Abstract Details

Title Establishing Psychometric Evidence of the Novel Patient-reported Functional Impacts of Narcolepsy Instrument (FINI) Using Data from Observational and Phase 2 Clinical Trials in Narcolepsy

Topic Sleep

Presentation(s) P12 - Poster Session 12 (11:45 AM-12:45 PM)

Poster/Presentation
Number 4-001

Objective To evaluate psychometric properties of the Functional Impacts of Narcolepsy Instrument (FINI) using data from two narcolepsy studies.

Background Narcolepsy-specific tools assessing disease impact are currently lacking. The FINI is a novel 28-item patient-reported outcome (PRO) measure developed to evaluate treatment outcomes in people with narcolepsy types 1 and 2 (NT1/NT2).

Design/Methods FINI psychometric properties were evaluated using pooled data from an observational study (NT1, n=126) and a Phase 2 clinical trial (n=125; NCT04096560). Reliability, validity (convergent and known-groups) and sensitivity to change were evaluated; scale structure was defined and confirmed using exploratory factor analysis (EFA) and Rasch analysis. Meaningful within-patient change (MWPC) thresholds were defined using an anchor-based approach.

Results EFA results supported a 6-domain model (Tiredness, Cognitive Functioning, Cataplexy, Social Activities, Everyday Activities, and Everyday Responsibilities). Rasch analysis confirmed FINI’s multidimensionality with each domain independently assessing a core narcolepsy concept. FINI domains demonstrated good internal consistency (Cronbach’s alpha >0.77) and moderate-to-strong test-retest reliability (ICCs: 0.61–0.86). Known-groups validity was supported by higher domain scores with increasing severity ratings on the Epworth Sleepiness Scale (ESS), and Clinical/Patient Global Impression of Severity (CGI-S/PGI-S). Convergent validity was supported by strong correlations of FINI domains with other PROs assessing similar concepts (EQ-5D-5L, FOSQ-10, MFI, SF-36, and ESS). Sensitivity to change was supported by correlational analyses and categorical change group comparisons across clinical outcome assessments measuring similar concepts using symptom-specific (CGI-S/PGI-S) and change-specific (CGI/PGI of Improvement) anchors. MWPC thresholds were estimated as ranges: Tiredness (-30 to -40), Cognitive Functioning (-30 to -40), Cataplexy (-20 to -25), Social Activities (-30 to -40), Everyday Activities (-30 to -40), and Everyday Responsibilities (-25 to -35).

Conclusions FINI psychometric properties were confirmed and MWPC thresholds proposed for each domain. The FINI is a robust, valid measure to assess treatment benefit related to improvement in functional outcomes in people with narcolepsy.

Authors/Disclosures
heather romero, PhD PRESENTER	Dr. romero has received personal compensation for serving as an employee of Takeda. Dr. romero has stock in Takeda.
Yulia Savva, PhD	Dr. Savva has stock in Takeda. Dr. Savva has stock in Thermo Fischer.
Helen A. Doll, PhD	Dr. Doll has nothing to disclose.
Amy Howerter, PhD	Dr. Howerter has received personal compensation for serving as an employee of Clinical Outcomes Solutions.
Shruti Dave, PhD	Dr. Dave has nothing to disclose.
Stephen Crawford, PhD	Dr. Crawford has received personal compensation for serving as an employee of Takeda. Dr. Crawford has stock in Takeda.

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