den Boer Susanna L, Baart Sara J, van der Meulen Marijke H, van Iperen Gabriëlle G, Backx Ad P, Ten Harkel Arend D, Rammeloo Lukas A, du Marchie Sarvaas Gideon J, Tanke Ronald B, Helbing Willem A, Utens Elisabeth M, Dalinghaus Michiel
1Department of Pediatrics,Division of Pediatric Cardiology,Sophia Children's Hospital,Rotterdam,The Netherlands.
2Department of Cardiology,Erasmus University Medical Center,Rotterdam,Erasmus University Medical Center,Rotterdam,The Netherlands.
Cardiol Young. 2017 Aug;27(6):1194-1202. doi: 10.1017/S1047951116002833. Epub 2017 Mar 14.
Dilated cardiomyopathy in children causes heart failure and has a poor prognosis. Health-related quality of life in this patient group is unknown. Moreover, results may provide detailed information of parents' sense of their child's functioning. We hypothesised that health-related quality of life, as rated by parents, and the paediatric heart failure score, as assessed by physicians, have both predictive value on outcome. Methods and results In this prospective study, health-related quality of life was assessed by parent reports: the Infant Toddler Quality of Life questionnaire (0-4 years) or Child Health Questionnaire-Parent Form 50 (4-18 years) at 3-6-month intervals. We included 90 children (median age 3.8 years, interquartile range (IQR) 0.9-12.3) whose parents completed 515 questionnaires. At the same visit, physicians completed the New York University Pediatric Heart Failure Index. Compared with Dutch normative data, quality of life was severely impaired at diagnosis (0-4 years: 7/10 subscales and 4-18 years: 8/11 subscales) and ⩾1 year after diagnosis (3/10 and 6/11 subscales). Older children were more impaired (p<0.05). After a median follow-up of 3 years (IQR 2-4), 15 patients underwent transplantation. Using multivariable time-dependent Cox regression, "physical functioning" subscale and the Heart Failure Index were independently predictive of the risk of death and heart transplantation (hazard ratio 1.24 per 10% decrease of predicted, 95% confidence interval (CI) 1.06-1.47 and hazard ratio 1.38 per unit, 95% CI 1.19-1.61, respectively).
Physical impairment rated by parents and heart failure severity assessed by physicians independently predicted the risk of death or heart transplantation in children with dilated cardiomyopathy.
儿童扩张型心肌病可导致心力衰竭，预后较差。该患者群体与健康相关的生活质量尚不清楚。此外，研究结果可能会提供有关父母对其孩子功能状况感受的详细信息。我们假设，父母评定的与健康相关的生活质量以及医生评估的小儿心力衰竭评分对预后均具有预测价值。
在这项前瞻性研究中，通过父母报告评估与健康相关的生活质量：每3至6个月使用婴幼儿生活质量问卷（0至4岁）或儿童健康问卷家长版50（4至18岁）。我们纳入了90名儿童（中位年龄3.8岁，四分位间距（IQR）0.9至12.3），其父母完成了515份问卷。在同一次就诊时，医生完成纽约大学小儿心力衰竭指数评估。与荷兰的标准数据相比，诊断时（0至4岁：10个分量表中的7个，4至18岁：11个分量表中的8个）以及诊断后≥1年（10个分量表中的3个，11个分量表中的6个）生活质量严重受损。年龄较大的儿童受损更严重（p<0.05）。中位随访3年（IQR 2至4）后，15名患者接受了移植。使用多变量时间依赖性Cox回归分析，“身体功能”分量表和心力衰竭指数可独立预测死亡和心脏移植风险（预测值每降低10%的风险比为1.24，95%置信区间（CI）1.06至1.47；每单位的风险比为1.38，95%CI 1.19至1.61）。
父母评定的身体损伤和医生评估的心力衰竭严重程度可独立预测扩张型心肌病患儿的死亡或心脏移植风险。
