Anti-estrogen therapy for hereditary hemorrhagic telangiectasia - a long-term clinical trial.

ABSTRACT

STATEMENT OF PROBLEM: Hereditary hemorrhagic telangiectasia (HHT) is associated with recurrent epistaxis in 90% of the 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 anti-estrogenic agent, tamoxifen, in the treatment of HHT-associated epistaxis. METHOD OF STUDY Out of 46 patients with diagnosis of epistaxis due to HHT who started treatment with tamoxifen 20 mg/d, 38 patients completed a mean of 23.4 months of treatment. All patients filled out a self-assessment questionnaire of rhinologic Quality of Life and epistaxis grading scale. MAIN RESULTS: The bleeding score and the Quality of Life score improved. Hemoglobin concentration also improved. None of the patients needed blood transfusions during the treatment period. Only one patient had minor side effects of the drug. PRINCIPAL CONCLUSIONS: Tamoxifen appears to be an effective agent for the treatment of epistaxis due to HHT.