U.S. flag

An official website of the United States government

Format

Send to:

Choose Destination

Hereditary hemorrhagic telangiectasia(HHT)

MedGen UID:
52657
Concept ID:
C0039445
Disease or Syndrome
Synonyms: HHT; ORW disease; Osler hemorrhagic telangiectasia syndrome; Osler Weber Rendu syndrome; Osler-Rendu-Weber disease
SNOMED CT: Osler hemorrhagic telangiectasia syndrome (21877004); Osler-Weber-Rendu disease (21877004); Hereditary hemorrhagic telangiectasia (21877004); Osler-Rendu-Weber syndrome (21877004); Osler-Rendu-Weber disease (21877004); HHT - Hereditary hemorrhagic telangiectasia (21877004)
Modes of inheritance:
Autosomal dominant inheritance
MedGen UID:
141047
Concept ID:
C0443147
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in heterozygotes. In the context of medical genetics, an autosomal dominant disorder is caused when a single copy of the mutant allele is present. Males and females are affected equally, and can both transmit the disorder with a risk of 50% for each child of inheriting the mutant allele.
 
Related genes: SMAD4, GDF2, ENG, ACVRL1
 
Monarch Initiative: MONDO:0019180
OMIM®: 187300
OMIM® Phenotypic series: PS187300
Orphanet: ORPHA774

Definition

Hereditary hemorrhagic telangiectasia (HHT) is an autosomal dominant vascular dysplasia leading to telangiectases and arteriovenous malformations of skin, mucosa, and viscera. Epistaxis and gastrointestinal bleeding are frequent complications of mucosal involvement. Visceral involvement includes that of the lung, liver, and brain. The most frequent form of hereditary hemorrhagic telangiectasia maps to the long arm of chromosome 9. Genetic Heterogeneity of Hereditary Hemorrhagic Telangiectasia See also HHT2 (600376), caused by mutation in the ALK1 gene (ACVRL1; 601284) on chromosome 12q13; HHT3 (601101), mapped to chromosome 5q31; HHT4 (610655), mapped to chromosome 7p14; and HHT5 (615506), caused by mutation in the GDF2 gene (605120) on chromosome 10q11. See also juvenile polyposis/HHT syndrome (175050), caused by mutation in the SMAD4 gene (600993). [from OMIM]

Professional guidelines

PubMed

Faughnan ME, Mager JJ, Hetts SW, Palda VA, Lang-Robertson K, Buscarini E, Deslandres E, Kasthuri RS, Lausman A, Poetker D, Ratjen F, Chesnutt MS, Clancy M, Whitehead KJ, Al-Samkari H, Chakinala M, Conrad M, Cortes D, Crocione C, Darling J, de Gussem E, Derksen C, Dupuis-Girod S, Foy P, Geisthoff U, Gossage JR, Hammill A, Heimdal K, Henderson K, Iyer VN, Kjeldsen AD, Komiyama M, Korenblatt K, McDonald J, McMahon J, McWilliams J, Meek ME, Mei-Zahav M, Olitsky S, Palmer S, Pantalone R, Piccirillo JF, Plahn B, Porteous MEM, Post MC, Radovanovic I, Rochon PJ, Rodriguez-Lopez J, Sabba C, Serra M, Shovlin C, Sprecher D, White AJ, Winship I, Zarrabeitia R
Ann Intern Med 2020 Dec 15;173(12):989-1001. Epub 2020 Sep 8 doi: 10.7326/M20-1443. PMID: 32894695
Tunkel DE, Anne S, Payne SC, Ishman SL, Rosenfeld RM, Abramson PJ, Alikhaani JD, Benoit MM, Bercovitz RS, Brown MD, Chernobilsky B, Feldstein DA, Hackell JM, Holbrook EH, Holdsworth SM, Lin KW, Lind MM, Poetker DM, Riley CA, Schneider JS, Seidman MD, Vadlamudi V, Valdez TA, Nnacheta LC, Monjur TM
Otolaryngol Head Neck Surg 2020 Jan;162(1_suppl):S1-S38. doi: 10.1177/0194599819890327. PMID: 31910111
Kritharis A, Al-Samkari H, Kuter DJ
Haematologica 2018 Sep;103(9):1433-1443. Epub 2018 May 24 doi: 10.3324/haematol.2018.193003. PMID: 29794143Free PMC Article

