Thalidomide for Osler-Weber-Randu – pro

In the management of Hereditary Hemorrhagic Teelangiectasia (HHT) epistaxis, multiple approaches have been tried, but currently there is no established medical treatment available for these patients. Surgical options, such as are being considered for this patient, are largely palliative, with variable and temporary results. One such option is thalidomide. The evidence for efficacy rests of case reports and case series. Uptodate 2015 says: ” Because of the lack of randomized controls and anecdotal nature of these reports, as well as the lack of long-term efficacy and safety data, we believe that use of these agents should be restricted to their evaluation in carefully selected, consenting patients in randomized controlled trials within experienced HHT centers “.

W Shovlin C, Ganesan V. Hereditary hemorrhagic telangiectasia (Osler-Weber-Rendu syndrome). In: UpToDate, Basow, DS (Ed), UpToDate, Waltham, MA, 2014. Literature review current through October 2014

Shovlin CL. Hereditary haemorrhagic telangiectasia: pathophysiology, diagnosis and treatment. Blood Rev 2010; 24:203.

Lebrin F, Srun S, Raymond K, Martin S, van den Brink S, Freitas C, Bréant C, Mathivet T, Larrivée B, Thomas JL, Arthur HM, Westermann CJ, Disch F, Mager JJ, Snijder RJ, Eichmann A, Mummery CL.Thalidomide stimulates vessel maturation and reduces epistaxis in individuals with hereditary hemorrhagic telangiectasia.Nat Med. 2010 Apr;16(4):420-8.

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