摘要目的 观察脂质体阿霉素联合依托泊苷和大剂量甲泼尼龙(DEP)方案挽救治疗成人难治性噬血细胞综合征(HLH)患者的有效性.方法 以41例难治性HLH患者为研究对象,采用DEP方案治疗2周和4周后,参照美国中西部协作组制订的标准进行疗效评价.结果 41例患者中,男28例,女13例,中位年龄31(18～62)岁.总反应率(ORR)为78.1％(32例),其中完全缓解(CR)率为29.3％(12例),部分缓解(PR)率为48.8％(20例).41例患者中有8例未找到引起HLH的原发病(CR 5例,PR2例);其余33例患者中原发性HLH 1例(CR 1例),淋巴瘤相关HLH 20例(CR 3例,PR 12例),EBV相关HLH 12例(CR 3例,PR 6例).对DEP方案无应答的9例患者均在开始挽救治疗后的2～4周内死亡;达到PR和CR的32例患者在诱导缓解后积极针对原发病治疗,包括化疗、异基因造血干细胞移植、脾脏切除等,其中20例患者病情持续缓解.结论 单臂研究结果提示,DEP方案可能是一种有效的用于成人难治性HLH患者的挽救治疗方案.

abstractsObjective To investigate the efficacy of liposomaldoxorubicin together with etoposide and high dose methylprednisolone (DEP) as a salvage therapy for adult refractory hemophagocytic lymphohistiocytosis (HLH).Methods Total 41 patients with refractory HLH were enrolled in this study.The efficacy of treatment with DEP regimen after 2 and 4 weeks were evaluated according to the United States Midwest Cooperative HLH Group.Results Of 41 refractory HLH patients,28 were males and 13 females.The median age was 31 (18-62) years old.The overall response rate (ORR) was 78.1％ (32/41),including 12 patients (29.3％) achieved complete remission (CR) and 20 (48.8％) achieved partial remission (PR).The underlying disease of HLH were identified in 33 patients,including 1 case of primary HLH (CR),20 cases of lymphoma associated HLH and 12 cases of EBV associated HLH.There were still 8 cases with unknown underlying disease.The 9 patients who had no response to DEP died within 2 to 4 weeks after salvage therapy.Twenty of the 32 patients who achieved PR or CR survived to undergo subsequent chemotherapy,allogenic hematopoietic stem cell transplantation (allo-HSCT) or splenectomy.Conclusion The single-arm study suggested that DEP regimen appeared to be an effective salvage protocol for adult patients with refractory HLH.