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The use of thalidomide therapy for refractory epistaxis in hereditary haemorrhagic telangiectasia: systematic review

Published online by Cambridge University Press:  07 September 2018

L Harrison ,
A Kundra  and
P Jervis
L Harrison*
Affiliation:
Department of ENT, Head and Neck Surgery, Northampton General Hospital, UK
A Kundra
Affiliation:
Department of ENT, Head and Neck Surgery, Northampton General Hospital, UK
P Jervis
Affiliation:
Department of ENT, Head and Neck Surgery, Northampton General Hospital, UK
*
Author for correspondence: Ms Laura Harrison, Department of ENT, Head and Neck Surgery, Northampton General Hospital, Northampton, UK E-mail: lauraharrison707@gmail.com
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Abstract

Background

Hereditary haemorrhagic telangiectasia is an autosomal dominant condition, characterised by mucocutaneous telangiectasia, aneurysm and arteriovenous malformations. Thalidomide has been used as a therapeutic strategy for refractory epistaxis in hereditary haemorrhagic telangiectasia patients. This review set out to examine the evidence for using thalidomide in the management of refractory epistaxis in hereditary haemorrhagic telangiectasia patients.

Methods

A systematic search of the available literature was performed using Medline, Embase, Cochrane Library and NHS Evidence databases, from inception to December 2017. The search terms used included: hereditary haemorrhagic telangiectasia (HHT), Osler-Weber-Rendu syndrome, epistaxis, haemorrhage and thalidomide.

Results

All studies using thalidomide therapy showed a reduction in the frequency and duration of epistaxis, as early as four weeks post-therapy. In addition, thalidomide therapy was shown to increase median haemoglobin levels and reduce blood transfusion dependence.

Conclusion

Current available evidence suggests that low-dose thalidomide is effective in transiently reducing epistaxis frequency and duration. Further studies are required to establish a treatment regimen to prevent side effects.

Keywords

EpistaxisHemorrhageThalidomide
Type
Review Articles
Information
The Journal of Laryngology & Otology , Volume 132 , Issue 10 , October 2018 , pp. 866 - 871
Copyright
Copyright © JLO (1984) Limited, 2018 

