Assessing and Reporting HR-QoL in Children With Cystic Fibrosis: A Systematic Literature Review of Clinical Trials

OBJECTIVES: The advancements in cystic fibrosis (CF) treatment have led to higher survival rates, underscoring significance of health-related quality of life (HR-QoL) assessment as key aspect of CF care. The impact of CF on HR-QoL is less explored in paediatric patients and needs proper understanding on status quo and challenges with respect to HR-QoL in children with CF. We aimed to assess HR-QoL parameters utilised in clinical trial settings among paediatric CF patients. We also searched clinicaltrials.gov registry to determine extent of inclusion of HR-QoL as clinical endpoint among registered paediatric trials.

METHODS: Systematic search was conducted in PubMed and Google Scholar (using evidence synthesis tool – MaiA). The search was restricted to study type as clinical trials (CTs), between Jan2014-Jun2024 for data recency. Two independent reviewers performed data extraction using pre-structured template to capture details including study and patient characteristics, QoL measurement tool and its domains.

RESULTS: A total of 210 articles were obtained from database search, of which 24 (11.4%) articles were included for analysis. The most used method for assessing HR-QoL was Cystic Fibrosis Questionnaire-Revised (CFQ-R) (79%) followed by Pediatric Quality of Life Inventory (PedsQL) (16.7%). Only 2 out of 24 CTs used HR-QoL as primary outcome measure. Clinical parameters such as forced expiratory volume, body mass index, and nutritional status were associated with HR-QoL. Further, registry findings showed 25 of 91 CTs measured HR-QoL as clinical endpoints, and CFQ-R as most widely used assessment method (76% CTs).

CONCLUSIONS: Although HR-QoL is recognised as important parameter, the use of HR-QoL as an outcome measure is limited in clinical settings. This review highlights current scenario and suggest inclusion of HR-QoL while designing clinical trials for CF to draw valid inferences on treatment.