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Hypercholanemia, familial, 2(FHCA2)

MedGen UID:
1780531
Concept ID:
C5543243
Disease or Syndrome
Synonym: NTCP deficiency
 
Gene (location): SLC10A1 (14q24.1)
 
Monarch Initiative: MONDO:0031003
OMIM®: 619256

Definition

Familial hypercholanemia-2 (FHCA2) is an autosomal recessive inborn error of metabolism characterized by persistently increased plasma levels of conjugated bile salts apparent from infancy. Most patients are asymptomatic and have no liver dysfunction, although some neonates may have transient jaundice or transiently elevated liver enzymes. These abnormalities improve with age. The bile acid defect can result in impaired absorption of fat-soluble vitamins, including D and K, causing decreased bone mineral density or prolonged prothrobin time (PT) (summary by Deng et al., 2016 and Liu et al., 2017). For a discussion of genetic heterogeneity of FHCA, see FHCA1 (607748). [from OMIM]

Clinical features

From HPO
Prolonged neonatal jaundice
MedGen UID:
347108
Concept ID:
C1859236
Finding
Neonatal jaundice refers to a yellowing of the skin and other tissues of a newborn infant as a result of increased concentrations of bilirubin in the blood. Neonatal jaundice affects over half of all newborns to some extent in the first week of life. Prolonged neonatal jaundice is said to be present if the jaundice persists for longer than 14 days in term infants and 21 days in preterm infants.
Osteopenia
MedGen UID:
18222
Concept ID:
C0029453
Disease or Syndrome
Osteopenia is a term to define bone density that is not normal but also not as low as osteoporosis. By definition from the World Health Organization osteopenia is defined by bone densitometry as a T score -1 to -2.5.
Decreased circulating vitamin D concentration
MedGen UID:
12114
Concept ID:
C0042870
Disease or Syndrome
The concentration of vitamin D in the blood circulation is below the lower limit of normal.
Unconjugated hyperbilirubinemia
MedGen UID:
82786
Concept ID:
C0268306
Disease or Syndrome
An increased amount of unconjugated (indirect) bilurubin in the blood.
Increased serum bile acid concentration
MedGen UID:
868605
Concept ID:
C4023004
Finding
An increase in the concentration of bile acid in the blood.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  

Professional guidelines

PubMed

Benedek P, Eriksson M, Duvefelt K, Freyschuss A, Frick M, Lundman P, Nylund L, Szummer K
J Intern Med 2018 Dec;284(6):674-684. Epub 2018 Jul 29 doi: 10.1111/joim.12812. PMID: 29974534

Recent clinical studies

Etiology

He Y, Zhang X, Shao Y, Xu B, Cui Y, Chen X, Chen H, Luo C, Ding M
Biochim Biophys Acta Mol Basis Dis 2022 Jan 1;1868(1):166269. Epub 2021 Sep 17 doi: 10.1016/j.bbadis.2021.166269. PMID: 34537368
Fathala AL, Alfaer F, Aldurabi A, Shoukri M, Alsergani H
Ann Saudi Med 2020 Mar-Apr;40(2):81-86. Epub 2020 Apr 2 doi: 10.5144/0256-4947.2020.81. PMID: 32241166Free PMC Article
Escate R, Mata P, Cepeda JM, Padró T, Badimon L
FASEB J 2018 Feb;32(2):601-612. doi: 10.1096/fj.201700476RR. PMID: 29457550
Gaudet D, Kereiakes DJ, McKenney JM, Roth EM, Hanotin C, Gipe D, Du Y, Ferrand AC, Ginsberg HN, Stein EA
Am J Cardiol 2014 Sep 1;114(5):711-5. Epub 2014 Jun 18 doi: 10.1016/j.amjcard.2014.05.060. PMID: 25060413
van Zyl T, Jerling JC, Conradie KR, Feskens EJ
J Hum Genet 2014 Feb;59(2):88-94. Epub 2013 Nov 28 doi: 10.1038/jhg.2013.123. PMID: 24284361

Diagnosis

He Y, Zhang X, Shao Y, Xu B, Cui Y, Chen X, Chen H, Luo C, Ding M
Biochim Biophys Acta Mol Basis Dis 2022 Jan 1;1868(1):166269. Epub 2021 Sep 17 doi: 10.1016/j.bbadis.2021.166269. PMID: 34537368
Fathala AL, Alfaer F, Aldurabi A, Shoukri M, Alsergani H
Ann Saudi Med 2020 Mar-Apr;40(2):81-86. Epub 2020 Apr 2 doi: 10.5144/0256-4947.2020.81. PMID: 32241166Free PMC Article

Therapy

Gaudet D, Kereiakes DJ, McKenney JM, Roth EM, Hanotin C, Gipe D, Du Y, Ferrand AC, Ginsberg HN, Stein EA
Am J Cardiol 2014 Sep 1;114(5):711-5. Epub 2014 Jun 18 doi: 10.1016/j.amjcard.2014.05.060. PMID: 25060413
van Zyl T, Jerling JC, Conradie KR, Feskens EJ
J Hum Genet 2014 Feb;59(2):88-94. Epub 2013 Nov 28 doi: 10.1038/jhg.2013.123. PMID: 24284361

Prognosis

Fathala AL, Alfaer F, Aldurabi A, Shoukri M, Alsergani H
Ann Saudi Med 2020 Mar-Apr;40(2):81-86. Epub 2020 Apr 2 doi: 10.5144/0256-4947.2020.81. PMID: 32241166Free PMC Article
Gaudet D, Kereiakes DJ, McKenney JM, Roth EM, Hanotin C, Gipe D, Du Y, Ferrand AC, Ginsberg HN, Stein EA
Am J Cardiol 2014 Sep 1;114(5):711-5. Epub 2014 Jun 18 doi: 10.1016/j.amjcard.2014.05.060. PMID: 25060413

Clinical prediction guides

Fathala AL, Alfaer F, Aldurabi A, Shoukri M, Alsergani H
Ann Saudi Med 2020 Mar-Apr;40(2):81-86. Epub 2020 Apr 2 doi: 10.5144/0256-4947.2020.81. PMID: 32241166Free PMC Article
Benedek P, Eriksson M, Duvefelt K, Freyschuss A, Frick M, Lundman P, Nylund L, Szummer K
J Intern Med 2018 Dec;284(6):674-684. Epub 2018 Jul 29 doi: 10.1111/joim.12812. PMID: 29974534

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