VCR治疗激素不敏感卡波西样血管内皮瘤伴K—M现象33例体会

Kaposiform hemangioendothelioma： a retrospective study of 33 steroid-resistant cases treated with vincristine and long follow-ups

目的评价长春新碱（vincristine，VCR）治疗卡波西样血管内皮瘤（Kaposiform hemangio endothelioma，KHE）伴K-M现象（Kasabach-Merritt phenomenon，KMP）的效用。方法回顾性分析我院2003年3月至2013年3月33例诊断为KHE伴KMP且激素治疗不敏感改用VCR治疗的患儿的临床资料并评估疗效、副作用及预后。33例患儿发病年龄1d-10个月。男19例，女14例。浅部KHE7例，深部KHE26例。33例均有血小板减少，25例同时伴有凝血功能异常。结果每例患儿前期均用激素治疗，但因病情反复或无效，改用VCR治疗。其中22例治愈，VCR治疗后血小板计数升至100×10^9／L以上平均时间（7．5±5．2）周，肿块开始软化或缩小时间为（4．9±2．6）周。2例显效，1例仍在治疗。8例无明显疗效或病情反复，改用其他治疗。24例VCR治疗患儿已停药，平均治疗周期为（31．1±6．0）周，平均随访时间3．7年，最长随访7．i年，无复发。结论激素治疗KHE伴KMP疗效有限，VCR治疗该病具有较好的安全性和有效性，对于激素无反应的患儿，建议改用VCR治疗。

Objective To evaluate the efficacy of vincristine （VCR） in the treatment of steroid- resistant patients of kaposiform hemangioendothelioma （KHE） with Kasabach-Merritt phenomenon （KMP）. Methods Retrospective analyses were conducted for the clinical data of 33 steroid-resistant cases of KHE with KMP on VCR at our hospital from March 2003 to March 2013. Results There were 19 boys and 14 girls. Their age during an initial diagnosis of KHE was 1 day to 10 months, Seven lesions were located in superficial soft tissue while deep soft tissue was involved in 26 cases. All cases were diagnosed with thrombocytopenia and 25 had coagulopathy. None of them was responsive to steroids before starting VCR treatment. And 22 cases achieved complete remission with a normalization of platelet count within （7. 5 ± 5.2） weeks after VCR treatment. The masses shrank in size or became softer at an average of （4. 9 ±2. 6） weeks. Two cases had a partial response and 1 case stayed in treatment. And 8 cases had no obvious response to VCR and switched to other treatments. For 24 withdrawal cases, the average course of treatment was （31.1 ±6. 0） weeks. The mean and longest follow-up periods was 3.7 and 7. 1 years respectively. And there was no recurrence. Conclusions VCR is both safe and effective in the treatment of steroid-resistant cases of KHE with KMP. And it should be applied early and recommended as a first line option.