西罗莫司治疗卡梅现象后的血小板动态变化  

Fluctuation of the platelet counts during the treatment of patients with sirolimus

作　　者：王媛[1] 高庆文[1] 孔亮亮 沈卫民[1] Wang Yuan;Gao Qingwen;Kong Liangliang;Shen Weimin(Department of Burn and Plastic Surgery,Children’s Hospital of Nanjing Medical University,Nanjing 210008,China)

机构地区：[1]南京医科大学附属儿童医院烧伤整形外科,210008

出　　处：《中华整形外科杂志》2020年第9期1018-1023,共6页Chinese Journal of Plastic Surgery

摘　　要：目的观察西罗莫司治疗卡梅现象(KMP)患儿后血小板的动态变化情况,探讨西罗莫司治疗KMP的效果。方法选取南京市儿童医院烧伤整形外科2017年1月至2019年6月收治的4例KMP病例,男2例,女2例,年龄2 d至3个月,均表现为巨大包块,其中3例位于头颈部,1例位于腹膜后。患儿均出现明显血小板减少(<100×10^9/L)、低纤维蛋白原血症(<100μg/L)及D-二聚体升高(>6000 ng/ml),手术切除病灶、血小板支持治疗或激素治疗均无法纠正血小板减少及凝血功能障碍。3例患儿根据术后病理结果结合血小板计数及凝血功能,诊断为KHE伴KMP。1例患儿未行活检,根据患儿的B超及CT结果,结合血小板计数及凝血功能,诊断为KMP。4例患儿均加用西罗莫司,初始剂量每天0.1 mg/kg或0.8 mg/m^2,每12 h 1次口服,用药后3 d测量药物谷浓度,适当调整剂量,将血药谷浓度维持在10~15 ng/ml。原激素用量逐渐减少,直至停药。观察患儿服药后情况,定期测量患儿血小板数值。结果患儿在使用西罗莫司后血小板均升至正常范围。3例患儿在接受西罗莫司治疗1~4 d内,均出现了短期内明显的血小板降低,甚至降至历史最低水平。2例患儿在使用西罗莫司治疗后病灶停止肿胀增大,包块逐渐缩小变硬,皮肤表面青紫减轻,但肿块未完全消退。另2例在病情稳定一段时间后出现肝功能损害和严重的肺部感染,其中1例经对症治疗后好转,另1例死亡。随访至1年,存活患儿血小板均维持在正常范围内。结论西罗莫司有助于KMP患者血小板的稳定,但不能完全治愈原发病灶。Objective To investigate the safety and efficacy of sirolimus through the change in platelet counts during the sirolimus therapy on Kasaback-Merritt phenomenon(KMP).Methods Four patients were treated in Nanjing Children Hospital between Jaunary 2017 and June 2019 were enrolled in the study,including two males and two females.Their age was ranged from two days to three months.They all presented with huge mass located in neck or retroperitoneal and thrombocytopenia,hypofibrinogenemia.Their coagulation could not be improved by surgery or palate transfusion or steroids.They were all diagnosed as Kaposiform hemangioendothelioma(KHE)with KMP according to the biopsy,coagulation index and CT.Sirolimus were administered with 0.1 mg^-1·kg^-1·d^-1 or 0.8 mg/m^2,twice daily.Subsequent dose was adjusted to maintain the trough level between 10-15 ng/ml.Steroids were weaned gradually.Blood accounts were measured following the sirolimus administration.Results All the four patients got improvement in platelet counts.Three patients went through a significant decreasing of palate counts after adding sirolimus for about 1-4 days.Two lesions prompted improvement with smaller size,lighter color and softer texture without complete regression.Two patients had elevated liver enzymes and/or interstitial pneumonia.One recovered while the other died of severe pneumonia with dyspnea.All the palate counts of survived patients remained in normal level in the following one year.Conclusions Sirolimus is an effective method in treating KMP and stabilizing platelet counts.However,it can not cure hemangioendothelioma.

关 键 词：西罗莫司 KASABACH-MERRITT综合征 血小板计数 治疗效果 

分 类 号：R969[医药卫生—药理学]