21例儿童重度温抗体型自身免疫性溶血性贫血临床治疗分析

Clinical analysis of 21 children with severe warm autoimmune hemolytic anemia

目的探讨重度温抗体型自身免疫性溶血性贫血(warm autoimmune hemolytic anemia,w-AIHA)的临床特征、疗效及临床干预策略选择。方法回顾性分析2007年6月至2019年3月在首都儿科研究所附属儿童医院血液科住院治疗的21例w-AIHA患儿的临床特点、治疗方案和疗效。结果21例重度w-AIHA患儿平均年龄8.0(2.5,20.0)个月,随访时间33.0(18.5,110.0)个月。10例(47.6%)患儿血红蛋白低于30 g/L,5例(23.8%)患儿表现为Evans综合征,5例(23.8%)患儿为继发性病例,9例(42.8%)患儿有感染史,2例患儿摄入花粉诱发;溶血危象5例(26.3%),12例(57.1%)存在配血困难,8例(38.1%)入住ICU,5例(23.8%)出现休克。所有患儿均使用了皮质类固醇和静脉用丙种球蛋白,16例(76.2%)患儿予以二线方案(环磷酰胺、利妥昔单抗等)治疗,15例完全缓解,3例部分缓解,3例无效及死亡。结论感染仍是儿童重度w-AIHA病例的重要诱发因素,继发性病例比例高,继发病因以免疫缺陷病为主,溶血危象发生率高,且存在配血、输血困难,成功输血为抢救成功的关键因素。重度w-AIHA患儿应早期入住ICU密切监测,利妥昔单抗需提前应用,保证成功输血。

Objective To explore the clinical manifestation,efficacy and treatment strategies in patients with severe warm autoimmune hemolytic anemia(w-AIHA).Methods A total of 21 patients with w-AIHA who were hospitalized in Children′s Hospital of Capital Institute of Pediatrics from June 2007 to March 2019 were included,and the clinical characteristics,treatment strategies and responses were retrospectively analyzed.Results A total of 21 children with severe w-AIHA had an average age of 8.0(2.5,20.0)months and a follow-up time of 33.0(18.5,110.0)months.In 10(47.6%)cases,the hemoglobin levels were lower than 30 g/L.Evans′syndrome was diagnosed in five(23.8%)cases.Five(23.8%)cases were secondary cases.Nine(42.8%)cases had a previous infection history and two cases were pollen-induced.Five(23.8%)cases had hemolytic crisis.A total of 12(57.1%)cases had cross-matching difficulty.Eight(38.1%)cases were admitted to the ICU,and five(23.8%)cases had shock.All children received corticosteroids and intravenous immunoglobulin,16(76.2%)cases were treated with second-line regimens(cyclophosphamide and rituximab,etc.),15 cases had complete response,three cases had partial response and three cases had no response and died.Conclusion Infection is an important predisposing factor in children with severe w-AIHA,and secondary cases have a higher proportion,mainly caused by immunodeficiency disease.Patients tend to have a high incidence of hemolytic crisis and have difficulty in matching and transfusion.Therefore,transfusion is the key for successful rescue.It is suggested that children with severe w-AIHA require ICU admission for early monitoring and rituximab should be applied in advance to ensure successful transfusion.