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Original Investigation
May 2018

Association of Vitamin A Supplementation With Disease Course in Children With Retinitis Pigmentosa

Author Affiliations
  • 1Berman-Gund Laboratory for the Study of Retinal Degenerations, Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston
  • 2Channing Division of Network Medicine, Brigham and Women’s Hospital, Harvard Medical School, Boston, Massachusetts
  • 3Eye Disease Consultants, West Hartford, Connecticut
JAMA Ophthalmol. 2018;136(5):490-495. doi:10.1001/jamaophthalmol.2018.0590
Key Points

Question  What were the outcomes in children with common forms of retinitis pigmentosa following supplementation with vitamin A palmitate?

Findings  In this case-control study of children with retinitis pigmentosa, including 55 taking an age-adjusted dose of vitamin A (5000-15 000 IU/d) and 25 not taking vitamin A, vitamin A supplementation was associated with almost 50% slowing of the mean exponential rate of decline of full-field cone electroretinogram amplitude.

Meaning  These findings, while not definitive in the absence of randomized controls, support the hypothesis that vitamin A palmitate can slow loss of cone function in children with common forms of 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 loge unit/y (−6.9% per year) for the vitamin A cohort and −0.1419 loge unit per year (−13.2% per year) for the control cohort (difference, 0.0706 loge unit per year; 95% CI for the difference, 0.0149-0.1263 loge unit per year; P = .01). The adjusted model confirmed a slower mean rate of decline in the vitamin A cohort (difference, 0.0771 loge-unit per year; 95% CI for the difference, 0.0191-0.1350 loge-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° 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.