Do idebenone and vitamin therapy shorten the time to achieve visual recovery in Leber hereditary optic neuropathy?
OBJECTIVE
The authors investigated the effectiveness of idebenone combined with vitamin B2 and vitamin C in the treatment of patients with Leber hereditary optic neuropathy (LHON) in an early stage as compared with untreated patients with LHON. These agents may stimulate the formation of ATP.


METHODS
For this retrospective study, the authors selected 28 outpatients with LHON from the Keio University Hospital. These patients were followed for 2 to 19 years from disease onset. They were divided into two groups: 14 untreated patients (11778 mutation in 10 patients, 3460 mutation in 2 patients, and 14484 mutation in 2 two patients); and 14 treated patients (11778 mutation in 11 patients, 3460 mutation in 1 patient, and 14484 mutation in 2 patients). The treated patients were administered medical treatment with idebenone, vitamin B2, and vitamin C for at least 1 year. The current study evaluated the following: 1) number of eyes with visual recovery > or = 0.3; 2) interval between the onset of LHON and the beginning of visual recovery; 3) interval between the onset of LHON and visual recovery to 0.3; and 4) interval between the beginning of medical treatment and the beginning of visual recovery in the treated subjects.


RESULTS
There was no significant difference in the number of eyes with visual recovery > or = 0.3 in the two groups with the 3460, 11778, or 14484 mutation. Patients with visual recovery showed a fenestrated scotoma or a clearing of central vision. The mean interval between the onset of LHON and the beginning of visual recovery was significantly shorter in the treated group (11.1 months) than in the untreated group (17.4 months) (P = 0.03). The mean interval between the onset of LHON and visual recovery to 0.3 was significantly shorter in the treated group (17.6 months) than in the untreated group (34.4 months) (P = 0.01). The mean interval between the initiation of medical treatment to the beginning of visual recovery was 5.4 months.


CONCLUSIONS
Results suggest that the administration of idebenone, vitamin B2, and vitamin C sped the recovery of vision in patients with LHON.
Mashima
Y
Y
Department of Ophthalmology, Keio University School of Medicine, Tokyo, Japan.
Kigasawa
K
K
Wakakura
M
M
Oguchi
Y
Y
D000293:Adolescent; D000328:Adult; D017668:Age of Onset; D000975:Antioxidants / Q000627:therapeutic use*; D001205:Ascorbic Acid / Q000627:therapeutic use*; D016227:Benzoquinones / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D009154:Mutation; D015418:Optic Atrophies, Hereditary / Q000150:complications / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology; D020127:Recovery of Function / Q000187:drug effects; D012189:Retrospective Studies; D012256:Riboflavin / Q000627:therapeutic use*; D013997:Time Factors; D014451:Ubiquinone / Q000031:analogs & derivatives; D014786:Vision Disorders / Q000188:drug therapy* / Q000209:etiology / Q000503:physiopathology; D014792:Visual Acuity / Q000187:drug effects* / Q000502:physiology
D000975:Antioxidants; D016227:Benzoquinones; D014451:Ubiquinone; C036619:idebenone; D001205:Ascorbic Acid; D012256:Riboflavin