Idebenone – a new drug for patients with Leber's hereditary optic neuropathy Review article
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Published:
Mar 17, 2021
Keywords:
idebenone, Leber hereditary optic neuropathy, LHON
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Abstract
Leber's Hereditary Optic Neuropathy is a rare disease caused by a mutation in the mitochondrial DNA. It appears most often in young men, leading to profound, permanent loss of vision in a short time. There is no specific treatment for this condition.
Idebenone is a medicine that administered to patients with Leber's hereditary optic neuropathy improves or stops the deterioration of vision. Increasing the number of people during therapy and the duration of treatment will allow in the future to answer the question, whether this drug is effective and safe in a larger group of patients.
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How to Cite
1.
Szumny D, Czajor K, Mulak M. Idebenone – a new drug for patients with Leber’s hereditary optic neuropathy. Ophthatherapy [Internet]. 2021Mar.17 [cited 2024Nov.24];8(1):32-6. Available from: https://journalsmededu.pl/index.php/ophthatherapy/article/view/1160
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Section
Conservative treatment
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Address reprint requests to: Medical Education, Marcin Kuźma (marcin.kuzma@mededu.pl)
References
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2. Majander A, Bowman R, Poulton J et al. Childhood-onset Leber hereditary optic neuropathy. Br J Ophthalmol. 2017; 101(11): 1505-9.
3. Priglinger C, Klopstock T, Rudolph G et al. Leber’sche hereditäre Optikusneuropathie. Klin Monbl Augenheilkd. 2019; 236(11): 1271-82.
4. Yu-Wai-Man P, Votruba M, Moore AT et al. Treatment strategies for inherited optic neuropathies: past, present and future. Eye (Lond). 2014; 28(5): 521-37.
5. Roskal-Wałek J, Gierada M, Mackiewicz J. Dziedziczna neuropatia wzrokowa Lebera – opis przypadku. Klinika Oczna / Acta Ophthalmologica Polonica. 2018; 120(4): 227-31.
6. Piotrowska-Nowak A, Krawczyński MR, Kosior-Jarecka E et al. Mitochondrial genome variation in male LHON patients with the m.11778G> A mutation. Metab Brain Dis. 2020; 35(8): 1317-27.
7. Wang M, Guo H, Li S et al. Electrophysiological and Structural Changes in Chinese Patients with LHON. J Ophthalmol. 2020; 2020: 4734276.
8. Newman NJ. Treatment of hereditary optic neuropathies. Nat Rev Neurol. 2012; 8(10): 545-56.
9. Suno M, Nagaoka A. Inhibition of lipid peroxidation by idebenone in brain mitochondria in the presence of succinate. Arch Gerontol Geriatr. 1989; 8(3): 291-7.
10. Lyseng-Williamson KA. Idebenone: a review in Leber’s hereditary optic neuropathy. Drugs. 2016; 76(7): 805-13.
11. Carelli V, Consensus Study Group. Consensus on guidelines for idebenone administration in Leber's hereditary optic neuropathy (LHON). Acta Ophthalmologica, 2016; 94.
12. Klopstock T, Metz G, Yu-Wai-Man P et al. Persistence of the treatment effect of idebenone in Leber’s hereditary optic neuropathy. Brain. 2013; 136(2): e230.
13. Carelli V, La Morgia C, Valentino ML et al. Idebenone treatment in Leber's hereditary optic neuropathy. Brain. 2011; 134(9): e188.
14. Mashima Y, Hiida Y, Oguchi Y. Remission of Leber's hereditary optic neuropathy with idebenone. Lancet. 1992; 340(8815): 368-9.
15. Barboni P, Savini G, Valentino ML et al. Leber’s hereditary optic neuropathy with childhood onset. Invest Ophthalmol Vis Sci. 2006; 47(12): 5303-9.
16. Dimitriadis K, Leonhardt M, Yu-Wai-Man P et al. Leber’s hereditary optic neuropathy with late disease onset: clinical and molecular characteristics of 20 patients. Orphanet J Rare Dis. 2014; 9(1): 158.
17. Carelli V, Valentino ML, Liguori R et al. Leber's hereditary optic neuropathy (LHON/11778) with myoclonus: report of two cases. J Neurol Neurosurg Psychiatry. 2001; 71(6): 813-6.
18. Watanabe M, Mita S, Takita T et al. Leber's hereditary optic neuropathy with dystonia in a Japanese family. J Neurol Sci. 2006; 243(1-2): 31-4.
19. Nikoskelainen E, Marttila RJ, Huoponen K et al. Leber's "plus": neurological abnormalities in patients with Leber's hereditary optic neuropathy. J Neurol Neurosurg Psychiatry. 1995; 59(2): 160-4.
20. Harding A, Sweeney MG, Miller DH et al. Occurrence of a multiple sclerosis-like illness in women who have a Leber's hereditary optic neuropathy mitochondrial DNA mutation. Brain. 1992; 115(4): 979-89.
21. Cortelli P, Montagna P, Pierangeli G et al. Clinical and brain bioenergetics improvement with idebenone in a patient with Leber's hereditary optic neuropathy: a clinical and 31P-MRS study. J Neurol Sci. 1997; 148(1): 25-31.
