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 2 cases (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. (Ophthalmol CHN, 2017, 26: 126-130)

Key words: Leber hereditary optic neuropathy (LHON); , creatine phosphate sodium