Recent clinical studies

Etiology

Al-Samkari H, Eng W
J Thromb Haemost 2022 May;20(5):1077-1088. Epub 2022 Apr 7 doi: 10.1111/jth.15715. PMID: 35343049Free PMC Article
Bofarid S, Hosman AE, Mager JJ, Snijder RJ, Post MC
Int J Mol Sci 2021 Mar 27;22(7) doi: 10.3390/ijms22073471. PMID: 33801690Free PMC Article
Raevens S, Boret M, De Pauw M, Fallon MB, Van Vlierberghe H
Hepatology 2021 Sep;74(3):1674-1686. Epub 2021 May 24 doi: 10.1002/hep.31770. PMID: 33636019
Shovlin CL, Simeoni I, Downes K, Frazer ZC, Megy K, Bernabeu-Herrero ME, Shurr A, Brimley J, Patel D, Kell L, Stephens J, Turbin IG, Aldred MA, Penkett CJ, Ouwehand WH, Jovine L, Turro E
Blood 2020 Oct 22;136(17):1907-1918. doi: 10.1182/blood.2019004560. PMID: 32573726Free PMC Article
Tellapuri S, Park HS, Kalva SP
Int J Cardiovasc Imaging 2019 Aug;35(8):1421-1428. Epub 2018 Nov 1 doi: 10.1007/s10554-018-1479-x. PMID: 30386957

Diagnosis

Al-Samkari H, Eng W
J Thromb Haemost 2022 May;20(5):1077-1088. Epub 2022 Apr 7 doi: 10.1111/jth.15715. PMID: 35343049Free PMC Article
Hammill AM, Wusik K, Kasthuri RS
Hematology Am Soc Hematol Educ Program 2021 Dec 10;2021(1):469-477. doi: 10.1182/hematology.2021000281. PMID: 34889398Free PMC Article
Al-Samkari H
Blood 2021 Feb 18;137(7):888-895. doi: 10.1182/blood.2020008739. PMID: 33171488
Tellapuri S, Park HS, Kalva SP
Int J Cardiovasc Imaging 2019 Aug;35(8):1421-1428. Epub 2018 Nov 1 doi: 10.1007/s10554-018-1479-x. PMID: 30386957
Kritharis A, Al-Samkari H, Kuter DJ
Haematologica 2018 Sep;103(9):1433-1443. Epub 2018 May 24 doi: 10.3324/haematol.2018.193003. PMID: 29794143Free PMC Article

Therapy

Al-Samkari H
Blood 2021 Feb 18;137(7):888-895. doi: 10.1182/blood.2020008739. PMID: 33171488
Robert F, Desroches-Castan A, Bailly S, Dupuis-Girod S, Feige JJ
Orphanet J Rare Dis 2020 Jan 7;15(1):4. doi: 10.1186/s13023-019-1281-4. PMID: 31910860Free PMC Article
Tellapuri S, Park HS, Kalva SP
Int J Cardiovasc Imaging 2019 Aug;35(8):1421-1428. Epub 2018 Nov 1 doi: 10.1007/s10554-018-1479-x. PMID: 30386957
Kritharis A, Al-Samkari H, Kuter DJ
Haematologica 2018 Sep;103(9):1433-1443. Epub 2018 May 24 doi: 10.3324/haematol.2018.193003. PMID: 29794143Free PMC Article
Auerbach M, Adamson JW
Am J Hematol 2016 Jan;91(1):31-8. Epub 2015 Nov 17 doi: 10.1002/ajh.24201. PMID: 26408108