22. Bianco A, Martínez-Romero I, Bisceglia L et al. Mitochondrial DNA copy number differentiates the Leber’s hereditary optic neuropathy affected individuals from the unaffected mutation carriers. Brain. 2016; 139(1): e1.
23. Augustyniak J, Lenart J, Zychowicz M et al. Mitochondrial biogenesis and neural differentiation of human iPSC is modulated by idebenone in a developmental stage-dependent manner. Biogerontology. 2017; 18(4): 665-77.
2. Majander A, Bowman R, Poulton J et al. Childhood-onset Leber hereditary optic neuropathy. Br J Ophthalmol. 2017; 101(11): 1505-9.
3. Priglinger C, Klopstock T, Rudolph G et al. Leber’sche hereditäre Optikusneuropathie. Klin Monbl Augenheilkd. 2019; 236(11): 1271-82.
4. Yu-Wai-Man P, Votruba M, Moore AT et al. Treatment strategies for inherited optic neuropathies: past, present and future. Eye (Lond). 2014; 28(5): 521-37.
5. Roskal-Wałek J, Gierada M, Mackiewicz J. Dziedziczna neuropatia wzrokowa Lebera – opis przypadku. Klinika Oczna / Acta Ophthalmologica Polonica. 2018; 120(4): 227-31.
6. Piotrowska-Nowak A, Krawczyński MR, Kosior-Jarecka E et al. Mitochondrial genome variation in male LHON patients with the m.11778G> A mutation. Metab Brain Dis. 2020; 35(8): 1317-27.
7. Wang M, Guo H, Li S et al. Electrophysiological and Structural Changes in Chinese Patients with LHON. J Ophthalmol. 2020; 2020: 4734276.
8. Newman NJ. Treatment of hereditary optic neuropathies. Nat Rev Neurol. 2012; 8(10): 545-56.
9. Suno M, Nagaoka A. Inhibition of lipid peroxidation by idebenone in brain mitochondria in the presence of succinate. Arch Gerontol Geriatr. 1989; 8(3): 291-7.
10. Lyseng-Williamson KA. Idebenone: a review in Leber’s hereditary optic neuropathy. Drugs. 2016; 76(7): 805-13.
11. Carelli V, Consensus Study Group. Consensus on guidelines for idebenone administration in Leber's hereditary optic neuropathy (LHON). Acta Ophthalmologica, 2016; 94.
12. Klopstock T, Metz G, Yu-Wai-Man P et al. Persistence of the treatment effect of idebenone in Leber’s hereditary optic neuropathy. Brain. 2013; 136(2): e230.
13. Carelli V, La Morgia C, Valentino ML et al. Idebenone treatment in Leber's hereditary optic neuropathy. Brain. 2011; 134(9): e188.
14. Mashima Y, Hiida Y, Oguchi Y. Remission of Leber's hereditary optic neuropathy with idebenone. Lancet. 1992; 340(8815): 368-9.
15. Barboni P, Savini G, Valentino ML et al. Leber’s hereditary optic neuropathy with childhood onset. Invest Ophthalmol Vis Sci. 2006; 47(12): 5303-9.
16. Dimitriadis K, Leonhardt M, Yu-Wai-Man P et al. Leber’s hereditary optic neuropathy with late disease onset: clinical and molecular characteristics of 20 patients. Orphanet J Rare Dis. 2014; 9(1): 158.
17. Carelli V, Valentino ML, Liguori R et al. Leber's hereditary optic neuropathy (LHON/11778) with myoclonus: report of two cases. J Neurol Neurosurg Psychiatry. 2001; 71(6): 813-6.
18. Watanabe M, Mita S, Takita T et al. Leber's hereditary optic neuropathy with dystonia in a Japanese family. J Neurol Sci. 2006; 243(1-2): 31-4.
19. Nikoskelainen E, Marttila RJ, Huoponen K et al. Leber's "plus": neurological abnormalities in patients with Leber's hereditary optic neuropathy. J Neurol Neurosurg Psychiatry. 1995; 59(2): 160-4.
20. Harding A, Sweeney MG, Miller DH et al. Occurrence of a multiple sclerosis-like illness in women who have a Leber's hereditary optic neuropathy mitochondrial DNA mutation. Brain. 1992; 115(4): 979-89.
21. Cortelli P, Montagna P, Pierangeli G et al. Clinical and brain bioenergetics improvement with idebenone in a patient with Leber's hereditary optic neuropathy: a clinical and 31P-MRS study. J Neurol Sci. 1997; 148(1): 25-31.
22. Bianco A, Martínez-Romero I, Bisceglia L et al. Mitochondrial DNA copy number differentiates the Leber’s hereditary optic neuropathy affected individuals from the unaffected mutation carriers. Brain. 2016; 139(1): e1.
23. Augustyniak J, Lenart J, Zychowicz M et al. Mitochondrial biogenesis and neural differentiation of human iPSC is modulated by idebenone in a developmental stage-dependent manner. Biogerontology. 2017; 18(4): 665-77.