosa and normal liver function, little data from children with this disease are available.OBJECTIVE To compare disease courses in children with retinitis pigmentosa taking or not taking vitamin A supplementation.DESIGN. SETTING. AND PARTICIPANTS Retrospective, nonrandomized comparison of vitamin A and control cohorts followed up for a mean of 4 to 5 years by the Electroretinography Service of the Massachusetts Eye and Ear Infirmary. The study included children with different genetic types of typical retinitis pigmentosa: 55 taking vitamin A and 25 not taking vitamin A. The dates for patient evaluations ranged from June 1976 to July 2016, and the data analysis occurred in October 2016.INTERVENTIONS Age-adjusted dose of oral vitamin A palmitate (<= 15 000 IU/d).MAIN OUTCOMES AND MEASURES Mean exponential rates of change of full-field cone electroretinogram amplitude to 30-Hz flashes estimated by repeated-measures longitudinal regression without and with adjusting for potential confounders.RESULTS Of the 55 children in the vitamin A cohort, 38 (69%) were male; the mean [SD] age was 9.1 [1.9] years; and 48 (87%) were white , 6 (11%) were Asian, and 1(2%) was black. Of the 25 members of the control cohort, 19 (76%) were male; the mean [SD] age was 9.2 [1.7] years; and 25 (100%) were white. The estimated mean rates of change with the unadjusted model were -0.0713 log(e) unit/y (-6.9% per year) for the vitamin A cohort and -0.1419 log(e) unit per year (-13.2% per year) for the control cohort (difference, 0.0706 log e unit per year; 95% CI for the difference, 0.0149-0.1263 log(e) unit per year; P = .01). The adjusted model confirmed a slower mean rate of decline in the vitamin A cohort (difference, 0.0771 log(e)-unit per year; 95% CI for the difference, 0.0191-0.1350 log(e)-unit per year; P = .009). With respect to ocular safety, the mean exponential rates of change of visual field area and visual acuity and the incidences of falling to a visual field diameter of 20 degrees or less or a visual acuity of 20/200 or less in at least 1 eye did not differ by cohort.CONCLUSIONS AND RELEVANCE A vitamin A palmitate supplement was associated with a slower loss of cone electroretinogram amplitude in children with retinitis pigmentosa. Although the relatively small-sample, retrospective, nonrandomized design does not allow a test of causation and is subject to possible biases, these findings support consideration of an age-adjusted dose of vitamin A in the management of most children with the common forms of retinitis pigmentosa.