Psychometric Validation of the CLN2 Quality of Life Questionnaire in Participants with CLN2 Disease Treated with Cerliponase Alfa.

ABSTRACT:

Objectives

This study evaluated the psychometric properties of the ceroid lipofuscinosis type 2 Quality of Life (CLN2 QoL) questionnaire.

Methods

Data from children with CLN2 disease aged 3-16 years receiving cerliponase alfa in the BMN 190-201 and BMN 190-202 clinical studies, collected via purposive sampling, were used to assess convergent and divergent validity, internal consistency and reliability. The clinically important difference (CID) was estimated with distribution- and anchor-based methods. Descriptive and inferential statistical analyses were conducted using IBM SPSS.

Results

CLN2 QoL data of 22 participants were analysed. Ceiling effects were observed in 22 items (35% threshold); no floor effects were observed. Internal consistency analysis showed good re

SUBMITTER: Due C 

PROVIDER: S-EPMC11593549 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature