大剂量甲泼尼龙联合依托泊苷挽救治疗成人噬血细胞性淋巴组织细胞增多症

Salvage therapy of adult refractory/recurrence hemophagocytic lymphohistiocytosis with high-dose methylprednisolone plus etoposide

目的：探讨大剂量甲泼尼龙联合依托泊苷方案挽救治疗成人噬血细胞综合征噬血细胞性淋巴组织细胞增多症的有效性及安全性。方法：18例难治/复发成人噬血细胞性淋巴组织细胞增多症患者,应用甲泼尼龙（20mg/kg,第1～3天）联合依托泊苷（100mg/m2,1次/周治疗）;对疗效、安全性及生存情况进行回顾性分析。结果：18例患者中难治13例,复发5例,共接受20例次大剂量甲泼尼龙联合依托泊苷治疗。15例可评价疗效患者,17例次治疗总体反应率70.5%,其中部分缓解11例,接近完全缓解1例。各项临床/实验室指标反应率：发热82.3%,铁蛋白58.8%,谷丙转氨酶41.2%,中性粒细胞29.4%。6例患者可评价最佳疗效持续时间,中位疗效持续时间10d（7～24d）。不良反应主要为水钠潴留（5/20）、消化道出血（4/20）、感染（4/20）、血糖升高（2/20）。中位随访4.5周（0.5～38周）,总生存率33.3%。结论：大剂量甲泼尼龙联合依托泊苷方案治疗难治/复发成人噬血细胞性淋巴组织细胞增多症具有良好的反应率及安全性,但疗效持续时间短,需及早序贯给予病因治疗。

Objective：To elucidate the curative effects and toxicity of high-dose methylprednisolone plus etopo- side in salvage therapy of adult refractory/recurrence hemophagocytic lymphohistiocytosis （HLH）. Method： Reviewed the charts of 18 adult patients who received Methylprednisolone 20 mg/kg d1～3 plus Etoposide 100 mg/ m2 qw for the treatment of refractory/recurrence HLH at our center. Result： 18 patients,including 13 refractory and 5 recurrence,received 20 courses of salvage therapy in total. Of 15 evaluable patients the overall response rate was 70.5 % in 17 courses treatment,out of 11 cases partial remission, 1 case almost complete remission. The clinical and laboratory response rate ： Fever response 82.3 %, ferritin response 58.8 %, transaminase response 41.2 %, neutrophil response 29.4%. The duration of best response was 10 days （7～24 days） in 6 evaluable patients. Ad- verse events included water sodium retention （5/20）,gastrointestinal bleeding （4/20）,infection （4/20）,hypergly- cemia （2/20）. After a median follow up of 4.5 weeks （0.5～38 weeks） ,the overall survival was 33.3%. Conclusion： High dose methylprednisolone plus etoposide appears to be an effective and safety salvage regimen for refractory/ recurrence HLH,leading to improvement and survival to primary disease therapy in many patients.