Development and pilot testing of the quality of life of parents of children with achondroplasia questionnaire.
The QOLA questionnaire shows promise in assessing the quality of life for parents of children with achondroplasia, but further validation is necessary.
Where it sits
this study against the rest of the vosoritide corpusSummary and findings
This study developed and pilot tested the Quality of Life of Parents of Children with Achondroplasia (QOLA) questionnaire. It involved 50 parents across Germany, Italy, and Portugal to assess various dimensions of well-being. The final version included 63 items across eight domains.
Abstract
<h4>Background</h4>Parents of children with achondroplasia face sustained caregiving demands that may affect multiple dimensions of well-being. Despite growing recognition of these challenges, no validated, condition-specific instrument exists to assess the quality of life (QoL) of parents of children with achondroplasia. This study aimed to develop, and pilot test the Quality of Life of Parents of Children with Achondroplasia (QOLA) questionnaire.<h4>Methods</h4>QOLA was developed using a multi-phase mixed-methods design in accordance with established standards for developing self-reported outcome measures for caregivers and parents. Phase 1 comprised semi-structured qualitative interviews with 17 parents of children with achondroplasia to identify relevant QoL domains and language. Interview data were analysed using qualitative content analysis and informed systematic item generation (Phase 2). Conceptual structure was examined through researcher-led card sorting (Phase 3) and two rounds of international card sorting following translation (Phase 4). The resulting 63-item questionnaire across eight domains was pilot-tested in a cross-sectional, multi-country study with embedded cognitive debriefing in Germany, Italy, and Portugal (total N = 50).<h4>Results</h4>The final pilot version of QOLA comprised 63 items across eight domains covering healthcare experiences, challenges and support, physical health, mental health, social life and relationships, coping, family and daily life, and worries and future concerns. Item-level missing data were minimal, and no pronounced floor or ceiling effects were observed. Internal consistency was acceptable to good for domains (α = 0.624-0.821) and good for the total scale (α = 0.798). Inter-domain correlations were generally moderate to strong. Cognitive debriefing was highly acceptable and relevant across countries, with some suggestions for further refinement.<h4>Conclusions</h4>QOLA shows strong preliminary evidence of acceptability and internal consistency and addresses a key measurement gap in achondroplasia research. Further large-scale psychometric validation is warranted.
Background
Parents of children with achondroplasia experience significant caregiving demands that can impact their quality of life (QoL). Prior to this study, there was no validated instrument specifically designed to measure the QoL of these parents. This research is important as it addresses a gap in the assessment tools available for this population.
Methods
The study employed a multi-phase mixed-methods design to develop the QOLA questionnaire. Phase 1 included semi-structured qualitative interviews with 17 parents to identify relevant QoL domains. In Phase 2, systematic item generation was informed by qualitative analysis. Phase 3 involved researcher-led card sorting, and Phase 4 included international card sorting after translation. The pilot test was conducted with a total of 50 parents across Germany, Italy, and Portugal.
Results
The pilot version of QOLA included 63 items across eight domains. The internal consistency for the total scale was α = 0.798, indicating good reliability. Domain-specific internal consistency ranged from α = 0.624 to 0.821. There were moderate to strong inter-domain correlations, and cognitive debriefing indicated high acceptability across countries.
Interpretation
The findings suggest that QOLA is a promising tool for assessing the QoL of parents of children with achondroplasia, filling a significant measurement gap. However, the effect sizes, while statistically significant, may not yet be clinically meaningful without further validation. The small sample size and lack of extensive psychometric testing limit the conclusions that can be drawn from this pilot study.
Key findings
- 63-item questionnaire developed across eight domains.
- Internal consistency for total scale α = 0.798.
- Internal consistency for domains α = 0.624-0.821.
- Moderate to strong inter-domain correlations observed.
- Minimal item-level missing data reported.
Limitations
- small sample size n=50
- pilot study, further validation needed
- no long-term follow-up reported