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OPA1-related optic atrophy with or without extraocular features

MedGen UID:
1002107
Concept ID:
CN322459
Disease or Syndrome
Monarch Initiative: MONDO:0800181

Definition

Any primary mitochondrial disease in which the cause of the disease is monoallelic or biallelic variants in the OPA1 gene. While optic atrophy is present in most affected cases, OPA1 is a mitochondrial protein and thus features of this disease include abnormal mitochondrial morphology and multiple mitochondrial DNA deletions, and can affect other organ systems and. Extraocular features can include progressive sensorineural hearing impairment, cognitive impairment, peripheral neuropathy, myopathy, ragged-red muscle fibers, and exercise-induced lactic acidemia, while additional ocular features can include progressive visual loss, central scotoma, and color vision abnormalities. [from MONDO]

Professional guidelines

PubMed

Keene KR, Kan HE, van der Meeren S, Verbist BM, Tannemaat MR, Beenakker JM, Verschuuren JJGM
J Cachexia Sarcopenia Muscle 2022 Dec;13(6):2820-2834. Epub 2022 Sep 29 doi: 10.1002/jcsm.13089. PMID: 36172973Free PMC Article
Dolman PJ
J Endocrinol Invest 2021 Mar;44(3):421-429. Epub 2020 Jul 29 doi: 10.1007/s40618-020-01361-y. PMID: 32729049
Bornfeld N, Biewald E, Bauer S, Temming P, Lohmann D, Zeschnigk M
Dtsch Arztebl Int 2018 Feb 16;115(7):106-111. doi: 10.3238/arztebl.2018.0106. PMID: 29510820Free PMC Article

Recent clinical studies

Etiology

Shoji MK, Chen Y, Topilow NJ, Abou Khzam R, Dubovy SR, Johnson TE
Ophthalmic Plast Reconstr Surg 2023 Jul-Aug 01;39(4):347-356. Epub 2023 Jan 18 doi: 10.1097/IOP.0000000000002314. PMID: 36661857
Shapira Y, Juniat V, Dave T, Hussain A, McNeely D, Watanabe A, Yoneda A, Saeed P, Woo KI, Hardy TG, Price B, Drummond K, Selva D
Eye (Lond) 2023 Jan;37(1):48-53. Epub 2022 Jan 9 doi: 10.1038/s41433-021-01850-1. PMID: 34999720Free PMC Article
Klopstock T, Priglinger C, Yilmaz A, Kornblum C, Distelmaier F, Prokisch H
Dtsch Arztebl Int 2021 Nov 5;118(44):741-748. doi: 10.3238/arztebl.m2021.0251. PMID: 34158150Free PMC Article
Dolman PJ
J Endocrinol Invest 2021 Mar;44(3):421-429. Epub 2020 Jul 29 doi: 10.1007/s40618-020-01361-y. PMID: 32729049
Costan VV, Bogdănici CM, Gheorghe L, Obadă O, Budacu C, Grigoraș C, Andronic DG, Niagu IA
Rom J Ophthalmol 2020 Apr-Jun;64(2):116-121. PMID: 32685776Free PMC Article

Diagnosis

Carelli V, La Morgia C, Yu-Wai-Man P
Handb Clin Neurol 2023;194:23-42. doi: 10.1016/B978-0-12-821751-1.00010-5. PMID: 36813316
Mombaerts I, McNab AA
Curr Rheumatol Rep 2022 Jan;24(1):20-26. Epub 2022 Feb 9 doi: 10.1007/s11926-022-01052-y. PMID: 35138592
Klopstock T, Priglinger C, Yilmaz A, Kornblum C, Distelmaier F, Prokisch H
Dtsch Arztebl Int 2021 Nov 5;118(44):741-748. doi: 10.3238/arztebl.m2021.0251. PMID: 34158150Free PMC Article
Goseki T
Jpn J Ophthalmol 2021 Jul;65(4):448-453. Epub 2021 May 20 doi: 10.1007/s10384-021-00839-3. PMID: 34014448
Hirabayashi K, Zamboni G
Pathologica 2012 Apr;104(2):43-55. PMID: 22953500

