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Combined oxidative phosphorylation deficiency 51(COXPD51)

MedGen UID:
1757992
Concept ID:
C5436703
Disease or Syndrome
Synonyms: COMBINED OXIDATIVE PHOSPHORYLATION DEFICIENCY 51; COXPD51
 
Gene (location): PTCD3 (2p11.2)
 
Monarch Initiative: MONDO:0033631
OMIM®: 619057

Definition

Combined oxidative phosphorylation deficiency-51 (COXPD51) is an autosomal recessive disorder characterized by a Leigh syndrome phenotype (see 256000). For a discussion of genetic heterogeneity of combined oxidative phosphorylation deficiency, see COXPD1 (609060). [from OMIM]

Clinical features

From HPO
Severe short stature
MedGen UID:
3931
Concept ID:
C0013336
Disease or Syndrome
A severe degree of short stature, more than -4 SD from the mean corrected for age and sex.
Fetal growth restriction
MedGen UID:
4693
Concept ID:
C0015934
Pathologic Function
An abnormal restriction of fetal growth with fetal weight below the tenth percentile for gestational age.
Small for gestational age
MedGen UID:
65920
Concept ID:
C0235991
Finding
Smaller than normal size according to sex and gestational age related norms, defined as a weight below the 10th percentile for the gestational age.
Growth delay
MedGen UID:
99124
Concept ID:
C0456070
Pathologic Function
A deficiency or slowing down of growth pre- and postnatally.
Nasogastric tube feeding
MedGen UID:
847783
Concept ID:
C3853581
Medical Device
The condition of inability to eat normally treated by placement of a thin tube through the nose into the stomach that is then used to carry food.
Hearing impairment
MedGen UID:
235586
Concept ID:
C1384666
Disease or Syndrome
A decreased magnitude of the sensory perception of sound.
Myoclonus
MedGen UID:
10234
Concept ID:
C0027066
Finding
Very brief, involuntary random muscular contractions occurring at rest, in response to sensory stimuli, or accompanying voluntary movements.
Cerebral atrophy
MedGen UID:
116012
Concept ID:
C0235946
Disease or Syndrome
Atrophy (wasting, decrease in size of cells or tissue) affecting the cerebrum.
Developmental regression
MedGen UID:
324613
Concept ID:
C1836830
Disease or Syndrome
Loss of developmental skills, as manifested by loss of developmental milestones.
Focal T2 hyperintense thalamic lesion
MedGen UID:
868380
Concept ID:
C4022774
Finding
A lighter than expected T2 signal on magnetic resonance imaging (MRI) of the thalamus. This term refers to a localized hyperintensity affecting a particular region of the thalamus.
Rigidity
MedGen UID:
7752
Concept ID:
C0026837
Sign or Symptom
Continuous involuntary sustained muscle contraction. When an affected muscle is passively stretched, the degree of resistance remains constant regardless of the rate at which the muscle is stretched. This feature helps to distinguish rigidity from muscle spasticity.
Respiratory failure
MedGen UID:
257837
Concept ID:
C1145670
Disease or Syndrome
A severe form of respiratory insufficiency characterized by inadequate gas exchange such that the levels of oxygen or carbon dioxide cannot be maintained within normal limits.
Neonatal respiratory distress
MedGen UID:
924182
Concept ID:
C4281993
Finding
Respiratory difficulty as newborn.
Aspiration pneumonia
MedGen UID:
10814
Concept ID:
C0032290
Disease or Syndrome
Pneumonia due to the aspiration (breathing in) of food, liquid, or gastric contents into the upper respiratory tract.
Caesarian section
MedGen UID:
863
Concept ID:
C0007876
Therapeutic or Preventive Procedure
Delivery of a fetus through surgical incisions made through the abdominal wall (laparotomy) and the uterine wall (hysterotomy).
Nystagmus
MedGen UID:
45166
Concept ID:
C0028738
Disease or Syndrome
Rhythmic, involuntary oscillations of one or both eyes related to abnormality in fixation, conjugate gaze, or vestibular mechanisms.
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.
Decreased activity of mitochondrial complex I
MedGen UID:
393796
Concept ID:
C2677650
Finding
A reduction in the activity of the mitochondrial respiratory chain complex I, which is part of the electron transport chain in mitochondria.

