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Optic atrophy 2(OPA2)

MedGen UID:
326915
Concept ID:
C1839576
Disease or Syndrome
Synonyms: OPA2; Optic atrophy, non-Leber type, with early onset; Optic atrophy, X-linked
SNOMED CT: Early-onset X-linked optic atrophy (721200000); Non-Leber type optic atrophy with early-onset (721200000); Optic atrophy type 2 (721200000)
Modes of inheritance:
X-linked recessive inheritance
MedGen UID:
375779
Concept ID:
C1845977
Finding
Source: Orphanet
A mode of inheritance that is observed for recessive traits related to a gene encoded on the X chromosome. In the context of medical genetics, X-linked recessive disorders manifest in males (who have one copy of the X chromosome and are thus hemizygotes), but generally not in female heterozygotes who have one mutant and one normal allele.
 
Monarch Initiative: MONDO:0010698
OMIM®: 311050
Orphanet: ORPHA98890

Definition

A rare form of hereditary optic atrophy seen in only 4 families to date. With onset in early childhood the disease has characteristics of progressive loss of visual acuity, significant optic nerve pallor and occasionally additional neurological manifestations, with females being unaffected. [from SNOMEDCT_US]

Clinical features

From HPO
Absent Achilles reflex
MedGen UID:
108240
Concept ID:
C0558845
Finding
Absence of the Achilles reflex (also known as the ankle jerk reflex), which can normally be elicited by tapping the tendon is tapped while the foot is dorsiflexed.
Dysarthria
MedGen UID:
8510
Concept ID:
C0013362
Mental or Behavioral Dysfunction
Dysarthric speech is a general description referring to a neurological speech disorder characterized by poor articulation. Depending on the involved neurological structures, dysarthria may be further classified as spastic, flaccid, ataxic, hyperkinetic and hypokinetic, or mixed.
Babinski sign
MedGen UID:
19708
Concept ID:
C0034935
Finding
Upturning of the big toe (and sometimes fanning of the other toes) in response to stimulation of the sole of the foot. If the Babinski sign is present it can indicate damage to the corticospinal tract.
Tremor
MedGen UID:
21635
Concept ID:
C0040822
Sign or Symptom
An unintentional, oscillating to-and-fro muscle movement about a joint axis.
Dysdiadochokinesis
MedGen UID:
115975
Concept ID:
C0234979
Sign or Symptom
A type of ataxia characterized by the impairment of the ability to perform rapidly alternating movements, such as pronating and supinating his or her hand on the dorsum of the other hand as rapidly as possible.
Hyperactive patellar reflex
MedGen UID:
66003
Concept ID:
C0240116
Finding
Intellectual disability
MedGen UID:
811461
Concept ID:
C3714756
Mental or Behavioral Dysfunction
Intellectual disability, previously referred to as mental retardation, is characterized by subnormal intellectual functioning that occurs during the developmental period. It is defined by an IQ score below 70.
Optic atrophy
MedGen UID:
18180
Concept ID:
C0029124
Disease or Syndrome
Atrophy of the optic nerve. Optic atrophy results from the death of the retinal ganglion cell axons that comprise the optic nerve and manifesting as a pale optic nerve on fundoscopy.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVOptic atrophy 2
Follow this link to review classifications for Optic atrophy 2 in Orphanet.

Professional guidelines

PubMed

Weber P, Scholl S, Baumgartner ER
Dev Med Child Neurol 2004 Jul;46(7):481-4. doi: 10.1017/s0012162204000799. PMID: 15230462

