Antiestrogen therapy for hereditary hemorrhagic telangiectasia: a double-blind placebo-controlled clinical trial.
OBJECTIVE
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.
Yaniv
Eitan
E
Nose and Sinus Institute, Rabin Medical Center, Petah Tiqwa, and the Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel. docyaniv@yahoo.com
Preis
Michal
M
Hadar
Tuvia
T
Shvero
Jacob
J
Haddad
Miriam
M
D000284:Administration, Oral; D000328:Adult; D000368:Aged; D004311:Double-Blind Method; D004844:Epistaxis / Q000188:drug therapy* / Q000209:etiology; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010919:Placebos; D012720:Severity of Illness Index; D013629:Tamoxifen / Q000627:therapeutic use*; D013683:Telangiectasia, Hereditary Hemorrhagic / Q000150:complications / Q000188:drug therapy*; D016896:Treatment Outcome
D010919:Placebos; D013629:Tamoxifen