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Polycystic liver disease 2(PCLD2)

MedGen UID:
934736
Concept ID:
C4310769
Disease or Syndrome
Synonyms: PCLD2; POLYCYSTIC LIVER DISEASE 2 WITH OR WITHOUT KIDNEY CYSTS
 
Gene (location): SEC63 (6q21)
 
Monarch Initiative: MONDO:0014860
OMIM®: 617004

Definition

PCLD2 is an autosomal dominant disease characterized by the presence of multiple liver cysts resulting from structural changes in the biliary tree during development. Abnormal biliary structures may be present early in life, but they usually remain asymptomatic until cyst growth initiates during adulthood. In advanced stages, patients may develop massively enlarged livers, which cause a spectrum of clinical features and complications. Genetic studies suggest that an accumulation of somatic hits in cyst epithelium determines the rate of cyst formation. A subset of patients (28-35%) may develop kidney cysts that are usually incidental findings and do not result in clinically significant renal disease (review by Cnossen and Drenth, 2014). For a discussion of genetic heterogeneity of polycystic liver disease, see PCLD1 (174050). [from OMIM]

Clinical features

From HPO
Hepatomegaly
MedGen UID:
42428
Concept ID:
C0019209
Finding
Abnormally increased size of the liver.
Hepatic cysts
MedGen UID:
82761
Concept ID:
C0267834
Disease or Syndrome
A cystic lesion located in the liver.

Professional guidelines

PubMed

Schönauer R, Sierks D, Boerrigter M, Jawaid T, Caroff L, Audrezet MP, Friedrich A, Shaw M, Degenhardt J, Forberger M, de Fallois J, Bläker H, Bergmann C, Gödiker J, Schindler P, Schlevogt B, Müller RU, Berg T, Patterson I, Griffiths WJ, Sayer JA; Genomics England Research Consortium, Popp B, Torres VE, Hogan MC, Somlo S, Watnick TJ, Nevens F, Besse W, Cornec-Le Gall E, Harris PC, Drenth JPH, Halbritter J
Gastroenterology 2024 May;166(5):902-914. Epub 2023 Dec 13 doi: 10.1053/j.gastro.2023.12.007. PMID: 38101549
van Aerts RMM, van de Laarschot LFM, Banales JM, Drenth JPH
J Hepatol 2018 Apr;68(4):827-837. Epub 2017 Nov 24 doi: 10.1016/j.jhep.2017.11.024. PMID: 29175241
Gevers TJ, Drenth JP
Nat Rev Gastroenterol Hepatol 2013 Feb;10(2):101-8. Epub 2013 Jan 8 doi: 10.1038/nrgastro.2012.254. PMID: 23296249

Recent clinical studies

Etiology

Yang H, Sieben CJ, Schauer RS, Harris PC
Adv Kidney Dis Health 2023 Sep;30(5):397-406. doi: 10.1053/j.akdh.2023.04.004. PMID: 38097330Free PMC Article
Waddell SH, Yao Y, Olaizola P, Walker A, Jarman EJ, Gournopanos K, Gradinaru A, Christodoulou E, Gautier P, Boerrigter MM, Cadamuro M, Fabris L, Drenth JP, Kendall TJ, Banales JM, Khamseh A, Mill P, Boulter L
Sci Transl Med 2023 Sep 13;15(713):eabq5930. doi: 10.1126/scitranslmed.abq5930. PMID: 37703354Free PMC Article
Lee-Law PY, van de Laarschot LFM, Banales JM, Drenth JPH
Curr Opin Gastroenterol 2019 Mar;35(2):65-72. doi: 10.1097/MOG.0000000000000514. PMID: 30652979
Cornec-Le Gall E, Torres VE, Harris PC
J Am Soc Nephrol 2018 Jan;29(1):13-23. Epub 2017 Oct 16 doi: 10.1681/ASN.2017050483. PMID: 29038287Free PMC Article
Leung JC
Curr Pediatr Rev 2014;10(2):95-100. doi: 10.2174/157339631002140513101755. PMID: 25088262

Diagnosis

van Aerts RMM, van de Laarschot LFM, Banales JM, Drenth JPH
J Hepatol 2018 Apr;68(4):827-837. Epub 2017 Nov 24 doi: 10.1016/j.jhep.2017.11.024. PMID: 29175241
Cornec-Le Gall E, Torres VE, Harris PC
J Am Soc Nephrol 2018 Jan;29(1):13-23. Epub 2017 Oct 16 doi: 10.1681/ASN.2017050483. PMID: 29038287Free PMC Article
Gevers TJ, Drenth JP
Nat Rev Gastroenterol Hepatol 2013 Feb;10(2):101-8. Epub 2013 Jan 8 doi: 10.1038/nrgastro.2012.254. PMID: 23296249
Qian Q
Adv Chronic Kidney Dis 2010 Mar;17(2):181-9. doi: 10.1053/j.ackd.2009.12.005. PMID: 20219621Free PMC Article
Everson GT, Taylor MR, Doctor RB
Hepatology 2004 Oct;40(4):774-82. doi: 10.1002/hep.20431. PMID: 15382167

