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

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. 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. 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. 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.