【摘要】 目的观察磷酸肌酸钠治疗Leber遗传性视神经病变(LHON)的有效性。设计回顾性病例系列。研究对象2014年1月至2016年1月武汉同济医院眼科确诊的LHON患者19例。根据患者是否同意住院治疗分为两组,10例住院者为治疗组,9例门诊观察未做治疗者为对照组。方法对治疗组的10例LHON患者进行磷酸肌酸钠治疗,每日2次,每次1 g,加入250 ml生理盐水中静脉滴注,共7天。观察患者治疗前后的视力、视野变化。对照组的9例患者未治疗。19例患者随访观察1年。主要指标视力、视野。结果与治疗前视力相比,治疗7天后,治疗组患者中3例患者(1号右眼,6号右眼,7号左眼)视力提高,分别提高0.3 LogMAR、0.4 LogMAR、0.7 LogMAR。随访1年后,1号右眼视力下降,6号和7号视力无变化。3例患者中14484位点突变的有2例(6号和7号),11778位点突变的有1例(1号);另外7例视力无显著改变。10例患者的视野(VFI和MD)治疗前后无显著差异(P=0.167,0.20)。未经治疗的9例患者随访观察1年,视力无变化。结论磷酸肌酸钠治疗Leber遗传性视神经病变或许对14484位点突变患者的视力提高或稳定有一定的疗效。更多还原

【Abstract】 Objective To observe the efficacy of sodium phosphate creatine in the treatment of Leber hereditary optic neuropathy(LHON). Design Retrospective case series. Participants Nineteen LHON patients diagnosed in Department of Ophthalmology, Wuhan Tongji Hospital from January 2014 to January 2016. They were divided into two groups according to whether the patients were admitted to hospital, 10 patients were Inpatient treatment and 9 in the untreated control group. Methods The 10 patients were treated with intravenous infusion of 1 g creatine phosphate sodium solved in 250 ml normal saline, twice a day for 7 days. The visual acuity and visual field of the patients before and after treatment were observed. Nine of the untreated patients were enrolled into the control group. The patients were followed for 1 year. Main Outcome Measures Visual acuity, visual field. Results The visual acuity of 3 treated patients(right eye of first patient, right eye of the 6th patient, left eye of the 7th patient) increased by 0.3 LogMAR, 0.4 LogMAR, 0.7 LogMAR,respectively. One year after treatment, visual acuity decreased in the 1st patient, no change in the 6th and 7th patient. There were 2cases(6th and 7th) of 14484 mutation in these 3 responsive cases, 1 case(1st) of 11778 mutation, and 7 cases had no significant change in visual acuity. There were no significant differences in the visual field(VFI and MD) between the 10 patients before and after treatment(P=0.167, 0.20). Untreated patients were followed for 1 year with no change in visual acuity. Conclusion Sodium phosphate creatine may have some effect in maintaining or improving visual acuity of LHON patients with 14484 mutations.更多还原