Heidendael Josephine F, Den Boer Suzanne L, Wildenbeest Joanne G, Dalinghaus Michiel, Straver Bart, Pajkrt Dasja
1Department of Cardiology,VU University Medical Center,Amsterdam,The Netherlands.
2Department of Pediatric Cardiology,Erasmus Medical Center,Sophia Children's Hospital,Rotterdam,The Netherlands.
Cardiol Young. 2018 Jan;28(1):46-54. doi: 10.1017/S1047951117001561. Epub 2017 Aug 11.
Dilated cardiomyopathy is a rare but serious disorder in children. No effective diagnostic or treatment tools are readily available. This study aimed to evaluate the efficacy of intravenous immunoglobulins in children with new onset dilated cardiomyopathy. Methods and results In this retrospective cohort study, 94 children with new onset dilated cardiomyopathy were followed during a median period of 33 months. All patients with secondary dilated cardiomyopathy - for example, genetic, auto-immune or structural defects - had been excluded. Viral tests were performed in all patients and 18 (19%) children met the criteria for the diagnosis "probable or definite viral myocarditis". Intravenous immunoglobulins were administered to 21 (22%) patients. Overall transplant-free survival was 75% in 5 years and did not differ between treatment groups. The treatment was associated with a higher recovery rate within 5 years, compared with non-treated children (70 versus 43%, log rank=0.045). After correction for possible confounders the hazard ratio for recovery with intravenous immunoglobulins was not significant (hazard ratio: 2.1; 95% CI: 1.0-4.6; p=0.056). Administration of intravenous immunoglobulins resulted in a greater improvement in the shortening fraction of the left ventricle.
In our population of children with new onset dilated cardiomyopathy, of either viral or idiopathic origin, intravenous immunoglobulins were administered to a minority of the patients and did not influence transplant-free survival, but were associated with better improvement of systolic left ventricular function and with better recovery. Our results support the concept that children with new onset dilated cardiomyopathy might benefit from intravenous immunoglobulins.
扩张型心肌病在儿童中较为罕见但病情严重。目前尚无有效的诊断或治疗手段。本研究旨在评估静脉注射免疫球蛋白对新发扩张型心肌病患儿的疗效。
在这项回顾性队列研究中,对94例新发扩张型心肌病患儿进行了为期33个月的中位随访。所有继发于例如遗传、自身免疫或结构缺陷等因素的扩张型心肌病患者均被排除。对所有患者进行了病毒检测,18例(19%)儿童符合“可能或确诊病毒性心肌炎”的诊断标准。21例(22%)患者接受了静脉注射免疫球蛋白治疗。5年总体无移植生存率为75%,治疗组之间无差异。与未治疗的儿童相比,该治疗在5年内的恢复率更高(70%对43%,对数秩检验=0.045)。在校正可能的混杂因素后,静脉注射免疫球蛋白恢复的风险比无统计学意义(风险比:2.1;95%可信区间:1.0 - 4.6;p = 0.056)。静脉注射免疫球蛋白使左心室缩短分数有更大改善。
在我们的新发扩张型心肌病患儿群体中,无论病因是病毒感染还是特发性,少数患者接受了静脉注射免疫球蛋白治疗,其对无移植生存率无影响,但与左心室收缩功能更好的改善及更好的恢复相关。我们的结果支持新发扩张型心肌病患儿可能从静脉注射免疫球蛋白中获益这一观点。