Hereditary hemorrhagic telangiectasia (Osler‑Weber‑Rendu syndrome) – Part II. Pharmacological therapy and international guidelines for the therapy 2020
Authors:
Zdeněk Adam 1; Dagmar Brančiková 1; Gabriela Romanová 2; Luděk Pour 1; Marta Krejčí 1; Jiří König 3; Tomáš Nebeský 4; Zuzana Adamová 5; Martin Štork 1; Martin Krejčí 1; Sabina Ševčíková 4; Michal Eid 1; Viera Sandecká 1; Zdeněk Král 1
Authors‘ workplace:
Interní hematologická a onkologická klinika LF MU a FN Brno
1; Oddělení klinické hematologie FN Brno
2; Oddělní krční, nosní, ušní FN Brno
3; Klinika radiologie a nukleární medicíny – FN Brno
4; Ústav patologické fyziologie LF MU a FN Brno
5; Chirurgické oddělení nemocnice Vsetín
6
Published in:
Vnitř Lék 2021; 67(7): 419-424
Category:
Review Articles
Overview
Hereditary hemorrhagic telangiectasia also known as Osler-Weber-Rendu syndrome, is an disorder that causes abnormal blood vessel formation with bleeding. Inhibition of angiogenesis amelioretes bleeding complication. Anti-angiogenic agents such as bevacizumab, aflibercept, thalidomid, lenadomid and other new anti-angiogenic thyrosinkinase inhibitors, as well as sirolimus and takrolimus have emerged as a promising systemic or local therapy in reducing bleeding complications but are not curative. Other pharmacological agents include iron supplementation, antifibrinolytics and hormonal treatment. This review concentrates on new anti-agioproliferative drugs with effect in HHT- discusses the new biology of HHT, management issues that face the practising hematologist, and considerations of future directions in HHT treatment.
Keywords:
sirolimus – bevacizumab – aflibercept – thalidomid – tacrolimus – hereditary hemorrhagic telangiectasia – lenadomid anti‑angiogenic thyrosinkinase inhibitors
Sources
1. Al‑Samkari H. Hereditary hemorrhagic telangiectasia: systemic therapies, guidelines, and an evolving standard of care. Blood 2021; 137(7): 888–895. doi: 10.1182/blood. 2020008739.
2. Faughnan ME, Mager JJ, Hetts SW et al. Second International Guidelines for the Diagnosis and Management of Hereditary Hemorrhagic Telangiectasia. Ann Intern Med. 2020; 173(12):989–1001. doi: 10.7326/M20-1443.
3. Kritharis A, Al‑Samkari H, Kuter DJ. Hereditary hemorrhagic telangiectasia: diagnosis and management from the hematologist’s perspective. Haematologica 2018; 103 (9): 1433–1443.
4. Tunkel DE, Anne S, Payne SC et al. Clinical Practice Guideline: Nosebleed (Epistaxis). Otolaryngol Head Neck Surg 2020; 162(1_suppl): S1–S38. doi: 10.1177/0194599819890327.
5. Kühnel T, Wirsching K, Wohlgemuth W et al. Hereditary Hemorrhagic Telangiectasia. Otolaryngol Clin North Am. 2018; 51 (1): 237–254.
6. Adam Z, Chlupová G, Neuman A et al. Teleangiectasia hereditaria haemorrhagica – syndrom Osler‑Weber‑Rendu. Popis případu a zkušeností s léčbou. Vnitřní lékařství 2012; 58(6): 477–489.
7. Brančíková D, Eid M, Král Z et al. Hereditární hemoragická teleangiectasie nebo syndrom Oslerův‑Renduův‑Weberův Klinický obraz a léčba. Acta Medicinae 2021, 3.
8. Kastner J, Betka J, Kuchyňková Z- Youngova modifikovaná operace v terapii recidivující epistaxe u hereditární hemorhagické teleangiektazie (Kazuistika). Otorinolaryngologie a foniatrie 2001; 50(4): 253–255.
9. Knížek Z, Vodička J, Vincent O et al. Hereditární hemoragická teleanginektázie – naše zkušenosti. Otorinolaryngologie a foniatrie 2020; 69(4):166–171.
10. Štastný B, Krošlák M. Skúsenosti s chirurgickou liečbou epistaxy pri m. Osler‑Rendu‑Weber. Československá otolaryngologie 1981; 30(1): 53–56.
11. Geisthoff UW, Seyfert UT, Kubler M et al. Treatment of epistaxis in HHT with tranexamic acid- double blind placebo controllet cross over phase IIIB study. Thromb Res 2014; 134:565–571.
12. Kroon S, Snijder RJ, Mager JJ et al. Octreotide for gastrointestinal bleeding in hereditary hemorrhagic telangiectasia: A prospective case series. Am J Hematol 2019; 94(9):E247–E249. doi: 10.1002/ajh.25563.
13. Houghton KD, Umar B, Schairer J. Successful Treatment of Hereditary Hemorrhagic Telangiectasia With Octreotide. ACG Case Rep J 2019; 6 (6): e00088. doi: 10.14309/ crj.0000000000000088.
