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Treatment of Leber's hereditary optic neuropathy with EPI ‐743: the Brazilian experience
Author(s) -
Karanjia R.,
Ganti A.,
Chu E.,
Wagschal T.,
Belfort R.,
MoraesFilho M.,
Salomao S.,
Chicani F.,
Guy M.,
Sadun A.
Publication year - 2015
Publication title -
acta ophthalmologica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.534
H-Index - 87
eISSN - 1755-3768
pISSN - 1755-375X
DOI - 10.1111/j.1755-3768.2015.0708
Subject(s) - medicine , nerve fiber layer , leber's hereditary optic neuropathy , ophthalmology , visual acuity , visual field , retinal , optic neuropathy , cohort , homogeneous , nerve fibre layer , pediatrics , optic nerve , physics , thermodynamics
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.