BACKGROUND The evidence regarding patient related outcomes in children with infrequent congenital heart defects (I-CHD) is very limited. We sought to measure quality of life (QoL) in children with I-CHD, and secondarily, to describe QoL changes after one-year of follow-up, self-reported by children and through their caregivers' perspective. METHODS We assembled a cohort of children diagnosed with an I-CHD in a cardiovascular referral center in Colombia, between August 2016 and September 2018. At baseline and at one-year follow-up, a clinical psychology assessment was performed to establish perception of QoL. The Pediatric Quality of Life Inventory (PedsQL) 4.0 scale was used in both general and cardiac modules for patients and for their caregivers. We used a Mann-Whitney U test to compare scores for general and cardiac modules between patients and caregivers, while a Wilcoxon test was used to compared patients' and caregivers' baseline and follow-up scores. Results are presented as median and interquartile range. RESULTS To date, QoL evaluation at one-year follow-up has been achieved in 112/157 patients (71%). Self-reported scores in general and cardiac modules were higher than the QoL perceived through their caregivers, both at baseline and after one-year of follow-up. When compared, there was no statistically significant difference in general module scores at baseline between patients (median = 74.4, IQR = 64.1-80.4) and caregivers scores (median = 68.4, IQR = 59.6-83.7), p = 0.296. On the contrary, there was a statistical difference in baseline scores in the cardiac module between patients (median = 79.6, IQR = 69.7-87.4) and caregivers (median = 73.6, IQR = 62.6-84.3), p = 0.019. At one-year of follow-up, scores for the general module between patients (median = 72.8, IQR = 59.2-85.9) and caregivers (median = 69.9, IQR = 58.1-83.7) were not statistically different (p = 0.332). Finally, a significant difference was found for cardiac module scores between patient (median = 75.0, IQR = 67.1-87.1) and caregivers (median = 73.1, IQR = 59.5-83.8), p = 0.034. CONCLUSIONS QoL in children with I-CHD can be compromised. However, children have a better perception of their QoL when compared with their caregivers' assessments. To provide high-quality care, besides a thorough clinical evaluation, QoL directly elicited by the child should be an essential aspect in the integral management of I-CHD.

中文翻译：

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先天性心脏病少见的儿童的生活质量：队列研究，为期一年的随访。

背景技术关于不常见的先天性心脏病（I-CHD）患儿中与患者有关的结局的证据非常有限。我们试图测量患有I-CHD的儿童的生活质量（QoL），其次，通过儿童的自我报告以及他们的看护者的观点，描述了一年随访后的QoL变化。方法我们于2016年8月至2018年9月在哥伦比亚的心血管转诊中心聚集了一群被诊断为I-CHD的儿童。在基线和一年的随访中，进行了临床心理学评估，以建立QoL知觉。儿科患者生活质量量表（PedsQL）4.0量表用于患者及其护理人员的普通模块和心脏模块。我们使用Mann-Whitney U检验比较患者和护理人员之间的常规和心脏模块得分，而Wilcoxon检验用于比较患者和护理人员的基线和随访得分。结果以中位数和四分位间距表示。结果迄今为止，已经在112/157名患者中进行了为期一年的随访QoL评价（71％）。在基线和随访一年后，一般和心脏模块的自我报告得分均高于其护理人员的QoL。比较时，患者（中位数= 74.4，IQR = 64.1-80.4）和护理人员评分（中位数= 68.4，IQR = 59.6-83.7）之间，基线时一般模块评分在统计学上无显着差异，p = 0.296。反之，患者（中位数= 79.6，IQR = 69.7-87.4）和护理人员（中位数= 73.6，IQR = 62.6-84.3）之间，心脏模块的基线评分存在统计学差异（p = 0.019）。在一年的随访中，患者（中位数= 72.8，IQR = 59.2-85.9）和护理人员（中位数= 69.9，IQR = 58.1-83.7）之间的一般模块得分在统计学上没有差异（p = 0.332）。最后，发现患者（中位数= 75.0，IQR = 67.1-87.1）和护理人员（中位数= 73.1，IQR = 59.5-83.8）之间的心脏模块评分存在显着差异，p = 0.034。结论I-CHD患儿的生活质量可能会受到影响。但是，与照看者的评估相比，儿童对自己的QoL有更好的理解。为了提供高质量的护理，除了进行全面的临床评估外，