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IEMbase 0256: COQ8A-related Coenzyme Q8A (ADCK3) deficiency

Scope

Field Value
IEMbase ID 256
Nosology 8.1.07.01
Gene COQ8A
External IDs OMIM:612016; ORPHA:139485
Generated mapping MAPPED; Autosomal_Recessive_Ataxia_Due_to_Ubiquinone_Deficiency.yaml
Candidate DisMech targets Autosomal_Recessive_Ataxia_Due_to_Ubiquinone_Deficiency.yaml; Primary_Coenzyme_Q10_Deficiency.yaml#COQ8A
Review date 2026-07-07

IEMbase phenotype signal

IEMbase represents this as COQ8A-related coenzyme Q8A (ADCK3) deficiency, with alternate labels chaperone-activity of BC1 complex-like/CABC1 deficiency and COQ8. The record is autosomal recessive and treatability is marked unknown.

The treatment section lists Coq10 as a vitamin and trace element strategy with level 4-5 evidence from PMID 32337771 for movement-disorder involvement. Biochemical rows include abnormal CoQ10 in fibroblasts and muscle and plasma lactate. Clinical rows include cognitive dysfunction, dystonia, and pyramidal signs. Characteristic rows include ataxia, epilepsy, and muscle weakness.

DisMech phenotype coverage

Autosomal_Recessive_Ataxia_Due_to_Ubiquinone_Deficiency.yaml is the correct canonical target. The local entry covers biallelic COQ8A disease, also known as primary coenzyme Q10 deficiency-4, ARCA2, or SCAR9, as a primary CoQ10 biosynthesis disorder with mitochondrial respiratory-chain dysfunction, oxidative phosphorylation failure, oxidative stress, cerebellar injury, progressive cerebellar ataxia, cerebellar atrophy, epilepsy, cognitive impairment, developmental regression, hyperkinetic movement disorders including dystonia and myoclonus, proximal muscle weakness, elevated lactate, reduced CoQ10, and CoQ10 supplementation. The broader Primary_Coenzyme_Q10_Deficiency.yaml#COQ8A subtype points back to this standalone curation target.

Concordance and completeness

Judgement: correct dedicated-file mapping with high concordance.

IEMbase and DisMech agree on COQ8A/ADCK3 identity, autosomal recessive primary CoQ10 deficiency, low CoQ10 in patient tissues/cells, lactate elevation, ataxia, epilepsy, cognitive involvement, dystonia/movement disorder, pyramidal or multisystem neurologic involvement, muscle weakness, and CoQ10 treatment. DisMech is substantially richer for COQ8A-specific mechanism, neuroimaging, movement-disorder detail, and treatment-response context.

Curation actions

  • Keep this record mapped to Autosomal_Recessive_Ataxia_Due_to_Ubiquinone_Deficiency.yaml.
  • Retain Primary_Coenzyme_Q10_Deficiency.yaml#COQ8A as secondary umbrella context rather than the canonical mapping.
  • No mapping correction is needed.