中华皮肤科杂志 ›› 2020, Vol. 53 ›› Issue (7): 514-518.doi: 10.35541/cjd.20191172

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西罗莫司联合泼尼松治疗卡波西样血管内皮瘤伴Kasabach-Merritt现象初探

周江元1,杨开颖1,彭素华1,邱桐1,代诗懿1,张学鹏2,陈思源2,夏春潮3,吉毅1   

  1. 1. 四川大学华西医院小儿外科，成都 610041
    2. 四川大学华西医院重症医学科，成都 610041
    3. 四川大学华西医院放射科，成都 610041
  • 收稿日期:2019-12-20 修回日期:2020-04-28 发布日期:2020-07-06
  • 通讯作者: 吉毅 E-mail:jijiyuanyuan@163.com
  • 基金资助:
    国家自然科学基金;国家自然科学基金;四川省科技厅重点研发项目;四川大学优秀青年学者基金;四川大学华西医院学科卓越发展1·3·5工程临床研究孵化项目

Sirolimus combined with prednisone in the treatment of Kaposiform hemangioendothelioma complicated by Kasabach-Merritt phenomenon: a preliminary study

Zhou Jiangyuan 1,Yang Kaiying 1,Peng Suhua 1,Qiu Tong 1,Dai Shiyi 1,Zhang Xuepeng 2,Chen Siyuan 2,Xia Chunchao 3,Ji Yi 1   

  1. 1. Department of Pediatric Surgery, West China Hospital, Sichuan University, Chengdu 610041, China;
    2. Department of Critical Care Medicine, West China Hospital, Sichuan University, Chengdu 610041, China;
    3. Department of Radiology, West China Hospital, Sichuan University, Chengdu 610041, China
  • Received:2019-12-20 Revised:2020-04-28 Published:2020-07-06
  • Contact: Ji Yi E-mail:jijiyuanyuan@163.com
  • Supported by:
    National Natural Science Foundation of China;National Natural Science Foundation of China;Key Project in the Science & Technology Program of Sichuan Province;Science Foundation for Excellent Youth Scholars of Sichuan University;The 1·3·5 Project for Disciplines of Excellence-Clinical Research Incubation Project of West China Hospital of Sichuan University

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摘要： 【摘要】 目的 初步探讨西罗莫司联合泼尼松治疗卡波西样血管内皮瘤（KHE）伴Kasabach-Merritt现象（KMP）的临床价值。方法 回顾性分析2011年1月至2018年1月于四川大学华西医院小儿外科接受西罗莫司联合泼尼松治疗的36例KHE伴KMP患儿的一般临床资料、治疗效果以及不良反应发生情况。结果 36例KHE伴KMP患儿中，男女比例为1∶0.8；平均年龄15个月（4.0 ~ 60.0个月）；平均发病年龄6.3个月（0.8 ~ 48.0个月）；混合型KHE 32例（88.9%），深部型KHE 4例（11.1%）；瘤体大小为2.5 cm × 4.0 cm ~ 20.0 cm × 24.0 cm。在联合治疗中，泼尼松平均用药时间为6.4周（范围5.0 ~ 9.0周），西罗莫司平均用药时间为19.3个月（范围13.0 ~ 27.0个月）。联合治疗1 ~ 5周，36例KHE伴KMP患者的血小板和纤维蛋白原逐渐恢复正常。在短期使用泼尼松联合长期使用西罗莫司治疗6、12个月后，疾病严重性平均评分由治疗前4.0分分别下降到2.4分、1.6分。在治疗12个月后，32例患者瘤体达到大部分消退的标准，3例达到部分消退的标准，1例无变化。治疗期间，常见的不良反应为胃肠道不适和口腔溃疡，无患儿因为严重不良反应而中止治疗，也未发生药物毒性相关性死亡。结论 西罗莫司联合泼尼松治疗KHE伴KMP安全有效。

关键词: 西罗莫司, 泼尼松, 血管内皮瘤, Kasabach-Merritt现象, 卡波西样血管内皮瘤

Abstract: 【Abstract】 Objective To preliminarily assess the clinical value of sirolimus combined with prednisone in the treatment of Kaposiform hemangioendothelioma （KHE） complicated by Kasabach-Merritt phenomenon （KMP）. Methods A retrospective study was conducted. General clinical data and relevant data on efficacy and adverse reactions were collected from 36 patients with KHE complicated by KMP, who received oral sirolimus combined with prednisone in Department of Pediatric Surgery, West China Hospital, Sichuan University from January 2011 to January 2018. Results Among the 36 patients with KHE complicated by KMP, the male-to-female ratio was 1∶0.8; their average age was 15.0 months （range, 4.0 - 60.0 months）, and the average age of onset was 6.3 months （range, 0.8 - 48.0 months）; 32 （88.9%） patients were diagnosed with mixed KHE, and 4 （11.1%） with deep KHE; the tumor size ranged from 2.5 cm × 4.0 cm to 20.0 cm × 24.0 cm. During the combined treatment, the average duration of prednisone administration was 6.4 weeks （range, 5.0 - 9.0 weeks）, and that of sirolimus administration was 19.3 months （range, 13.0 - 27.0 months）. After 1 - 5 weeks of combined treatment, platelet counts and fibrinogen levels of the 36 patients gradually returned to normal. After short-term prednisone combined with long-term sirolimus treatment, the average disease severity score decreased from 4.0 before treatment to 2.4 at 6 months and 1.6 at 12 months. After 12 months of the combined treatment, tumors mostly regressed in 32 （88.9%） patients, partially regressed in 3 （8.3%）, showed no obvious change in 1 （2.8%）. During the treatment, common adverse reactions included gastrointestinal discomfort and oral ulcers, no patient discontinued the treatment due to severe adverse reactions, and no drug toxicity-related death occurred. Conclusion Sirolimus combined with prednisone is effective and safe for the treatment of KHE complicated by KMP.

Key words: Sirolimus, Prednisone, Hemangioendothelioma, Kasabach-Merritt phenomenon, Kaposiform hemangioendothelioma

引用本文

周江元 杨开颖 彭素华 邱桐 代诗懿 张学鹏 陈思源 夏春潮 吉毅. 西罗莫司联合泼尼松治疗卡波西样血管内皮瘤伴Kasabach-Merritt现象初探[J]. 中华皮肤科杂志, 2020,53(7):514-518. doi:10.35541/cjd.20191172

Zhou Jiangyuan Yang Kaiying Peng Suhua Qiu Tong Dai Shiyi Zhang Xuepeng Chen Siyuan Xia Chunchao Ji Yi. Sirolimus combined with prednisone in the treatment of Kaposiform hemangioendothelioma complicated by Kasabach-Merritt phenomenon: a preliminary study[J]. Chinese Journal of Dermatology, 2020, 53(7): 514-518.doi:10.35541/cjd.20191172