Professional guidelines

PubMed

Trinh J, Imhoff S, Dulovic-Mahlow M, Kandaswamy KK, Tadic V, Schäfer J, Dobricic V, Nolte A, Werber M, Rolfs A, Münchau A, Klein C, Lohmann K, Brüggemann N
J Neurol 2020 Mar;267(3):770-782. Epub 2019 Nov 20 doi: 10.1007/s00415-019-09640-2. PMID: 31745726
Roe CR, Brunengraber H
Mol Genet Metab 2015 Dec;116(4):260-8. Epub 2015 Oct 24 doi: 10.1016/j.ymgme.2015.10.005. PMID: 26547562Free PMC Article
Bricout M, Grévent D, Lebre AS, Rio M, Desguerre I, De Lonlay P, Valayannopoulos V, Brunelle F, Rötig A, Munnich A, Boddaert N
J Med Genet 2014 Jul;51(7):429-35. Epub 2014 May 1 doi: 10.1136/jmedgenet-2013-102256. PMID: 24793058

Recent clinical studies

Etiology

Dobrewa W, Bielska M, Bąbol-Pokora K, Janczar S, Młynarski W
Mutat Res Rev Mutat Res 2024 Jan-Jun;793:108476. Epub 2023 Nov 19 doi: 10.1016/j.mrrev.2023.108476. PMID: 37989463
Tein I
Epilepsy Behav 2023 Aug;145:109338. Epub 2023 Jul 13 doi: 10.1016/j.yebeh.2023.109338. PMID: 37453291
Kohsaka S, Hirata M, Ikegami M, Ueno T, Kojima S, Sakai T, Ito K, Naka N, Ogura K, Kawai A, Iwata S, Okuma T, Yonemoto T, Kobayashi H, Suehara Y, Hiraga H, Kawamoto T, Motoi T, Oda Y, Matsubara D, Matsuda K, Nishida Y, Mano H
Eur J Cancer 2021 Mar;145:109-120. Epub 2021 Jan 11 doi: 10.1016/j.ejca.2020.12.001. PMID: 33444924
Bricout M, Grévent D, Lebre AS, Rio M, Desguerre I, De Lonlay P, Valayannopoulos V, Brunelle F, Rötig A, Munnich A, Boddaert N
J Med Genet 2014 Jul;51(7):429-35. Epub 2014 May 1 doi: 10.1136/jmedgenet-2013-102256. PMID: 24793058
Voet NB, van der Kooi EL, Riphagen II, Lindeman E, van Engelen BG, Geurts AC
Cochrane Database Syst Rev 2013 Jul 9;(7):CD003907. doi: 10.1002/14651858.CD003907.pub4. PMID: 23835682

Diagnosis

Tein I
Epilepsy Behav 2023 Aug;145:109338. Epub 2023 Jul 13 doi: 10.1016/j.yebeh.2023.109338. PMID: 37453291
Sun J, Brown TT, Tong W, Samuels D, Tien P, Aissani B, Aouizerat B, Villacres M, Kuniholm MH, Gustafson D, Michel K, Cohen M, Schneider M, Adimora AA, Ali MK, Bolivar H, Hulgan T
Clin Infect Dis 2020 Nov 5;71(8):e218-e225. doi: 10.1093/cid/ciaa026. PMID: 31927570Free PMC Article
van der Meulen PM, Barendregt AM, Cuadrado E, Magro-Checa C, Steup-Beekman GM, Schonenberg-Meinema D, Van den Berg JM, Li QZ, Baars PA, Wouters D, Voskuyl AE, Ten Berge IRJM, Huizinga TWJ, Kuijpers TW
Rheumatology (Oxford) 2017 Aug 1;56(8):1407-1416. doi: 10.1093/rheumatology/kex073. PMID: 28460084
Bricout M, Grévent D, Lebre AS, Rio M, Desguerre I, De Lonlay P, Valayannopoulos V, Brunelle F, Rötig A, Munnich A, Boddaert N
J Med Genet 2014 Jul;51(7):429-35. Epub 2014 May 1 doi: 10.1136/jmedgenet-2013-102256. PMID: 24793058
Voet NB, van der Kooi EL, Riphagen II, Lindeman E, van Engelen BG, Geurts AC
Cochrane Database Syst Rev 2013 Jul 9;(7):CD003907. doi: 10.1002/14651858.CD003907.pub4. PMID: 23835682

