Thalidomide for Epistaxis in Patients with Hereditary Hemorrhagic Telangiectasia: A Preliminary Study
Thalidomide for Epistaxis in Patients with Hereditary Hemorrhagic Telangiectasia: A Preliminary Study
复制标题
沙利度胺治疗遗传性出血性毛细血管扩张症患者鼻出血的初步研究
DOI：
10.1177/0194599817700573
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发表时间：
2017-08-01
影响因子：
3.4
通讯作者：
Su, Kaiming
中科院分区：
文献类型：
--
作者：
Fang, Jia;Chen, Xiaomeng;Su, Kaiming
为了评估沙利度胺治疗遗传性出血性毛细血管扩张症 (HHT) 鼻出血的有效性，7 例复发性鼻出血的 HHT 患者接受了沙利度胺治疗，初始剂量为 50 mg/d，如果需要，逐渐增加至 100 mg/d。鼻衄严重程度评分（ESS）用于评估治疗效果。患者报告说，开始使用沙利度胺后 1 至 3 周，鼻出血有所改善。治疗前、治疗结束时和停止治疗后 3 个月的平均 ESS 分别为 5.03 ± 2.05、0.90 ± 0.84 (P = .003) 和 1.98 ± 1.33 (P = .006)。四名患者报告有轻度至中度副作用，包括嗜睡、头晕、便秘、恶心和周围神经病变。两名患者因不良反应而停止治疗。这些结果表明，沙利度胺可能是 HHT 患者复发性鼻出血的治疗选择，但应考虑其副作用。进一步的研究应侧重于剂量和疗程指南，并研究如何减少不良反应。
To evaluate the effectiveness of thalidomide for epistaxis in hereditary hemorrhagic telangiectasia (HHT), 7 HHT patients with recurrent epistaxis were treated with thalidomide at an initial dose of 50 mg/d, gradually increasing to 100 mg/d if needed. The Epistaxis Severity Score (ESS) was used to evaluate the treatment effects. Patients reported that epistaxis improved 1 to 3 weeks after starting thalidomide. The mean ESS before treatment, at the end of treatment, and 3 months after stopping treatment was 5.03 ± 2.05, 0.90 ± 0.84 (P = .003), and 1.98 ± 1.33 (P = .006), respectively. Four patients reported mild to moderate side effects, including drowsiness, dizziness, constipation, nausea, and peripheral neuropathy. Two patients stopped the treatment because of adverse effects. Those results showed that thalidomide may be a treatment choice for recurrent epistaxis in HHT patients, although the side effects should be considered. Further study should focus on guidelines for dosing and course and investigate how to reduce the adverse effects.