Feasibility and acceptance of KIDSCREEN-52 as a screening tool for unmet needs in children with rare inflammatory diseases

Abstract

Background: Children living with rare diseases often face significant psychosocial challenges; recognizing and addressing these effectively is crucial. However, there is a paucity of comprehensive screening tools. This study aimed to assess the feasibility and acceptance of the comprehensive KIDSCREEN-52 tool in identifying unmet needs of children with rare inflammatory diseases and their caregivers and identifying factors associated with low health-related quality of life (HRQoL). Methods A prospective single-center study of consecutive pediatric patients aged 8-18 with inflammatory diseases and their caregivers was performed to assess HRQoL utilizing the multidimensional KIDSCREEN-52 self-report and proxy tool. The validated KIDSCREEN-52 tool is available in 13 languages with corresponding Norm Data. It captures HRQoL across 10 domains including 52 inquiries. HRQoL of children with rare inflammatory diseases was described utilizing the multidimensional KIDSCREEN-52 self-report and proxy tool. The feasibility and acceptability of KIDSCREEN-52 was determined using a simple, dichotomous three item acceptance tool. Factors associated with low self-reported HRQoL were explored. Results: A total of 104 participants, comprising 51 pediatric patients and their 53 caregivers, were included. The patients were 35 females and 16 males, with a median age of 16 years (range: 9-18). Among them, 25 (49%) had autoinflammatory diseases, 26 (51%) had rheumatic diseases. Mean values from self-reports and proxies were consistent with the Norm Data across all domains. Self-report and proxy assessments showed high-degree agreement. Patients reported lower HRQoL levels compared to the control population in nearly all domains. Both caregivers and children expressed strong acceptance of the KIDSCREEN-52 questionnaire's clarity, relevance, and adequacy. The overall completion rate was 75%, the mean completion time 17 minutes (range: 10-25). Factors associated with low HRQoL included female gender, adolescent age and evidence of a rheumatic disease. Conclusion: The KIDSCREEN-52 demonstrated promise as a feasible and accepted tool for capturing the HRQoL and identifying unmet needs in children with rare inflammatory diseases. Its comprehensiveness and the availability in multiple languages with corresponding Norm Data, offers a unique opportunity to implement strategies to identify and address HRQoL challenges of children with rare diseases in routine clinical care.