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Treatment of Leber's hereditary optic neuropathy with EPI-743: the Brazilian experience

2015; Wiley; Volume: 93; Linguagem: Inglês

10.1111/j.1755-3768.2015.0708

1755-3768

Rustum Karanjia, Amitha Ganti, Edward Chu, Tana Wagschal, Rubens Belfort, Milton N. Moraes-Filho, Solange Rios Salomão, Filipe Chicani, Michelle E Guy, Alfredo A. Sadun,

Drug-Induced Ocular Toxicity

Purpose To determine the degree of visual improvement in a homogeneous Brazilian cohort of Leber's Hereditary Optic Neuropathy (LHON) 11778 receiving 18–24 months of therapy with the quinone EPI-743 Methods Six patients with severe vision loss from LHON were enrolled in an open label clinical trial. Each patient received 400 mg TID of EPI-743. Two patients were treated at the time of conversion, while 4 patients had chronic disease (>5 years). Outcome measures included best corrected visual acuity (BCVA), retinal nerve fiber layer (RNFL) thickness as measured by Optical Coherence Tomography and Humphrey Visual Fields (HVF) mean deviations. Mean deviations were calculated using the HVF algorithm (Stim III) or University of Iowa Visual Field Reading Center Algorithm (Stim V) Results VA declined or stabilized in the first 6 months, then improved gradually for all 6 patients and remained stable for 18–24 months with EPI-743. VF increased marginally in one chronic and both acute patients, while 3 chronic patients remained at baseline. The acute patients showed continued bilateral progression for the first 6 months, consistent with the natural history of LHON. As expected, all eyes showed a decline in RNFL that stabilized after treatment Conclusions VA improvements continued beyond the initial 1 year period to 18–24 months of treatment, including 4 patients who had lost vision over 5 years prior to start of EPI-743. This data is consistent with data from the United States where an EPI-743.