Antiestrogen Therapy for Hereditary Hemorrhagic Telangiectasia: A Double-Blind Placebo-Controlled Clinical Trial

Objectives/Hypothesis: Hereditary hemorrhagic telangiectasia (HHT) is associated with recurrent epistaxis in 90% of cases. Good response to hormone treatment has been documented, although its use remains controversial. The aim of this study was to examine the efficacy of an antiestrogenic agent, Tamoxifen, in the treatment of HHT-associated epistaxis. Methods: Twenty-five patients (11 men, 14 women; mean age 51 years) with a diagnosis of epistaxis due to HHT were randomly assigned to receive treatment with oral tamoxifen 20 mg/d or placebo for 6 months. Follow-up consisted of physical examination and once-monthly blood tests. Results: The groups were similar in age and sex distribution. Of the 21 participants who - completed the trial, alleviation of the epistaxis was noted in 9 of 10 tamoxifen-treated patients and 3 of 11 placebo-treated patients (including 2 with only temporary improvement). The difference between the groups at the trial end point was significant for both frequency (P = .01) and severity (P = .049) of the disease. Hemoglobin concentration rose in 4 tamoxifen-treated patients and decreased in 5 controls. Conclusions: Tamoxifen appears to be an effective agent for the treatment of epistaxis due to HHT.