Therapy

Capuano I, Buonanno P, Riccio E, Rizzo M, Pisani A
Clin Nephrol 2022 Mar;97(3):131-140. doi: 10.5414/CN110510. PMID: 34846296
Li AY, Bergquist JR, August AT, Dua MM, Poultsides GA, Visser BC
HPB (Oxford) 2021 Jan;23(1):56-62. Epub 2020 May 22 doi: 10.1016/j.hpb.2020.04.010. PMID: 32451237
Takita M, Iwanishi M, Minami T, Kono M, Chishina H, Arizumi T, Yada N, Hagiwara S, Minami Y, Ida H, Ueshima K, Naoshi N, Kudo M
Dig Dis 2016;34(6):654-658. Epub 2016 Oct 17 doi: 10.1159/000448826. PMID: 27750233
Khan S, Dennison A, Garcea G
Ann R Coll Surg Engl 2016 Jan;98(1):18-23. doi: 10.1308/rcsann.2016.0023. PMID: 26688394Free PMC Article
Sun L, Yu CY, Mackey LV, Coy DH
Curr Top Med Chem 2015;16(2):133-40. doi: 10.2174/1568026615666150701115157. PMID: 26126910

Prognosis

Mahboobipour AA, Ala M, Safdari Lord J, Yaghoobi A
Orphanet J Rare Dis 2024 Apr 26;19(1):175. doi: 10.1186/s13023-024-03187-w. PMID: 38671465Free PMC Article
Roediger R, Dieterich D, Chanumolu P, Deshpande P
Clin Liver Dis 2022 May;26(2):229-243. Epub 2022 Apr 1 doi: 10.1016/j.cld.2022.01.009. PMID: 35487607
Durand F
Curr Opin Organ Transplant 2020 Apr;25(2):132-138. doi: 10.1097/MOT.0000000000000749. PMID: 32073486
Cornec-Le Gall E, Torres VE, Harris PC
J Am Soc Nephrol 2018 Jan;29(1):13-23. Epub 2017 Oct 16 doi: 10.1681/ASN.2017050483. PMID: 29038287Free PMC Article
Takita M, Iwanishi M, Minami T, Kono M, Chishina H, Arizumi T, Yada N, Hagiwara S, Minami Y, Ida H, Ueshima K, Naoshi N, Kudo M
Dig Dis 2016;34(6):654-658. Epub 2016 Oct 17 doi: 10.1159/000448826. PMID: 27750233

Clinical prediction guides

Mahboobipour AA, Ala M, Safdari Lord J, Yaghoobi A
Orphanet J Rare Dis 2024 Apr 26;19(1):175. doi: 10.1186/s13023-024-03187-w. PMID: 38671465Free PMC Article
Alsager M, Neong SF, Gandhi R, Teriaky A, Tang E, Skaro A, Qumosani K, Lilly L, Galvin Z, Selzner N, Bhat MP, Puka K, Brahmania M
BMC Gastroenterol 2021 Mar 9;21(1):115. doi: 10.1186/s12876-021-01703-x. PMID: 33750299Free PMC Article
Durand F
Curr Opin Organ Transplant 2020 Apr;25(2):132-138. doi: 10.1097/MOT.0000000000000749. PMID: 32073486
Khan S, Dennison A, Garcea G
Ann R Coll Surg Engl 2016 Jan;98(1):18-23. doi: 10.1308/rcsann.2016.0023. PMID: 26688394Free PMC Article
Masyuk TV, Masyuk AI, LaRusso NF
Dig Dis 2015;33(3):420-5. Epub 2015 May 27 doi: 10.1159/000371696. PMID: 26045278Free PMC Article

Recent systematic reviews

Suwabe T, Barrera FJ, Rodriguez-Gutierrez R, Ubara Y, Hogan MC
PLoS One 2021;16(9):e0257606. Epub 2021 Sep 24 doi: 10.1371/journal.pone.0257606. PMID: 34559824Free PMC Article
Natale P, Hannan E, Sautenet B, Ju A, Perrone RD, Burnette E, Casteleijn N, Chapman A, Eastty S, Gansevoort R, Hogan M, Horie S, Knebelmann B, Lee R, Mustafa RA, Sandford R, Baumgart A, Tong A, Strippoli GFM, Craig JC, Rangan GK, Cho Y
PLoS One 2021;16(5):e0252479. Epub 2021 May 27 doi: 10.1371/journal.pone.0252479. PMID: 34043715Free PMC Article
Griffiths J, Mills MT, Ong AC
BMJ Open 2020 Jan 9;10(1):e032620. doi: 10.1136/bmjopen-2019-032620. PMID: 31924636Free PMC Article
Wijnands TF, Görtjes AP, Gevers TJ, Jenniskens SF, Kool LJ, Potthoff A, Ronot M, Drenth JP
AJR Am J Roentgenol 2017 Jan;208(1):201-207. Epub 2016 Nov 8 doi: 10.2214/AJR.16.16130. PMID: 27824501
Leombroni M, Buca D, Celentano C, Liberati M, Bascietto F, Gustapane S, Marrone L, Manzoli L, Rizzo G, D'Antonio F
Ultrasound Obstet Gynecol 2017 Aug;50(2):167-174. Epub 2017 Jul 9 doi: 10.1002/uog.17244. PMID: 27553859

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