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Footnotes

Ms L Harrison takes responsibility for the integrity of the content of the paper

References

1Franchini, M, Frattini, F, Crestani, S, Bonfanti, C. Novel treatments for epistaxis in hereditary hemorrhagic telangiectasia: a systematic review of the clinical experience with thalidomide. J Thromb Thrombolysis 2013;36:355–7Google Scholar
2Merlo, CA, Yin, LX, Hoag, JB, Mitchell, SE, Reh, DD. The effects of epistaxis on health-related quality of life in patients with hereditary hemorrhagic telangiectasia. Int Forum Allergy Rhinol 2014;4:921–5Google Scholar
3Pasculli, G, Resta, F, Guastamacchia, E, Di Gennaro, L, Suppressa, P, Sabba, C. Health-related quality of life in a rare disease: hereditary hemorrhagic telangiectasia (HHT) or Rendu-Osler-Weber disease. Qual Life Res 2004;13:1715–23Google Scholar
4McDonald, J, Wooderchak-Donahue, W, Webb, CV, Whitehead, K, Stevenson, DA, Bayrak-Toydemir, P. Hereditary hemorrhagic telangiectasia: genetics and molecular diagnostics in a new era. Front Genet 2015;6:1Google Scholar
5Franks, ME, Macpherson, GR, Figg, WD. Thalidomide. Lancet 2004;363:1802–11Google Scholar
6Lebrin, F, Srun, S, Raymond, K, Martin, S, van den Brink, S, Freitas, C et al. Thalidomide stimulates vessel maturation and reduces epistaxis in individuals with hereditary haemorrhagic telangiectasia. Nat Med 2010;16:420–8Google Scholar
7Invernizzi, R, Quaglia, F, Klersy, C, Pagella, F, Ornati, F, Chu, F. Efficacy and safety of thalidomide for the treatment of severe recurrent epistaxis in hereditary haemorrhagic telangiectasia: results of a non-randomised, single-centre, phase 2 study. Lancet Haematol 2015;2:e465–73Google Scholar
8Peng, HL, Yi, YF, Zhou, SK, Xie, SS, Zhang, GS. Thalidomide effects in patients with hereditary haemorrhagic telangiectasia during therapeutic treatment in Fli-EGFP transgenic zebrafish model. Chin Med J (Engl) 2015;128:3050–4Google Scholar
9Hosman, A, Westermann, CJ, Snijder, R, Disch, F, Mummery, CL, Mager, JJ. Follow-up of thalidomide treatment in patients with hereditary haemorrhagic telangiectasia. Rhinology 2015;53:340–4Google Scholar
10Penaloza, A, Vekemans, MC, Lambert, C, Hermans, C. Deep vein thrombosis induced by thalidomide to control epistaxis secondary to hereditary hemorrhagic telangiectasia. Blood Coagul Fibrinolysis 2011;22:616–18Google Scholar
11Kurstin, R. Using thalidomide in a patient with epithelioid leiomyosarcoma and Osler-Weber-Rendu disease. Oncology 2002;16:21–4Google Scholar
12Fang, J, Chen, X, Zhu, B, Ye, H, Zhang, W, Guan, J et al. Thalidomide for epistaxis in patients with hereditary haemorrhage telangiectasia: a preliminary study. Otolaryngol Head Neck Surgery 2017;157:217–21Google Scholar
13Thalgott, J, Dos-Santos-Luis, D, Lebrin, F. Pericytes as targets in hereditary haemorrhagic telangiectasia. Front Genet 2015;6:37Google Scholar
14Berg, J, Porteous, M, Reinhardt, D, Gallione, C, Holloway, S, Umasunthar, T et al. Hereditary haemorrhagic telangiectasia: a questionnaire based study to delineate the different phenotypes caused by endoglin and ALK1 mutations. J Med Genet 2013;40:585–90Google Scholar
15Teo, SK, Stirling, DI, Zeldis, JB. Thalidomide as a novel therapeutic agent: new uses for an old product. Drug Discov Today 2005;10:107–14Google Scholar
16Chaudhry, V, Comblath, DR, Polydefkis, M, Ferguson, A, Borrello, I. Characteristics of bortezomib- and thalidomide-induced peripheral neuropathy. J Peripher Nerv Syst 2008;13:275–82Google Scholar
17Colagrande, M, Di Ianni, M, Coletti, G, Peris, K, Fargnoli, MC, Moretti, L et al. Toxic epidermal necrolysis in a patient with primary myelofibrosis receiving thalidomide therapy. Int J Hematol 2009;89:76–9Google Scholar
18Boey, JP, Hahn, U, Sagheer, S, McRae, SJ. Thalidomide in angiodysplasia-related bleeding. Intern Med J 2015;45:972–6Google Scholar
19Colombo, G, Bortolotti, F, Chiapponi, V, Buttini, F, Sonvico, F, Invernizzi, R et al. Nasal powders of thalidomide for local treatment of nose bleeding in persons affected by hereditary hemorrhagic telangiectasia. Int J Pharm 2016;30:229–37Google Scholar
20Livesey, JA, Manning, RA, Meek, JH, Jackson, JE, Kulinskaya, E, Laffan, MA et al. Low serum iron levels are associated with elevated plasma levels of coagulation factor VIII and pulmonary emboli/deep venous thromboses in replicate cohorts of patients with hereditary haemorrhagic telangiectasia. Thorax 2012;7:328–33Google Scholar
21Shovlin, CL, Sulaiman, NL, Govani, FS, Jackson, JE, Begbie, ME. Elevated factor VIII in hereditary haemorrhagic telangiectasia (HHT): association with venous thromboembolism. Thromb Haemost 2007;98:1031–9Google Scholar
22International Myeloma Foundation. Understanding Thalidomide Therapy. In: https://www.myeloma.org/sites/default/files/images/publications/UnderstandingPDF/u-thalidomide.pdf [20 August 2018]Google Scholar
23National Institute for Health and Care Excellence. Bortezomib and thalidomide for the first-line treatment of multiple myeloma. In: https://www.nice.org.uk/guidance/ta228 [20 August 2018]Google Scholar
24Gallitelli, M, Pasculli, G, Fiore, T, Carella, A, Sabba, C. Emergencies in hereditary haemorrhagic telangiectasia. QJM 2006;99:15–22Google Scholar
25Folz, BJ, Tennie, J, Lippert, BM, Werner, JA. Natural history and control of epistaxis in a group of German patients with Rendu-Osler-Weber disease. Rhinology 2005;43:40–6Google Scholar
26Assar, OS, Friedman, CM, White, RI Jr. The natural history of epistaxis in hereditary hemorrhagic telangiectasia. Laryngoscope 1991;101:977–80Google Scholar