Recent clinical studies

Etiology

Kumar H, Pant H, Chaudhuri S, Kalluri V, Sarvepally VK, Govindahari V, Vashist P, Tandon R, Murthy G
Indian J Ophthalmol 2024 Jul 1;72(7):994-1000. Epub 2024 Mar 8 doi: 10.4103/IJO.IJO_2785_22. PMID: 38454844Free PMC Article
Handzic A, Xie JS, Margolin EA
J Neuroophthalmol 2024 Sep 1;44(3):365-370. Epub 2023 Aug 24 doi: 10.1097/WNO.0000000000001947. PMID: 37616072
Erdurman CF, Ceylan MO, Acikel CH, Durukan HA, Mumcuoglu T
Eur J Ophthalmol 2011 May-Jun;21(3):296-302. doi: 10.5301/EJO.2010.5732. PMID: 20872356
Macedo-Souza LI, Kok F, Santos S, Licinio L, Lezirovitz K, Cavaçana N, Bueno C, Amorim S, Pessoa A, Graciani Z, Ferreira A, Prazeres A, de Melo AN, Otto PA, Zatz M
Ann Hum Genet 2009 May;73(Pt 3):382-7. Epub 2009 Mar 4 doi: 10.1111/j.1469-1809.2009.00507.x. PMID: 19344448
Sullivan TJ, Lambert SR, Buncic JR, Musarella MA
J Pediatr Ophthalmol Strabismus 1992 Jul-Aug;29(4):246-9. doi: 10.3928/0191-3913-19920701-14. PMID: 1512668

Diagnosis

Handzic A, Xie JS, Margolin EA
J Neuroophthalmol 2024 Sep 1;44(3):365-370. Epub 2023 Aug 24 doi: 10.1097/WNO.0000000000001947. PMID: 37616072
Coşkun T, Tokatli A, Ozalp I
Turk J Pediatr 1994 Oct-Dec;36(4):267-78. PMID: 7825232

Therapy

Kumar H, Pant H, Chaudhuri S, Kalluri V, Sarvepally VK, Govindahari V, Vashist P, Tandon R, Murthy G
Indian J Ophthalmol 2024 Jul 1;72(7):994-1000. Epub 2024 Mar 8 doi: 10.4103/IJO.IJO_2785_22. PMID: 38454844Free PMC Article
Ersanli D, Sonmez M, Unal M, Gulecek O
Retina 2006 Jan;26(1):32-6. doi: 10.1097/00006982-200601000-00006. PMID: 16395136

Prognosis

Handzic A, Xie JS, Margolin EA
J Neuroophthalmol 2024 Sep 1;44(3):365-370. Epub 2023 Aug 24 doi: 10.1097/WNO.0000000000001947. PMID: 37616072
Erdurman CF, Ceylan MO, Acikel CH, Durukan HA, Mumcuoglu T
Eur J Ophthalmol 2011 May-Jun;21(3):296-302. doi: 10.5301/EJO.2010.5732. PMID: 20872356
Ersanli D, Sonmez M, Unal M, Gulecek O
Retina 2006 Jan;26(1):32-6. doi: 10.1097/00006982-200601000-00006. PMID: 16395136
Trautner C, Haastert B, Richter B, Berger M, Giani G
Invest Ophthalmol Vis Sci 2003 Mar;44(3):1031-4. doi: 10.1167/iovs.02-0304. PMID: 12601025

Clinical prediction guides

Handzic A, Xie JS, Margolin EA
J Neuroophthalmol 2024 Sep 1;44(3):365-370. Epub 2023 Aug 24 doi: 10.1097/WNO.0000000000001947. PMID: 37616072
Macedo-Souza LI, Kok F, Santos S, Licinio L, Lezirovitz K, Cavaçana N, Bueno C, Amorim S, Pessoa A, Graciani Z, Ferreira A, Prazeres A, de Melo AN, Otto PA, Zatz M
Ann Hum Genet 2009 May;73(Pt 3):382-7. Epub 2009 Mar 4 doi: 10.1111/j.1469-1809.2009.00507.x. PMID: 19344448
Macedo-Souza LI, Kok F, Santos S, Amorim SC, Starling A, Nishimura A, Lezirovitz K, Lino AM, Zatz M
Ann Neurol 2005 May;57(5):730-7. doi: 10.1002/ana.20478. PMID: 15852396

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