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Antiestrogen therapy for hereditary hemorrhagic telangiectasia: a double-blind placebo-controlled clinical trial.

机译:遗传性出血性毛细血管扩张的抗雌激素治疗:一项双盲安慰剂对照临床试验。

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摘要

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.
机译:目的/假设:遗传性出血性毛细血管扩张(HHT)与90%的复发性鼻epi有关。尽管激素的使用仍存在争议,但已证明对激素的良好反应。这项研究的目的是检查抗雌激素剂他莫昔芬在治疗HHT相关的鼻出血中的功效。方法:25名诊断为HHT导致鼻epi的患者(11例男性,14例女性,平均年龄51岁)被随机分配接受口服他莫昔芬20 mg / d或安慰剂治疗6个月。随访包括体格检查和每月一次的血液检查。结果:两组的年龄和性别分布相似。在完成该试验的21名参与者中,在他莫昔芬治疗的10名患者中有9名在安慰剂治疗的11名患者中有3名(包括2名仅有暂时性改善)的患者,鼻the减轻。在试验终点时,两组之间的差异对于该疾病的发生频率(P = 0.01)和严重程度(P = .049)均很显着。 4名他莫昔芬治疗的患者血红蛋白浓度上升,而5名对照者血红蛋白浓度下降。结论:他莫昔芬似乎是治疗因HHT引起的鼻axis的有效药物。

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