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ENGLISH ABSTRACT
外用西罗莫司治疗儿童结节性硬化症血管纤维瘤的疗效及安全性研究
王森分
王旭
魏京海
张建
刘元香
徐子刚
作者及单位信息
·
DOI： 10.3760/cma.j.issn.0412-4030.2016.07.005
Efficacy and safety of topical sirolimus in the treatment of facial angiofibromas in children with tuberous sclerosis complex
Wang Senfen
Wang Xu
Wei Jinghai
Zhang Jian
Liu Yuanxiang
Xu Zigang
Authors Info & Affiliations
Wang Senfen
Department of Dermatology, Beijing Children's Hospital, Capital Medical University, Beijing 100045, China
Wang Xu
Department of Neurology, Beijing Children's Hospital, Capital Medical University, Beijing 100045, China
Wei Jinghai
Department of Pharmacy, Beijing Children's Hospital, Capital Medical University, Beijing 100045, China
Zhang Jian
Department of Pharmacy, Beijing Children's Hospital, Capital Medical University, Beijing 100045, China
Liu Yuanxiang
Department of Dermatology, Beijing Children's Hospital, Capital Medical University, Beijing 100045, China
Xu Zigang
Department of Dermatology, Beijing Children's Hospital, Capital Medical University, Beijing 100045, China
·
DOI： 10.3760/cma.j.issn.0412-4030.2016.07.005
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摘要

目的研究0.1％西罗莫司软膏治疗儿童结节性硬化症血管纤维瘤的临床疗效及安全性。

方法自制0.1％西罗莫司软膏。选取结节性硬化症面部血管纤维瘤患儿20例，予0.1％西罗莫司软膏外用，每天2次。分别于治疗4周、12周时，记录面部血管纤维瘤严重程度指数(FASI评分)、皮损治疗满意度及不良反应等。治疗前、治疗12周时，检测西罗莫司血药浓度、血尿常规、凝血功能、血生化及免疫指标。

结果治疗前、治疗4周、治疗12周时，20例患儿FASI评分分别为5.750±1.175、4.400±1.284和2.975±1.543，治疗4周、12周时的FASI评分均较治疗前显著下降(均 P < 0.000 1)，且治疗12周时显著低于治疗4周时( P < 0.000 1)。治疗12周时平均疗效指数(49.87%±22.08%)显著高于治疗4周时(24.43%±10.18%)，差异有统计学意义( t= 7.338， P < 0.01)。治疗12周时，皮损颜色明显变淡，体积明显缩小，瘤体数量减少，2例皮损完全消退。治疗4周时，10例对红斑减淡程度满意，3例对皮损体积改善满意，3例对皮损面积改善满意。治疗12周时，家长的满意度均提高。20例患儿治疗前后西罗莫司血药浓度均低于1.0 μg/L，无严重系统及皮肤不良反应发生。

结论0.1％西罗莫司软膏治疗结节性硬化症血管纤维瘤疗效显著，安全性好。

结节性硬化症;血管纤维瘤;西罗莫司;治疗结果
ABSTRACT

ObjectiveTo investigate the efficacy and safety of sirolimus 0.1% ointment in the treatment of facial angiofibromas in children with tuberous sclerosis complex.

MethodsSirolimus 0.1% ointment was prepared. Twenty children with tuberous sclerosis complex who had facial angiofibromas were enrolled in this study. Facial angiofibromas were topically treated with the self-prepared sirolimus 0.1% ointment twice a day for 12 weeks. The facial angiofibroma severity index (FASI)was calculated, the degree of satisfaction with the treatment was evaluated, and adverse reactions were analyzed at weeks 4 and 12. Plasma sirolimus concentrations as well as blood biochemical and immunological parameters were measured, blood coagulation activity was evaluated, and routine blood tests as well as urine tests were performed at baseline and week 12.

ResultsThe FASI of patients significantly decreased at weeks 4 (4.400±1.284)and 12 (2.975±1.543)compared with that at baseline (5.750±1.175, both P < 0.000 1), and was significantly lower at week 12 than at week 4 ( P < 0.000 1). The efficacy index was 49.87%±22.08% at week 12, significantly higher than that at week 4 (24.43%±10.18%, t = 7.338, P < 0.01). The color, size and number of lesions significantly decreased in all the patients, and facial angiofibromas completely disappeared in 2 patients at week 12. At week 4, 10 parents were satisfied with the improvement of erythema, 3 parents with that of lesion volume, and 3 parents with that of lesion area. The degree of parent satisfaction increased at week 12 in all the cases. The blood concentration of sirolimus was lower than 1.0 μg/L both before and after the treatment. No severe systemic or local adverse reactions were noted in these patients.

ConclusionSirolimus 0.1% ointment is markedly effective and safe for the treatment of facial angiofibromas in patients with tuberous sclerosis complex.

Tuberous sclerosis;Angiofibroma;Sirolimus;Treatment outcome
Xu Zigang, Email: mocdef.oabohayuxgnagiz
the Capital Clinical Characteristic Application Research Program of Beijing Municipal Science and Technology Commission (Z161100000516070); Beijing Municipal Administration of Hospitals Clinical Medicine Development of Special Funding Support (ZYLX201601)
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引用本文

王森分,王旭,魏京海,等. 外用西罗莫司治疗儿童结节性硬化症血管纤维瘤的疗效及安全性研究[J]. 中华皮肤科杂志,2016,49(7)：469-473.

DOI:10.3760/cma.j.issn.0412-4030.2016.07.005

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结节性硬化症(tuberous sclerosis complex ，TSC)是一种常染色体显性遗传的神经皮肤综合征，其发病率一般为1 /6 000～1/10 000，受累器官主要为神经系统、皮肤、心脏、肾脏等 [ 1 , 2 , 3 ]。面部血管纤维瘤是TSC特征性损害，可见于约80％的TSC患者，具有损容性，对患者的身心健康造成极大的影响 [ 1 , 2 , 3 , 4 ]。目前治疗面部血管纤维瘤的方法包括冷冻、磨削、激光和光动力等，这些治疗方法均为有创治疗，可能会遗留永久性瘢痕，且由于复发，患者需要反复治疗 [ 1 , 2 , 3 ]，故对儿童患者多采用随诊观察。有研究报道，口服或外用西罗莫司治疗结节性硬化症皮肤症状，取得一定疗效 [ 2 , 3 , 5 ]。2015年3月以来，我们应用0.1％西罗莫司软膏治疗儿童TSC面部血管纤维瘤，获得较好疗效。
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备注信息
A
徐子刚，Email ： mocdef.oabohayuxgnagiz
B
北京市科委"首都临床特色应用研究"专项课题 (Z161100000516070)
北京市医院管理局临床医学发展专项 (ZYLX201601)
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