Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data
ABSTRACT
The CLN2 Clinical Rating Scale assesses disease progression in CLN2 disease, a rare neurodegenerative disorder. To validate the scale, it was compared to the Pediatric Quality of Life Inventory (PedsQL) using clinical trial data on cerliponase alfa treatment for CLN2 disease.
Linear regression and mixed effects models were used to study the relationship between the Clinical Rating Scale and PedsQL in a clinical trial involving 23 children with CLN2 disease treated with cerliponase alfa for at least 96 weeks.
Correlations between the Clinical Rating Scale domains were low. However, a significant relationship was found between PedsQL and the Clinical Rating Scale, particularly in the motor domain, suggesting that motor function significantly impacts patients’ quality of life.
It was concluded that each domain of the Clinical Rating Scale provides unique information about the disease state. While the motor domain had the highest correlation with PedsQL, other domains did not show a strong association, indicating the need for additional disease-specific measures to fully capture the quality of life impact of CLN2 disease.
TRIAL REGISTRATION: NCT01907087, NCT02485899.
PMID: 38776275 | DOI: 10.1371/journal.pone.0302382
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