The Impact of Raising Children with Barth Syndrome on Parental Health-Related Quality of Life and Family Functioning: Preliminary Reliability and Validity of the PedsQL™ Family Impact Module

Objective. This study examined the preliminary reliability and validity of the PedsQL™ Family Impact Module (PedsQL FIM) in families of children with Barth syndrome (BTHS). Method. A total of 72 parents with children or youth between the ages of 5 and 19 participated in this study. Thirty-three parents of children with BTHS and 39 parents of unaffected children completed the PedsQL FIM and a demographic information form. Internal consistency reliability and item-total correlations were calculated to test the reliability of the PedsQL FIM. Construct validity was examined using the known-groups method. We estimated the mean score differences of the PedsQL FIM between the two groups using three different models, including unadjusted, multivariate regression, and propensity score matching with inverse probability of treatment weighting (PS-IPTW) models. Results. The Cronbach’s alpha coefficients were greater than 0.70 for all scales of the PedsQL FIM, except for the communication scale. The item-total correlations were significant for all scales with moderate to high correlations ($p<.05$). In construct validity, the mean scores of the PedsQL FIM between the two groups were significantly different ($p<.05$) for all scales and total score in the unadjusted and PS-IPTW models. However, in the multivariate regression model, the family relationships scale was not significant between the two groups. Conclusion. The PedsQL FIM demonstrated adequate measurement properties of preliminary reliability and validity in assessing the impact of children with BTHS on parental health-related quality of life (HRQoL) and family functioning. Further research needs to be conducted to examine the psychometric properties of the PedsQL FIM with a large sample of BTHS and with other pediatric rare diseases.