儿童Evans综合征8例临床分析

Clinical observation of 8 children with Evans syndrome

目的探讨儿童Evans综合征的诊断及治疗。方法 2004年3月-2013年7月郑州大学第一附属医院儿科共收治儿童Evans综合征8例,分析临床特征及治疗效果。结果 8例患儿中6例男性,2例女性,首次诊断中位年龄1岁(4个月~3岁10个月)。8例患儿中,5例直接抗人球蛋白试验(DAT)阳性(62%);3例行血小板抗体(HPA)检查,均阳性(100%);8例患儿均行骨髓穿刺检查,提示增生性贫血5例(62%),免疫性血小板减少症2例(25%),骨髓象正常1例(13%);3例行细胞免疫功能检查,均有B淋巴细胞(CD19^+)比例增高(100%),其中2例合并有Th淋巴细胞(CD3^+CD4^+)比例下降(67%)。8例患儿中,5例同时接受糖皮质激素及丙种球蛋白(IVIG)治疗,IVIG用量均为500mg/(kg·d)×5d,糖皮质激素应用地塞米松磷酸钠/甲泼尼龙琥珀酸钠/泼尼松,4例达CR或PR(总有效率80%),1例无效;单一应用IVIG和糖皮质激素治疗各1例,均无效(0/2);1例仅经过抗感染治疗后达PR。中位随访时间18(14~24)个月,随访期间2例复发,最长维持缓解期为3个月。截止至末次随访时间,2例死亡,3例持续CR,3例失访。结论 Evans综合征难治且易复发,患儿可因出血或感染而死亡。初发病例激素与IVIG联用疗效肯定;复发或耐药病例,长期单用激素疗效不满意,且易合并严重感染,建议多药联合治疗。

Objective To improve diagnostic method and treatment protocol, analyze the clinical characteristics and treatment results of childhood Evans syndrome in the first affiliated hospital of Zhengzhou University. Methods In this retrospective study, we evaluated the clinical features and the effects of first- line therapy on the clinical course of childhood patients with Evans syndrome in our center between 2004- 2013. Results Eight（6 males, 2 females）patients were included, the median age was 1 year（4months-3. 8years）. At onset, 75% patients had dermal ecchynosis, 50% patients had icterus, 37.5% patients had epistaxis and 25% patients had hemoglobinuria. At physical examination, 7-5% patients had hepatosplenomegaly. Biologically, there was 2 patients accompanied with neutropenia, hyper bilirubin was found in 62.5% of cases;Direct Coombs test was positive for IgG ＋ C3 ＋ IgM ＋ IgA （2 cases）, IgG ＋ C3 （2 cases）, IgM （ 1 case） and was negative in 3 cases ;3 of the patients took a platelet antibody test, and all of them were positive （ 100% ） ; bone marrow test showed hyperplasia anemia in 5 patients, megakaryocyte maturation disorder in 2 patients and 1 was normal. 3 tested patients showed an increase in CD19^＋ B lymphoeyte, and 2 patients also showed a decline of CD3^＋ CD4^＋ Th eell. 7 patients initially received first-line therapy（ IV steroids and/or IVIG） ,while one patient only reeeived anti-infeetive therapy. CR and PR were reaehed in 62.5% patients, relapse oecurred within median 18 months in 25% patients, 2 eases died, 2 eases got eontinued complete remission, unfortunatley 3 eases were lost to follow-up. Conclusions In view of the high relapse rate and poor therapeutie response in our study, we suggest that medieal treatment with eombination agents （IVIG, steroids, Rituximab, Myeophenolate mofetil）may provide a useful approach to children with Evans syndrome.