α-干扰素联合短疗程糖皮质激素治疗儿童持续性和慢性免疫性血小板减少症

α-Interferon in combination with a short course of glucocorticoid for the treatment of persistent,chronic immune thrombocytopenia in children

目的研究α-干扰素联合短疗程糖皮质激素治疗儿童持续性和慢性免疫性血小板减少症(ITP)的有效性、安全性及其可能的作用机制。方法 28例于2014年6月-2016年10月在中山大学孙逸仙纪念医院儿科就诊的持续或慢性ITP患儿参与此研究,他们被随机按2.5:1分为治疗组和对照组,治疗组20例患儿予以α-干扰素联合短疗程糖皮质激素治疗,对照组8例患儿单用短疗程糖皮质激素治疗。治疗组根据外周血淋巴细胞亚群进一步分为调节T细胞下降B细胞上升(Treg↓B↑)亚组和调节T细胞正常B细胞上升(Treg-B↑)亚组。组间差异采用成组t检验,组内差异采用配对t检验;采用Krusal-Wallis秩和检验分析治疗后的疗效对比。结果治疗后,治疗组患儿的血小板计数明显升高,相比对照组差异有显著性(P<0.001)。治疗组20例患儿用药前PLT的平均值为(17.90±6.43)×10~9/L,治疗后PLT的峰值平均为(117.45±50.14)×10~9/L,中位起效时间为26.7(9~72)天,中位疗效维持时间为10.5(6~34)个月。治疗24周后,Treg↓B↑亚组的有效率高于Treg-B↑亚组(P<0.05);Treg↓B↑亚组治疗后的外周血Treg%、CD4^+%、CD4^+/CD8^+较治疗前升高(P<0.05)。不良反应为1例出现流感样症状。结论α-干扰素联合短疗程糖皮质激素治疗儿童持续性和慢性ITP既能获得较短的起效时间,又能较长时间维持疗效,特别对于Treg↓B↑类型,其机制可能为促使淋巴细胞亚群达到平衡。

Objective To evaluate the efficiency,safety and possible machanism of α-interferon in combination with a short course of glucocorticoid for the treatment of persistent,chronic immune thrombocytopenia in children.Methods 28 children with persistent,chronic immune thrombocytopenia in the department of pediatrics in Sun Yat-sen Memorial Hospital of Sun Yat-sen University between June 2014 and October 2016 were enrolled in this study,they were randomly divided into treatment group and control group according to 2.5:1,20 children in the treatment group were treated with a-interferon in combination with a short course of glucocorticoid,while 8 children in the control group were treated with a short course of glucocorticoid alone. The treatment group was further divided into Treg cell descending B cell ascending(Treg ↓B↑)subgroup and Treg cell normal B cell ascending(Treg-B ↑)subgroup according to the peripheral blood lymphocyte subsets.Group t test was used for inter-group differences,and paired t test was used for intra-group differences,Krusal-wallis rank sum test was used to analyze the efficiency after treatment.Results After treatment,platelet count in the treatment group was significantly higher than that in the control group(P<0.001).In the treatment group,the peak platelet counts[(117.45±50.14)×10~9/L]after treatment were significantly higher than that[(17.90±6.43)×10~9/L]before treatment(P<0.001),the median onset time was 26.7(9-72)days,the median efficiency maintenance time was 10.5(6-34)months;at week 24,the therapeutic efficiency of Treg ↓B↑subgroup was significantly higher than that of Treg-B ↑ subgroup(P<0.05);in the Treg↓ B↑subgroup,the percentage of Treg cells,CD4^+cells,the ratio of CD4^+/CD8^+ were significantly higher than that before treatment(P<0.05).Influenza-like symptom was observed in 1 patient.Conclusions For children with persistent,chronic immune thrombocytopenia,a-interferon in combination with a short course of glucocorticoid can get shorter onset time,and can maintain therapeutic efficiency for a long time,especially for Treg ↓B↑ type,and patients were well tolerated,its mechanism may balance Th cell subsets and Treg cells.