Is leber hereditary optic neuropathy treatable? Encouraging results with idebenone in both prospective and retrospective trials and an illustrative case

Journal of Neuro-Ophthalmology

Volumn 32, Issue 1, 2012, Pages 54-57

  Sabet Peyman, Esfandiar J ^a   Khaderi, Khizer R ^a   Sadun, Alfredo A ^a  

^a UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

IDEBENONE; METHYLPREDNISOLONE; UBIDECARENONE;

ADULT; ARTICLE; CASE REPORT; DRUG EFFICACY; DRUG MEGADOSE; FEMALE; FOLLOW UP; HUMAN; LEBER HEREDITARY OPTIC NEUROPATHY; LONG TERM CARE; OPHTHALMOSCOPY; PRIORITY JOURNAL; PROSPECTIVE STUDY; RETROSPECTIVE STUDY; TREATMENT OUTCOME; VISUAL ACUITY; VISUAL FIELD DEFECT; VISUAL IMPAIRMENT;

ADULT; ANTIOXIDANTS; CLINICAL TRIALS AS TOPIC; FEMALE; HUMANS; OPTIC ATROPHY, HEREDITARY, LEBER; PROSPECTIVE STUDIES; RECOVERY OF FUNCTION; RETROSPECTIVE STUDIES; TREATMENT OUTCOME; UBIQUINONE;

EID: 84857820970     PISSN: 10708022     EISSN: 15365166     Source Type: Journal    
DOI: 10.1097/WNO.0b013e318241da45     Document Type: Article

References (16)

1. Klopstock T, Yu-Wai-Man P, Dimitriadis K, Rouleau J, Heck S, Bailie M, Atawan A, Chattopadhyay S, Schubert M, Garip A, Kernt M, Petraki D, Rummey C, Leinonen M, Metz G, Griffiths P, Meier T, Chinnery P. A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy. Brain. 2011;134:2677-2686.
2. Carelli V, La Morgia C, Valentino ML, Rizzo G, Carbonelli M, De Negri AM, Sadun F, Carta A, Guerriero S, Simonelli F, Sadun AA, Aggarwal D, Liguori R, Avoni P, Baruzzi A, Zeviani M, Montagna P, Barboni P. Idebenone treatment in Leber's hereditary optic neuropathy. Brain. 2011;134:e188.
3. Stone EM, Newman NJ, Miller NR, Johns DR, Lott MT, Wallace DC. Visual recovery in patients with Leber's hereditary optic neuropathy and the 11778 mutation. J Clin Neuroophthalmol. 1992;12:10-14.
4. Barboni P, Carbonelli M, Savini G, Ramos Cdo V, Carta A, Berezovsky A, Salomao S, Carelli V, Sadun AA. Natural history of Leber's hereditary optic neuropathy: Longitudinal analysis of the retinal nerve fiber layer by optical coherence tomography. Ophthalmology. 2010;117:623-627.
5. Fraser J, Biousse V, Newman N. The neuro-ophthalmology of mitochondrial disease. Surv Ophthalmol. 2010;55:299-334.
6. Carelli V, Ross-Cisneros FN, Sadun AA. Mitochondrial dysfunction as a cause of optic neuropathies. Prog Retin Eye Res. 2004;23:53-89.
7. Wallace DC, Singh G, Lott MT, Hodge JA, Schurr TG, Lezza AM, Elsas LJ II, Nikoskelainen EK. Mitochondrial DNA mutation associated with Leber's hereditary optic neuropathy. Science. 1988;242:1427-1430.
8. Geromel V, Darin N, Chrétien D, Bénit P, DeLonlay P, Rötig A, Munnich A, Rustin P. Coenzyme Q(10) and idebenone in the therapy of respiratory chain diseases: Rationale and comparative benefits. Mol Genet Metab. 2002;77:21-30.
9. Sadun AA, La Morgia C, Carelli V. Leber's hereditary optic neuropathy. Curr Treat Options Neurol. 2011;13:109-117.
10. Sadun AA, Wang MY. Ethambutol optic neuropathy: How we can prevent 100,000 new cases of blindness each year. J Neuroophthalmol. 2008;28:265-268.
11. Shrader WD, Amagata A, Barnes A, Enns GM, Hinman A, Jankowski O, Kheifets V, Komatsuzaki R, Lee E, Mollard P, Murase K, Sadun AA, Thoolen M, Wesson K, Miller G. Alphatocotrienol quinone modulates oxidative stress response and the biochemistry of aging. Bioorg Med Chem Lett. 2011;21:3693-3698.
12. Mashima Y, Hiida Y, Oguchi Y. Remission of Leber's hereditary optic neuropathy with idebenone. Lancet. 1992;340:368-369.
13. Mashima Y, Kigasawa K, Wakakura M, Oguchi Y. Do idebenone and vitamin therapy shorten the time to achieve visual recovery in Leber hereditary optic neuropathy? J Neuroophthalmol. 2000;20:166-170.
14. Eng J, Aggarwal D, Sadun AA. Idebenone treatment in patients with Leber hereditary optic neuropathy. Invest Ophthalmol Vis Sci. 2009;50:1440.
15. Newman NJ. Treatment of Leber hereditary optic neuropathy. Brain. 2011;134:2447-2455.
16. Sadun AA, Chicani C, Ross-Cisneros F, Barboni P, Thoolen M, Shrader W, Kubis K, Carelli V, Miller G. Epi-743 alters the clinical course of the mitochondrial disease Leber's hereditary optic neuropathy Arch Neurol. In press.