Therapy

Del-Rio-Vellosillo M, Garcia-Medina JJ, Pinazo-Duran MD, Abengochea-Cotaina A, Barbera-Alacreu M
Reg Anesth Pain Med 2017 Jan/Feb;42(1):1-9. doi: 10.1097/AAP.0000000000000504. PMID: 27846186
Hatemi G, Seyahi E, Fresko I, Talarico R, Hamuryudan V
Clin Exp Rheumatol 2016 Sep-Oct;34(6 Suppl 102):10-22. Epub 2016 Oct 13 PMID: 27791958
Chow MT, Anderson SF
Optom Vis Sci 2000 Jun;77(6):286-92. doi: 10.1097/00006324-200006000-00008. PMID: 10879785
Scott IU, Siatkowski MR
Semin Ophthalmol 1999 Jun;14(2):52-61. doi: 10.3109/08820539909056064. PMID: 10758212
Ezra E, Spalton D, Sanders MD, Graham EM, Plant GT
Br J Ophthalmol 1996 Apr;80(4):350-5. doi: 10.1136/bjo.80.4.350. PMID: 8703889Free PMC Article

Prognosis

Shoji MK, Chen Y, Topilow NJ, Abou Khzam R, Dubovy SR, Johnson TE
Ophthalmic Plast Reconstr Surg 2023 Jul-Aug 01;39(4):347-356. Epub 2023 Jan 18 doi: 10.1097/IOP.0000000000002314. PMID: 36661857
Costan VV, Bogdănici CM, Gheorghe L, Obadă O, Budacu C, Grigoraș C, Andronic DG, Niagu IA
Rom J Ophthalmol 2020 Apr-Jun;64(2):116-121. PMID: 32685776Free PMC Article
Weidauer S, Hofmann C, Wagner M, Hattingen E
Neuroradiology 2019 Apr;61(4):365-387. Epub 2019 Feb 12 doi: 10.1007/s00234-019-02183-3. PMID: 30747268
Kaliki S, Shields CL, Shields JA
Indian J Ophthalmol 2015 Feb;63(2):93-102. doi: 10.4103/0301-4738.154367. PMID: 25827538Free PMC Article
Lenaers G, Hamel C, Delettre C, Amati-Bonneau P, Procaccio V, Bonneau D, Reynier P, Milea D
Orphanet J Rare Dis 2012 Jul 9;7:46. doi: 10.1186/1750-1172-7-46. PMID: 22776096Free PMC Article

Clinical prediction guides

Shapira Y, Juniat V, Dave T, Hussain A, McNeely D, Watanabe A, Yoneda A, Saeed P, Woo KI, Hardy TG, Price B, Drummond K, Selva D
Eye (Lond) 2023 Jan;37(1):48-53. Epub 2022 Jan 9 doi: 10.1038/s41433-021-01850-1. PMID: 34999720Free PMC Article
Keene KR, Kan HE, van der Meeren S, Verbist BM, Tannemaat MR, Beenakker JM, Verschuuren JJGM
J Cachexia Sarcopenia Muscle 2022 Dec;13(6):2820-2834. Epub 2022 Sep 29 doi: 10.1002/jcsm.13089. PMID: 36172973Free PMC Article
Tong JY, Juniat V, McKelvie PA, O'Donnell BA, Hardy TG, McNab AA, Selva D
Ophthalmic Plast Reconstr Surg 2022 May-Jun 01;38(3):234-241. Epub 2021 Sep 8 doi: 10.1097/IOP.0000000000002061. PMID: 34516528
Kaliki S, Shields CL, Shields JA
Indian J Ophthalmol 2015 Feb;63(2):93-102. doi: 10.4103/0301-4738.154367. PMID: 25827538Free PMC Article
Haik BG
Trans Am Ophthalmol Soc 1991;89:371-476. PMID: 1808814Free PMC Article

Recent systematic reviews

Vrabič N, Fakin A, Tekavčič Pompe M
J Fr Ophtalmol 2024 Oct;47(8):104232. Epub 2024 Aug 29 doi: 10.1016/j.jfo.2024.104232. PMID: 39213781
Garg D, Patel S, Sankhla CS, Holla VV, Paramanandam V, Kukkle PL, Pandey S, Schneider SA, Pal PK
Mov Disord Clin Pract 2024 Jul;11(7):770-785. Epub 2024 May 15 doi: 10.1002/mdc3.14062. PMID: 38748762Free PMC Article
Van Swol JM, Myers WK, Nguyen SA, Wilson ME
J Binocul Vis Ocul Motil 2023 Apr-Jun;73(2):43-52. Epub 2023 Feb 13 PMID: 36780505
Tan YJ, Ramesh R, Tan YH, Tan SML, Setiawan S
Clin Neurol Neurosurg 2023 Feb;225:107601. Epub 2023 Jan 19 doi: 10.1016/j.clineuro.2023.107601. PMID: 36696848Free PMC Article
Song C, Luo Y, Yu G, Chen H, Shen J
Front Endocrinol (Lausanne) 2022;13:991588. Epub 2022 Sep 29 doi: 10.3389/fendo.2022.991588. PMID: 36267571Free PMC Article

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