Prognosis

Hammill AM, Wusik K, Kasthuri RS
Hematology Am Soc Hematol Educ Program 2021 Dec 10;2021(1):469-477. doi: 10.1182/hematology.2021000281. PMID: 34889398Free PMC Article
Bofarid S, Hosman AE, Mager JJ, Snijder RJ, Post MC
Int J Mol Sci 2021 Mar 27;22(7) doi: 10.3390/ijms22073471. PMID: 33801690Free PMC Article
Raevens S, Boret M, De Pauw M, Fallon MB, Van Vlierberghe H
Hepatology 2021 Sep;74(3):1674-1686. Epub 2021 May 24 doi: 10.1002/hep.31770. PMID: 33636019
Shovlin CL, Simeoni I, Downes K, Frazer ZC, Megy K, Bernabeu-Herrero ME, Shurr A, Brimley J, Patel D, Kell L, Stephens J, Turbin IG, Aldred MA, Penkett CJ, Ouwehand WH, Jovine L, Turro E
Blood 2020 Oct 22;136(17):1907-1918. doi: 10.1182/blood.2019004560. PMID: 32573726Free PMC Article
Rimmer J, Lund VJ
Rhinology 2015 Sep;53(3):195-203. doi: 10.4193/Rhino14.274. PMID: 26460394

Clinical prediction guides

Hyldahl SJ, El-Jaji MQ, Schuster A, Kjeldsen AD
Int J Dermatol 2022 Dec;61(12):1497-1505. Epub 2022 Jul 6 doi: 10.1111/ijd.16320. PMID: 35792874Free PMC Article
Topiwala KK, Patel SD, Saver JL, Streib CD, Shovlin CL
Neurology 2022 Feb 1;98(5):188-198. Epub 2021 Dec 8 doi: 10.1212/WNL.0000000000013169. PMID: 34880092Free PMC Article
Al-Samkari H, Kasthuri RS, Parambil JG, Albitar HA, Almodallal YA, Vázquez C, Serra MM, Dupuis-Girod S, Wilsen CB, McWilliams JP, Fountain EH, Gossage JR, Weiss CR, Latif MA, Issachar A, Mei-Zahav M, Meek ME, Conrad M, Rodriguez-Lopez J, Kuter DJ, Iyer VN
Haematologica 2021 Aug 1;106(8):2161-2169. doi: 10.3324/haematol.2020.261859. PMID: 32675221Free PMC Article
Shovlin CL, Simeoni I, Downes K, Frazer ZC, Megy K, Bernabeu-Herrero ME, Shurr A, Brimley J, Patel D, Kell L, Stephens J, Turbin IG, Aldred MA, Penkett CJ, Ouwehand WH, Jovine L, Turro E
Blood 2020 Oct 22;136(17):1907-1918. doi: 10.1182/blood.2019004560. PMID: 32573726Free PMC Article
Dupuis O, Delagrange L, Dupuis-Girod S
Orphanet J Rare Dis 2020 Jan 7;15(1):5. doi: 10.1186/s13023-019-1286-z. PMID: 31910869Free PMC Article

Recent systematic reviews

Thiele B, Abdel-Aty Y, Marks L, Lal D, Marino M
Ann Otol Rhinol Laryngol 2023 Jan;132(1):82-90. Epub 2022 Feb 12 doi: 10.1177/00034894221078075. PMID: 35152768
Albarki H, Rimmer J
Am J Rhinol Allergy 2022 Nov;36(6):890-896. Epub 2022 Aug 4 doi: 10.1177/19458924221118131. PMID: 35929049
Halderman AA, Ryan MW, Marple BF, Sindwani R, Reh DD, Poetker DM
Am J Rhinol Allergy 2018 Jul;32(4):258-268. Epub 2018 May 10 doi: 10.1177/1945892418768588. PMID: 29745243
Felli E, Addeo P, Faitot F, Nappo G, Oncioiu C, Bachellier P
HPB (Oxford) 2017 Jul;19(7):567-572. Epub 2017 Apr 17 doi: 10.1016/j.hpb.2017.03.005. PMID: 28427830
Sautter NB, Smith TL
Int Forum Allergy Rhinol 2012 Sep-Oct;2(5):422-31. Epub 2012 May 7 doi: 10.1002/alr.21046. PMID: 22566463

Supplemental Content

Table of contents

    Clinical resources

    Practice guidelines

    • PubMed
      See practice and clinical guidelines in PubMed. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.
    • Bookshelf
      See practice and clinical guidelines in NCBI Bookshelf. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.

    Recent activity

    Your browsing activity is empty.

    Activity recording is turned off.

    Turn recording back on

    See more...