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目的寻找预测丙种球蛋白对于儿童扩张性心肌病疗效的生物标志物。方法对2000-2016年北京大学第一医院36例诊断为扩张型心肌病的患儿资料进行回顾分析,每例患儿除了给予对症治疗外均予丙种球蛋白治疗。以丙种球蛋白治疗后左心室射血分数(LVEF)绝对值上升≥10%或治疗后LVEF≥45%作为治疗有效的标准,将患儿分为丙种球蛋白治疗有效组和无效组,选取病程以及基线水平的肌钙蛋白(c Tn I)、肌酸激酶同工酶(CK-MB)、中性粒细胞计数/淋巴细胞计数、红细胞分布宽度(RDW)、血小板分布宽度(PDW)、心电图(ST-T改变,病理性Q波≥1项)、心脏MRI(早期强化、延迟强化、T2WI高信号≥1项)、抗磷脂抗体(狼疮抗凝物、抗心磷脂抗体、抗β2糖蛋白抗体≥1项)9项作为基线预测候选指标。结果丙种球蛋白治疗有效组与无效组比较,抗磷脂抗体阳性率的差异有统计学意义(P<0.05);抗磷脂抗体与丙种球蛋白疗效呈正相关(r=0.416,P<0.05),利用四格表计算其疗效预测灵敏度为64.3%,特异度为77.3%。结论抗磷脂抗体(狼疮抗凝物、抗心磷脂抗体、抗β2糖蛋白抗体≥1项为阳性)阳性的患儿丙种球蛋白疗效更好,提示抗磷脂抗体可以作为辅助临床预测丙种球蛋白治疗扩张型心肌病疗效的标志物。
Objective To find a biomarker to predict the therapeutic effect of gamma globulin on dilated cardiomyopathy in children. Methods The data of 36 children diagnosed as dilated cardiomyopathy in Peking University First Hospital from 2000 to 2016 were retrospectively analyzed. Each patient was treated with gamma globulin except for symptomatic treatment. The treatment of gamma globulin after left ventricular ejection fraction (LVEF) absolute increase ≥ 10% or LVEF ≥ 45% after treatment as a treatment effective standard, the children were divided into gamma globulin treatment effective group and invalid group, select the duration And baseline levels of cTn I, CK-MB, neutrophil count / lymphocyte count, RDW, PDW, electrocardiogram (ST-T changes, pathological Q-wave≥1), cardiac MRI (early enhancement, delayed enhancement, T2WI high signal≥1), antiphospholipid antibodies (lupus anticoagulant, anticardiolipin antibody, anti-β2 glycoprotein Antibody ≥ 1 item) 9 as a baseline prediction candidate. Results The positive rates of anti-phospholipid antibodies were significantly different between active and inactive groups (P <0.05). The anti-phospholipid antibodies were positively correlated with the therapeutic effect of gamma globulin (r = 0.416, P <0.05) Four grid table calculation of its efficacy prediction sensitivity of 64.3%, a specificity of 77.3%. Conclusion The anti-phospholipid antibodies (lupus anticoagulant, anticardiolipin antibodies, anti-β2 glycoprotein antibodies ≥ 1 positive) positive children with gamma globulin efficacy is better, suggesting that antiphospholipid antibodies can be used as a clinical prediction of gamma globulin treatment Dilated cardiomyopathy marker of efficacy.