Therapy

Tein I
Epilepsy Behav 2023 Aug;145:109338. Epub 2023 Jul 13 doi: 10.1016/j.yebeh.2023.109338. PMID: 37453291
Shrestha R, Johnson E, Byrne FL
Mol Metab 2021 Sep;51:101222. Epub 2021 Mar 26 doi: 10.1016/j.molmet.2021.101222. PMID: 33781939Free PMC Article
Staikou C, Stamelos M, Stavroulakis E
Anaesthesiol Intensive Ther 2019;51(2):133-146. doi: 10.5114/ait.2019.86278. PMID: 31268275
Voet NB, van der Kooi EL, Riphagen II, Lindeman E, van Engelen BG, Geurts AC
Cochrane Database Syst Rev 2013 Jul 9;(7):CD003907. doi: 10.1002/14651858.CD003907.pub4. PMID: 23835682
Bartlett K, Ghneim HK, Stirk HJ, Wastell H
J Inherit Metab Dis 1985;8 Suppl 1:46-52. doi: 10.1007/BF01800659. PMID: 2864473

Prognosis

Zhang X, Xiang F, Li D, Yang F, Yu S, Wang X
BMC Neurol 2024 Jan 2;24(1):15. doi: 10.1186/s12883-023-03480-4. PMID: 38166857Free PMC Article
Dobrewa W, Bielska M, Bąbol-Pokora K, Janczar S, Młynarski W
Mutat Res Rev Mutat Res 2024 Jan-Jun;793:108476. Epub 2023 Nov 19 doi: 10.1016/j.mrrev.2023.108476. PMID: 37989463
Kohsaka S, Hirata M, Ikegami M, Ueno T, Kojima S, Sakai T, Ito K, Naka N, Ogura K, Kawai A, Iwata S, Okuma T, Yonemoto T, Kobayashi H, Suehara Y, Hiraga H, Kawamoto T, Motoi T, Oda Y, Matsubara D, Matsuda K, Nishida Y, Mano H
Eur J Cancer 2021 Mar;145:109-120. Epub 2021 Jan 11 doi: 10.1016/j.ejca.2020.12.001. PMID: 33444924
Trinh J, Imhoff S, Dulovic-Mahlow M, Kandaswamy KK, Tadic V, Schäfer J, Dobricic V, Nolte A, Werber M, Rolfs A, Münchau A, Klein C, Lohmann K, Brüggemann N
J Neurol 2020 Mar;267(3):770-782. Epub 2019 Nov 20 doi: 10.1007/s00415-019-09640-2. PMID: 31745726
Jeong HO, Park D, Im E, Lee J, Im DS, Chung HY
J Frailty Aging 2017;6(2):97-102. doi: 10.14283/jfa.2017.13. PMID: 28555711

Clinical prediction guides

Kunová N, Ondrovičová G, Bauer JA, Krajčovičová V, Pinkas M, Stojkovičová B, Havalová H, Lukáčová V, Kohútová L, Košťan J, Martináková L, Baráth P, Nováček J, Zoll S, Kereϊche S, Kutejová E, Pevala V
Sci Rep 2024 Apr 30;14(1):9923. doi: 10.1038/s41598-024-60030-9. PMID: 38688959Free PMC Article
Dobrewa W, Bielska M, Bąbol-Pokora K, Janczar S, Młynarski W
Mutat Res Rev Mutat Res 2024 Jan-Jun;793:108476. Epub 2023 Nov 19 doi: 10.1016/j.mrrev.2023.108476. PMID: 37989463
Kohsaka S, Hirata M, Ikegami M, Ueno T, Kojima S, Sakai T, Ito K, Naka N, Ogura K, Kawai A, Iwata S, Okuma T, Yonemoto T, Kobayashi H, Suehara Y, Hiraga H, Kawamoto T, Motoi T, Oda Y, Matsubara D, Matsuda K, Nishida Y, Mano H
Eur J Cancer 2021 Mar;145:109-120. Epub 2021 Jan 11 doi: 10.1016/j.ejca.2020.12.001. PMID: 33444924
Jeong HO, Park D, Im E, Lee J, Im DS, Chung HY
J Frailty Aging 2017;6(2):97-102. doi: 10.14283/jfa.2017.13. PMID: 28555711
Bricout M, Grévent D, Lebre AS, Rio M, Desguerre I, De Lonlay P, Valayannopoulos V, Brunelle F, Rötig A, Munnich A, Boddaert N
J Med Genet 2014 Jul;51(7):429-35. Epub 2014 May 1 doi: 10.1136/jmedgenet-2013-102256. PMID: 24793058

Recent systematic reviews

Voet NB, van der Kooi EL, Riphagen II, Lindeman E, van Engelen BG, Geurts AC
Cochrane Database Syst Rev 2013 Jul 9;(7):CD003907. doi: 10.1002/14651858.CD003907.pub4. PMID: 23835682

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