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贝伐单抗对遗传性出血性毛细血管扩张症所致家族性鼻出血的影响

[Effects of bevacizumab on familial epistaxis caused by hereditary hemorrhagic telangiectasia].

作者信息

Zhang J, Wang X S, Wang H T

机构信息

Department of Otorhinolaryngology Head and Neck Surgery, Beijing Anzhen Hospital, Capital Medical University, Beijing 100029, China Department of Otorhinolaryngology Head and Neck Surgery, the First Clinical Center of Chinese People's Liberation Army General Hospital, Beijing 100853, China.

Department of Otorhinolaryngology, Binzhou Central Hospital, Binzhou Medical University, Huimin 251700, Shandong Province, China.

出版信息

Zhonghua Er Bi Yan Hou Tou Jing Wai Ke Za Zhi. 2021 May 7;56(5):436-441. doi: 10.3760/cma.j.cn115330-20201222-00939.

DOI:10.3760/cma.j.cn115330-20201222-00939
PMID:34010995
Abstract

To observe the clinical effects of bevacizumab in the treatment of familial epistaxis caused by hereditary hemorrhagic telangiectasia (HHT). The data of 27 patients with familial epistaxis caused by HHT who were treated with bevacizumab intravenously from Beijing Anzhen Hospital, the First Clinical Center of Chinese People's Liberation Army General Hospital and Binzhou Central Hospital between December 2016 and December 2019 were retrospectively analyzed. There were 14 males and 13 females, aged (55.3±11.2) years. The dose of bevacizumab was calculated according to the body weight of 5 mg/kg. The curative effect was observed one month after the first treatment. Visual analogue scale (VAS) was used to compare patients' self-scores of systemic symptoms before and after treatment. Epistaxis severity score (ESS) was used to compare and analyze the six problems (including the frequency, duration, intensity, treatment demand, anemia and blood transfusion) of the patients before and after treatment. The changes of hemoglobin levels before and after treatment were compared. SPSS 20.0 statistical software was used to process the data. Among the 27 patients at one month after the first bevacizumab treatment, 22 cases reported that the severity of epistaxis was improved significantly, and 5 cases reported that the treatment effect was not significant. The effective rate was 81.5% (22/27). The significant effect in 22 patients lasted for 5-24 months, with a median duration of 11.23 months. The VAS score of systemic symptoms decreased significantly compared with that before treatment (2.41±2.55 8.19±1.47, =9.708, <0.01). The scores of six aspects and standardized scores of ESS were significantly decreased after treatment (epistaxis frequency: 1.78±1.22 3.44±0.80, =6.814, <0.01; epistaxis duration: 0.85±0.91 3.00±0.73, =8.845, <0.01; epistaxis intensity: 0.19±0.40 1.00±0.00, =10.696, <0.01; treatment demand: 0.22 ± 0.42 1.00±0.00, =9.539, <0.01; anemia: 0.41±0.50 0.89±0.32, =4.914, <0.01; blood transfusion: 0.11±0.32 0.41±0.50, =3.309, <0.01; ESS standardized score: 2.50±2.45 7.60±1.30, =9.344, <0.01). The hemoglobin level after treatment was significantly higher than that before treatment ((105.48±24.31) g/L (73.07±23.71) g/L, =6.864, <0.01). Among the 27 patients, there were 8 cases of HHT1 ( gene) and 19 cases of HHT2 ( gene). The improvement duration of epistaxis in group HHT1 and group HHT2 was (4.76±5.12) months and (7.60±10.84) months, respectively, which was in group HHT2 longer than that of group HHT1, but there was no significant difference between the two groups (>0.05). There was no significant difference in ESS scores between the two groups before and after treatment (>0.05). Two female patients had amenorrhea after the first medication. All patients had no other adverse reactions and complications. Intravenous bevacizumab is significantly effective and safe in the treatment of familial epistaxis caused by HHT.

摘要

观察贝伐单抗治疗遗传性出血性毛细血管扩张症(HHT)所致家族性鼻出血的临床疗效。回顾性分析2016年12月至2019年12月在北京安贞医院、中国人民解放军总医院第一临床中心和滨州市中心医院接受静脉注射贝伐单抗治疗的27例HHT所致家族性鼻出血患者的数据。其中男性14例,女性13例,年龄(55.3±11.2)岁。贝伐单抗剂量按体重5mg/kg计算。首次治疗1个月后观察疗效。采用视觉模拟评分法(VAS)比较患者治疗前后全身症状的自我评分。采用鼻出血严重程度评分(ESS)比较分析患者治疗前后的六个问题(包括发作频率、持续时间、严重程度、治疗需求、贫血和输血情况)。比较治疗前后血红蛋白水平的变化。采用SPSS 20.0统计软件处理数据。27例患者首次使用贝伐单抗治疗1个月后,22例报告鼻出血严重程度明显改善,5例报告治疗效果不明显。有效率为81.5%(22/27)。22例显效患者的疗效持续5 - 24个月,中位持续时间为11.23个月。全身症状VAS评分较治疗前显著降低(2.41±2.55比8.19±1.47,t =9.708,P<0.01)。治疗后六个方面的评分及ESS标准化评分均显著降低(鼻出血发作频率:1.78±1.22比3.44±0.80,t =6.814,P<0.01;鼻出血持续时间:0.85±0.91比3.00±0.73,t =8.845,P<0.01;鼻出血严重程度:0.19±0.40比1.00±0.00,t =10.696,P<0.01;治疗需求:0.22±0.42比1.00±0.00,t =9.539,P<0.01;贫血:0.41±0.50比0.89±0.32,t =4.914,P<0.01;输血:0.11±0.32比0.41±0.50,t =3.309,P<0.01;ESS标准化评分:2.50±2.45比7.60±1.30,t =9.344,P<0.01)。治疗后血红蛋白水平显著高于治疗前((105.48±24.31)g/L比(73.07±23.71)g/L,t =6.864,P<0.01)。27例患者中,HHT1型(基因)8例,HHT2型(基因)19例。HHT1组和HHT2组鼻出血改善持续时间分别为(4.76±5.12)个月和(7.60±10.84)个月,HHT2组长于HHT1组,但两组间差异无统计学意义(P>0.05)。两组治疗前后ESS评分差异无统计学意义(P>0.05)。2例女性患者首次用药后出现闭经。所有患者均无其他不良反应及并发症。静脉注射贝伐单抗治疗HHT所致家族性鼻出血疗效显著且安全。

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