14. Jeanneret S, Regazzoni L, Favrat B. Rendu‑Osler disease: treatment with oestrogen/ progestagen versus octreotide. BMJ Case Rep 2011; 2011: bcr1120103534. doi: 10.1136/ bcr.11.2010.3534
15. Lee BL, Turner J, Hurley J et al. Two for the price of one: a dual treatment benefit of long‑acting octreotide in occult bleeding and diuretic intractable ascites. Frontline Gastroenterol 2011; 2 (4): 226–229.
16. Kopáčová M, Bureš J, Vykouřil L et al. Intraoperative enteroscopy: ten years‘ experience at a single tertiary center. Surgical endoscopy 2007; 21(7):1111–1116.
17. Harwin J, Sugi MD, Hetts SW et al. The Role of Liver Imaging in Hereditary Hemorrhagic Telangiectasia. J Clin Med 2020; 9(11):3750. doi: 10.3390/jcm9113750.
18. Ernes J, Kramplová V, Charvát F. Oční komplikace po embolizaci v povodí arteria carotis interna – kazuistika. Česká a slovenská oftalmologie 2008; 64(5): 202–206.
19. Lantz KE, Armstrong SQ, Butt F et al. Arteriovenous Malformations in the Setting of Osler‑Weber‑Rendu: What the Radiologist Needs to Know. Curr Probl Diagn Radiol 2021 Mar 11:S0363-0188(21)00046-3. doi: 10.1067/j.cpradiol.2021. 03. 009. Epub ahead of print.
20. Krajina A, Koblížek V, Lojík M et al. Endovaskulární léčba plicních arteriovenózních zkratů. Česká radiologie 2012; 66(2): 125–132.
21. Krajina A, Peregrin JH. Intervenční radiologie: miniinvazivní terapie. vyd. Hradec Králové 2005; 835.
22. Krajina A, Nožička J, Malý J. Embolizace plicni arteriovenózní malformace. Intervenční radiologie. Hradec Králové: Radiodiagnostická klinika 1996; 173–175.
23. Krajina A, Lojík M, Česák T et al. Endovaskulární léčba intrakraniálních aneuryzmat – metodika, indikace, komplikace. Česká a slovenská neurologie a neurochirurgie 2012; 75(5): 552–560.
24. Ozawa H, Ohki T, Kanaoka Y et al. Open arterial reconstruction of multiple hepatic artery aneurysms in a patient with hereditary hemorrhagic telangiectasia: A case report. Medicine (Baltimore) 2016; 95(46): e5430. doi:10.1097/MD.0000000000005430.
25. Felli E, Addeo P, Faitot F et al. Liver transplantation for hereditary hemorrhagic telangiectasia: a systematic review. HPB (Oxford). 2017; 19(7): 567–572. doi: 10.1016/j. hpb.2017. 03. 005.
26. Iyer VN, Saberi B, Heimbach JK et al. Liver Transplantation Trends and Outcomes for Hereditary Hemorrhagic Telangiectasia in the United States. Transplantation 2019; 103(7): 1418–1424.
27. Dumortier J, Dupuis‑Girod S, Valette PJ et al. Recurrence of Hereditary Hemorrhagic Telangiectasia After Liver Transplantation: Clinical Implications and Physiopathological Insights. Hepatology 2019; 69(5): 2232–2240.
28. Ejiri K, Akagi S, Nakamura K et al. H. Liver transplantation in a patient with hereditary haemorrhagic telangiectasia and pulmonary hypertension. Pulm Circ. 2019; 9(4): 2045894019896677. doi 10.1177/2045894019896677.
29. Chavan A, Schumann‑Binarsch S, Schmuck B et al. Emerging role of bevacizumab in management of patients with symptomatic hepatic involvement in Hereditary Hemorrhagic Telangiectasia. Am J Hematol 2017; 92 (11): E641–E644. doi: 10.1002/ajh.24878.
30. Vázquez C, Gonzalez ML, Ferraris et al. Bevacizumab for treating Hereditary Hemorrhagic Telangiectasia patients with severe hepatic involvement or refractory anemia. PLoS One 2020; 15(2): e0228486. doi: 10.1371/journal.pone.0228486.
31. Albiñana V, Bernabeu‑Herrero ME et al. Estrogen therapy for hereditary haemorrhagic telangiectasia (HHT): Effects of raloxifene, on Endoglin and ALK1 expression in endothelial cells. Thromb Haemost 2010; 103(3): 525–534.
32. Yaniv E, Preis M, Shevro J et al. Anti‑estrogen therapy for hereditary hemorrhagic telangiectasia - a long‑term clinical trial. Rhinology 2011; 49(2): 214–2146.
33. D‘Amato RJ, Loughnan MS, Flynn E et al. Thalidomide is an inhibitor of angiogenesis. Proc Natl Acad Sci 1994; 91: 4082–4085.
34. Buscarini E, Botella LM, Geisthoff U et al. Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia. Orphanet J Rare Dis 2019; 14(1):28. doi: 10.1186/s13023-018-0982-4.
Další literatura u autora a na www.casopisvnitrnilekarstvi.cz
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Diabetology Endocrinology Internal medicineArticle was published in
Internal Medicine
2021 Issue 7
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