Rasch analysis of the NEI-VFQ-25: vision-related quality of life in Leber hereditary optic neuropathy after lenadogene nolparvovec gene therapy.

Authors

• Benson S Chen
• Stéphanie Perot
• Magali Taiel
• Patrick Yu-Wai-Man
• Mike Horton

Publication/Presentation Date

9-17-2025

Abstract

OBJECTIVES: This study aimed to evaluate the suitability of the National Eye Institute Visual Function Questionnaire (NEI-VFQ-25) for measuring vision-related quality of life (VRQoL) in patients with Leber hereditary optic neuropathy receiving lenadogene nolparvovec gene therapy in three Phase III randomised controlled clinical trials.

METHODS: VRQoL was assessed using the NEI-VFQ-25 at baseline (n=174) and 2 years after treatment (n=152). All participants received lenadogene nolparvovec in at least one eye. The scoring structure of the original NEI-VFQ-25 was evaluated for fit to the Rasch model, and a post hoc revision was created and psychometrically reevaluated. Stacked analysis was conducted to compare Rasch-revised scores at baseline and 2 years after treatment.

RESULTS: The original NEI-VFQ-25 exhibited multiple issues including limitations in response functioning and scale dimensionality. These issues were rectified by revising the NEI-VFQ25 into two separate unidimensional scales measuring 'Vision-related Activity Limitation' (VAL) and 'Socioemotional Functioning' (SEF). Participants' mean VAL score at baseline on a Rasch-transformed 0-100 scale was 46.1 (11.7), improving to 48.4 (13.7) after treatment (

CONCLUSIONS: The scoring structure of the original NEI-VFQ-25 has limitations that undermine its psychometric validity as a measure of VRQoL. Using the Rasch-revised NEI-VFQ-25, we determined that improvement in VRQoL after treatment with lenadogene nolparvovec was driven predominantly by an improvement in socioemotional functioning.

Volume

10

Issue

1

ISSN

2397-3269

Published In/Presented At

Chen, B. S., Perot, S., Taiel, M., Yu-Wai-Man, P., Horton, M., & LHON Study Group (2025). Rasch analysis of the NEI-VFQ-25: vision-related quality of life in Leber hereditary optic neuropathy after lenadogene nolparvovec gene therapy. BMJ open ophthalmology, 10(1), e002164. https://doi.org/10.1136/bmjophth-2025-002164

Disciplines

Business Administration, Management, and Operations | Health and Medical Administration | Management Sciences and Quantitative Methods

PubMedID

40967677

Department(s)

Administration and Leadership

Document Type

Article