Berman-Gund Laboratory for the Study of Retinal Degenerations, Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston.
Channing Division of Network Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts.
JAMA Ophthalmol. 2018 May 1;136(5):490-495. doi: 10.1001/jamaophthalmol.2018.0590.
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.
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.
Age-adjusted dose of oral vitamin A palmitate (≤15 000 IU/d).
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.
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.
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.
虽然口服维生素 A 补充剂被认为可能会减缓成年色素性视网膜炎患者视网膜功能的丧失，且这些患者的肝功能正常，但针对此类疾病患儿的数据却很少。
比较服用和未服用维生素 A 补充剂的色素性视网膜炎患儿的疾病进程。
设计、地点和参与者：回顾性、非随机比较，在马萨诸塞州眼耳医院的视网膜电图服务处接受了平均 4 至 5 年随访的维生素 A 和对照组患儿。该研究包括具有不同遗传类型的典型色素性视网膜炎患儿：55 名服用维生素 A，25 名未服用维生素 A。患者评估日期范围为 1976 年 6 月至 2016 年 7 月，数据分析于 2016 年 10 月进行。
口服维生素 A 棕榈酸酯（≤15000 IU/d）的年龄调整剂量。
使用重复测量的纵向回归，在没有和调整潜在混杂因素的情况下，估计全视野圆锥体视网膜电图幅度 30-Hz 闪烁的平均指数变化率。
维生素 A 组的 55 名儿童中，38 名（69%）为男性；平均（SD）年龄为 9.1（1.9）岁；48 名（87%）为白人，6 名（11%）为亚洲人，1 名（2%）为黑人。对照组的 25 名成员中，19 名（76%）为男性；平均（SD）年龄为 9.2（1.7）岁；25 名（100%）为白人。未调整模型的估计平均变化率为维生素 A 组为 -0.0713 loge 单位/年（-6.9%/年），对照组为 -0.1419 loge 单位/年（-13.2%/年）（差异，0.0706 loge 单位/年；95%CI 的差异，0.0149-0.1263 loge 单位/年；P = .01）。调整模型证实，维生素 A 组的平均下降速度较慢（差异，0.0771 loge 单位/年；95%CI 的差异，0.0191-0.1350 loge 单位/年；P = .009）。关于眼部安全性，视野面积和视力的平均指数变化率以及至少 1 只眼的视野直径降至 20°或更小或视力降至 20/200 或更小的发生率在两组之间没有差异。
棕榈酸维生素 A 补充剂与色素性视网膜炎患儿的圆锥体视网膜电图幅度丧失速度较慢有关。尽管相对较小的样本量、回顾性、非随机设计不能检验因果关系，并且可能存在偏倚，但这些发现支持在大多数常见形式的色素性视网膜炎患儿的管理中考虑使用年龄调整剂量的维生素 A。
