Liu Qianlong, Xiong Na, Gong Xinyuan, Tong Haochongyang, Tan Xuanfeng, Guo Xinkui
Department of Pediatric Surgery, The Second Affiliated Hospital of Xi'an Jiaotong University, Xi'an, Shaanxi 710004, P.R. China.
Department of Science and Education, Tianjin First Central Hospital, Tianjin 300192, P.R. China.
Exp Ther Med. 2022 Aug 9;24(4):621. doi: 10.3892/etm.2022.11558. eCollection 2022 Oct.
Kasabach-Merritt phenomenon (KMP) is a life-threatening condition caused by rare vascular tumors. To reduce drug resistance observed in monotherapy of KMP with prednisone, vincristine (VCR) or sirolimus, the present study evaluated the efficacy and safety of triad therapy in the treatment of KMP. A total of 10 KMP infants managed with prednisolone, VCR and sirolimus in The Second Affiliated Hospital of Xi'an Jiaotong University (Xi'an, China) between April 2017 and August 2021 were retrospectively reviewed. The three female and seven male infants with KMP underwent cocktail therapy with prednisone, VCR and sirolimus. At diagnosis, the infants, aged 49.1±41.0 days, showed laboratory test results with platelet counts 22±15.4x10/l, fibrinogen 81.7±26.9 mg/dl and D-dimer 38649±13443.6 ng/ml. The average maximal diameter of the tumors at diagnosis was 84.5±25.1 mm. KMP risk is increased by large tumors with deep lesions infiltrating the muscle. Platelet counts normalized after a median 10 days (range, 5-69 days) of treatment. With combination therapy maintained for 46.8±24.4 days, ultrasound showed that the thickness of the tumors decreased by 51% from 28.9±12.1 to 13.9±6.2 mm. Neutropenia and gastrointestinal disorders were the most common adverse effects. The present study found that the cocktail therapy with prednisolone, VCR and sirolimus has favorable tolerance and efficacy for life-threatening KMP. Once a stable condition has been achieved, cocktail therapy should be replaced by sirolimus monotherapy to reduce potential side effects.
卡萨巴赫-梅里特现象（KMP）是一种由罕见血管肿瘤引起的危及生命的疾病。为了降低在使用泼尼松、长春新碱（VCR）或西罗莫司单药治疗KMP时观察到的耐药性，本研究评估了三联疗法治疗KMP的疗效和安全性。回顾性分析了2017年4月至2021年8月期间在西安交通大学第二附属医院（中国西安）接受泼尼松龙、VCR和西罗莫司治疗的10例KMP婴儿。3例女性和7例男性KMP婴儿接受了泼尼松、VCR和西罗莫司联合治疗。诊断时，这些年龄为49.1±41.0天的婴儿实验室检查结果显示血小板计数为22±15.4×10⁹/L、纤维蛋白原为81.7±26.9mg/dl、D-二聚体为38649±13443.6ng/ml。诊断时肿瘤的平均最大直径为84.5±25.1mm。伴有深部病变浸润肌肉的大肿瘤会增加KMP风险。治疗中位数10天（范围5 - 69天）后血小板计数恢复正常。联合治疗维持46.8±24.4天后，超声显示肿瘤厚度从28.9±12.1mm降至13.9±6.2mm，减少了51%。中性粒细胞减少和胃肠道疾病是最常见的不良反应。本研究发现，泼尼松龙、VCR和西罗莫司联合治疗对危及生命的KMP具有良好的耐受性和疗效。一旦病情稳定，应将联合治疗替换为西罗莫司单药治疗以减少潜在副作用。
