Objective: To study the clinical features, treatment, and prognosis of neonates with Kasabach-Merritt syndrome (KMS), and to provide a reference for optimizing the diagnosis and treatment of this disease.
Methods: A retrospective analysis was performed for the clinical and follow-up data of 16 neonates with KMS who were admitted to the Anhui Children's Hospital, Anhui Medical University, from January 2016 to December 2020.
Results: Of the 16 neonates, there were 13 boys (81%) and 3 girls (19%), with an age of 1 hour to 10 days on admission. Among these neonates, 13 (81%) had cutaneous hemangioma (2 in the head and face, 5 in the trunk, and 6 in the extremities) and 3 (19%) had liver hemangioma. The main clinical manifestations of bleeding tendency and scattered petechiae and ecchymosis were observed in 10 neonates (62%). All the 16 neonates had varying degrees of thrombocytopenia and coagulation disorders. They all received glucocorticoid treatment after admission and 7 (44%) of them had response, among whom 4 experienced recurrence. Among the neonates with no response to glucocorticoid treatment, 3 received sirolimus treatment, among whom 1 had the tumor volume reduced by 58.8% after 4 weeks of treatment, with platelet count and coagulation function returning to normal, while 2 had no significant reduction in tumor volume or significant increase in platelet count and achieved a tumor volume reduced by (43.7±0.4)% after 4 weeks of combined treatment with bleomycin arterial embolization, with platelet count and coagulation function returning to normal. After 4 weeks of bleomycin arterial embolization alone for 4 neonates, tumor volume was reduced by (52.0±3.4)%, and platelet count and coagulation function returned to normal. Blunt and sharp dissection was performed for 2 neonates. The tumor was removed completely during surgery in the 2 neonates, with no infection or recurrence after surgery, and platelet count and coagulation function returned to normal. The postoperative pathological examination showed Kaposiform hemangioendothelioma in 1 out of the 2 neonates.
Conclusions: KMS has characteristic clinical manifestations, histopathological features, and laboratory examination results. The KMS neonates who are not sensitive to glucocorticoids can achieve a good curative effect through arterial embolization and sirolimus treatment.
目的: 分析总结新生儿卡梅综合征（Kasabach-Merritt syndrome，KMS）的临床特点、治疗及预后，为优化该病的诊断及治疗提供参考依据。
方法: 回顾性分析安徽医科大学附属省儿童医院2016年1月至2020年12月收治的16例KMS新生儿的住院及随访资料。
结果: 16例KMS患儿中，男13例（81%），女3例（19%），入院年龄为1 h至10 d。13例（81%）为皮肤血管瘤（头面部2例、躯干5例、四肢6例），3例（19%）为肝脏血管瘤。10例（62%）临床表现以出血倾向、全身散在瘀点瘀斑为主。16例患儿均有不同程度的血小板减少及凝血功能异常。所有患儿入院后均采取糖皮质激素保守治疗，7例（44%）有效，其中复发4例。糖皮质激素治疗无效患儿中3例接受西罗莫司治疗，1例治疗4周后瘤体缩小58.8%，血小板计数及凝血功能恢复正常；2例瘤体无明显缩小，血小板计数无明显回升，联合博来霉素动脉栓塞治疗4周后瘤体缩小（43.7±0.4）%，血小板计数及凝血功能恢复正常。4例单独接受博来霉素动脉栓塞治疗4周后瘤体缩小（52.0±3.4）%，血小板计数及凝血功能恢复正常。2例接受外科钝性+锐性剥离切除术，术中切除全部瘤体，术后无感染及复发，血小板计数及凝血功能恢复正常，1例术后病理结果为卡波西样血管内皮瘤。
结论: KMS具有特征性临床表现、组织病理特征及实验室检查结果。对激素反应不敏感的KMS通过动脉栓塞和西罗莫司治疗可取得较好的疗效。