Psychometric Evaluation of the HFDD, PROMIS SD SF 8b, and MENQOL for Vasomotor Symptoms Caused by Endocrine Therapy: Reliability, Validity, and Differential Item Functioning
Author(s)
Andrew Trigg, MSc1, Christian Seitz, PhD2, Christoph Gerlinger, PhD2, Helena Bradley, BSc3, Melissa Lucy Barclay, BSc, MSc3, Adam Gater, MSc3, Claudia Haberland, MSc, PhD4.
1Bayer Plc, Reading, United Kingdom, 2Bayer AG, Berlin, Germany, 3Adelphi Values Ltd, Bollington, United Kingdom, 4BAYER AG, Berlin, Germany.
1Bayer Plc, Reading, United Kingdom, 2Bayer AG, Berlin, Germany, 3Adelphi Values Ltd, Bollington, United Kingdom, 4BAYER AG, Berlin, Germany.
OBJECTIVES: Women with breast cancer are usually prescribed endocrine therapy (ET) after initial treatment to reduce recurrence; however, vasomotor symptoms (VMS; hot flashes) are a significant ET side effect, often causing treatment discontinuation. This study evaluated the psychometric properties of scores from the Hot Flash Daily Diary (HFDD), PROMIS Sleep Disturbance Short Form 8b (PROMIS SD SF 8b), and Menopause-Specific Quality of Life (MENQOL) in women experiencing VMS caused by ET.
METHODS: HFDD, PROMIS SD SF 8b, and MENQOL data collected at baseline and over 12 weeks was analyzed from a double-blind, randomized, placebo-controlled multicenter Phase 3 study of elinzanetant to treat VMS caused by ET (OASIS 4; NCT05587296). Analyses assessed distributional properties, reliability and validity. Differential item functioning (DIF) was assessed according to VMS cause (ET versus menopause) by pooling OASIS 4 and OASIS 2 data (a Phase 3 study of elinzanetant in VMS associated with menopause; NCT05099159).
RESULTS: Data from 474 OASIS 4 trial participants were used for analyses (mean age 51.0, StdDev 7.3). No evidence of floor or ceiling effects was found. Inter-item correlations, confirmatory factor analysis (MENQOL) and item-response theory (PROMIS SD SF 8b) supported dimensionality and scoring algorithms. Good to excellent test-retest reliability of HFDD scores was observed (intra-class correlation coefficients 0.869-0.935). Convergent/divergent correlations with measures of similar/distinct concepts were mostly consistent with pre-specified hypotheses. Large differences in PROMIS SD SF 8b scores (effect sizes ≥0.80, p<0.01) were observed between clinically distinct subgroups. No DIF was observed for the PROMIS SD SF 8b and MENQOL according to VMS cause (ET [OASIS 4] versus menopause [OASIS 2, n=359]).
CONCLUSIONS: Consistent with previous results in menopause, findings demonstrate that the HFDD, PROMIS SD SF 8b and MENQOL scores are valid and reliable, supporting their application in assessing clinical trial endpoints in women experiencing VMS caused by ET.
METHODS: HFDD, PROMIS SD SF 8b, and MENQOL data collected at baseline and over 12 weeks was analyzed from a double-blind, randomized, placebo-controlled multicenter Phase 3 study of elinzanetant to treat VMS caused by ET (OASIS 4; NCT05587296). Analyses assessed distributional properties, reliability and validity. Differential item functioning (DIF) was assessed according to VMS cause (ET versus menopause) by pooling OASIS 4 and OASIS 2 data (a Phase 3 study of elinzanetant in VMS associated with menopause; NCT05099159).
RESULTS: Data from 474 OASIS 4 trial participants were used for analyses (mean age 51.0, StdDev 7.3). No evidence of floor or ceiling effects was found. Inter-item correlations, confirmatory factor analysis (MENQOL) and item-response theory (PROMIS SD SF 8b) supported dimensionality and scoring algorithms. Good to excellent test-retest reliability of HFDD scores was observed (intra-class correlation coefficients 0.869-0.935). Convergent/divergent correlations with measures of similar/distinct concepts were mostly consistent with pre-specified hypotheses. Large differences in PROMIS SD SF 8b scores (effect sizes ≥0.80, p<0.01) were observed between clinically distinct subgroups. No DIF was observed for the PROMIS SD SF 8b and MENQOL according to VMS cause (ET [OASIS 4] versus menopause [OASIS 2, n=359]).
CONCLUSIONS: Consistent with previous results in menopause, findings demonstrate that the HFDD, PROMIS SD SF 8b and MENQOL scores are valid and reliable, supporting their application in assessing clinical trial endpoints in women experiencing VMS caused by ET.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO193
Topic
Clinical Outcomes, Patient-Centered Research
Topic Subcategory
Clinical Outcomes Assessment
Disease
Reproductive & Sexual Health