Evaluation of the Health-Related Quality of Life and Mental Health of Parents With Children and Adolescents With a Rare Disease Based on the Results of a Randomized Controlled Trial to Investigate a Family-Based Intervention and an Online Intervention for Affected Families (CARE-FAM-NET)

Abstract

Parents caring for children with rare diseases are more impaired regarding health-related quality of life (HRQoL) and mental health than healthy controls and norm data. To address the research gap in psychological care for these parents, this study evaluates the effectiveness of two family-based interventions. The children affected by rare disease and their families network (CARE-FAM-NET) study is a multicenter randomized controlled 2 × 2 factorial trial for affected families with children (0-21 years). This paper focuses on evaluating the impact of two interventions, one face-to-face (CARE-FAM) and one online (WEP-CARE), on the HRQoL and mental health of parents. One thousand, one hundred sixty-eight parents participated: TAU = 291, CARE-FAM = 296, WEP-CARE = 300, and CARE-FAM + WEP-CARE combined = 281. Data were collected at four time points over a period of 18 months using standardized questionnaires. The results had to be interpreted exploratively. The results indicate that there are no clinically relevant differences in the parents' HRQoL and mental health between the treatment groups. However, time-dependent differences in the intervention effects for WEP-CARE were observed. Although the results did not show clear relevant differences between conditions, trends in improvement in HrQoL and mental health were identified. CARE-FAM shows a greater reduction in parental distress and WEP-CARE shows a greater distortion of distress, particularly at T3 and T4. Given the exploratory nature of this study, it highlights the urgent need for further confirmatory research in this area.

Keywords: family intervention; health‐related quality of life; mental health; parents; rare disease.

FIGURE 1

Description of the study population, number of randomized participants, allocation to three intervention groups (CARE‐FAM, WEP‐CARE, CARE‐FAM + WEP‐CARE) and one control group (TAU). Numbers of participants at every measurement point after 6, 12, and 18 m. “Family missing” indicated that the entire family did not provide any information at that time.