biostudies-literatureUnknown10(1)Chen BS<h4>Objectives</h4>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.<h4>Methods</h4>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.<h4>ResulBMJ open ophthalmologye002164https://www.ebi.ac.uk/biostudies/studies/S-EPMC12458720biostudies-literatureRasch analysis of the NEI-VFQ-25: vision-related quality of life in Leber hereditary optic neuropathy after lenadogene nolparvovec gene therapy.PMC12458720Wilkins SAcheson JMathias MHubbard GBSergott RCHaller JACalcagno FThornton SPatel SDeBusk AAHendrick AMNeuenhahn MPreston MHwang JMSchertler CLHON Study GroupSilvestri SHorton MRomagnoli MMemon MATsui IDuBois LWang AGCastillo LCarbonelli MPriglinger CRudolph GHawy EHage RGibbs DCheng HCVito LDKhemliche WCestari DPriglinger SLeitch-Devlin LBoston HAung ALeroy BPEleftheriadou MBanik RBurale APlaine LEsposti SPecen PTai KYu-Wai-Man PJorany RMuth DContin MCarelli VMoster MLGirmens JFRuark SAmore GSheel PDonahue SZaeytijd JJurkute NSun CTollis HBiousse VCatarino CBBattista MVerhauwen HFernandez GRHussain RMunoz Negrete FJPerot SLeruez SLivonius BVSadun AATaiel MThurau SGemenetzi MSahel JAVignal-Clermont CDattilo MGarcia VAnand SBarboni PChen CFilho AFGangaputra SHeilweil GBandello FChen BSHildebrandt MPressler AD'Agati PKlopstock TMorgia CTucker WRCascavilla MLPeragallo JHMohamed SCauwenbergh CVPina ASantaMaria MWolf AAl-Tamami JBarrett MForoozan RKaranjia RCho SO'Keefe GMorilla ASubramanian PSMassini MDobbs JFortin EChwalisz BKNewman NJUrquijo LJfalseRasch analysis of the NEI-VFQ-25: vision-related quality of life in Leber hereditary optic neuropathy after lenadogene nolparvovec gene therapy.<h4>Objectives</h4>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.<h4>Methods</h4>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.<h4>Resul2025-01-01T00:00:00Z2025 Sep2026-07-15T06:51:37.276Z2026-06-30T03:27:24.821ZS-EPMC124587204096767710.1136/bmjophth-2025-002164