The results of a phase II study suggest a role for thalidomide in patients with hereditary haemorrhagic telangiectasia.

Published in The Lancet Haematology the study involved 31 patients with hereditary haemorrhagic telangiectasia and severe recurrent epistaxis.

Each patient received thalidomide at a starting daily dose of 50 mg, which was progressively increased up to 200 mg/day if they had no response.

After a median follow-up of 15·9 months, 50mg of thalidomide a day was highly effective in reduction of the frequency, intensity, and duration of nose bleeds in 25 (81%) of 31 of patients.

The remaining patients who were initially non-responders (six [19%] patients) had a response at increased doses of thalidomide (100–150 mg/day).

The treatment significantly increased patients’ haemoglobin concentrations and prevented the need for transfusion in 20 (87%) of 23 patients who were dependent on transfusions, the researchers reported.

The median time to relapse after the end of treatment with thalidomide was more than 6 months.

An accompanying editorial described the results as ‘intriguing’ but said further trials with more patients was needed to assess the long-term safety and efficacy, as well as the effects of thalidomide treatment on quality of life.