Purpose: Barth syndrome is an X-linked rare disorder that typically affects only males. This study investigates 1) agreement between child self-reports and parent proxy-reports of HRQoL in boys with Barth syndrome and 2) relationship between parental HRQoL and parent proxy-reports of HRQoL for the child.
Materials and methods: Twenty-eight boys with Barth syndrome and their parents participated in this study. The PedsQL™ 4.0 and the PedsQL™ Family Impact Module were used to measure HRQoL of the boys, and the parents' HRQoL, respectively. The Intraclass Correlation Coefficient was used to test agreement between the child self-reports and parent proxy-reports of HRQoL. The Spearman correlation coefficient was used to test the relationship between parental HRQoL and parent proxy-reports of HRQoL for the child.
Results: The agreement between the child self-reports and the parent proxy-reports showed moderate-to-good agreement. Higher parental HRQoL was significantly related to higher ratings of the parents on their children's HRQoL (p < .05).
Conclusions: This study broadens understanding of HRQoL of boys with Barth syndrome using both child self-reports and parent proxy-reports. The findings indicate that the parent proxy-report of HRQoL should be used in conjunction with the child self-report when making client-centered health decisions.
Keywords: Barth síndrome; HRQoL; Health-related quality of life; Proxy-report; Rare Diseases; Self-report.