Myelofibrosis symptom assessment form total symptom score version 4.0: measurement properties from the MOMENTUM phase 3 study.

Autor: Daskalopoulou C; IQVIA, Athens, Greece. christina.daskalopoulou@iqvia.com., Gorsh B; Daiichi Sankyo, Inc., Basking Ridge, NJ, USA., Dumi G; IQVIA, Athens, Greece., Deheshi S; GSK plc, Philadelphia, PA, USA., Gwaltney C; Gwaltney Consulting, Westerly, RI, USA., Paty J; IQVIA, New York, NY, USA., Ellis C; GSK plc, Philadelphia, PA, USA., Kawashima J; Sierra Oncology, a GSK company, San Mateo, CA, USA., Mesa R; Atrium Health Wake Forest Baptist Comprehensive Cancer Center, Winston-Salem, NC, USA.
Jazyk: angličtina
Zdroj: Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation [Qual Life Res] 2024 Nov 25. Date of Electronic Publication: 2024 Nov 25.
DOI: 10.1007/s11136-024-03855-1
Abstrakt: Purpose: The Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) comprises 7 common MF symptom items (fatigue, night sweats, pruritus, abdominal discomfort, pain under the left ribs, early satiety, bone pain) and is the first patient-reported outcome (PRO) instrument designed to assess MF symptom burden. Given that information on the psychometric properties of this instrument has been limited, we sought to evaluate its measurement properties and validate its use in the phase 3 MOMENTUM trial.
Methods: Data were pooled to assess MFSAF item distribution, structural validity, reliability (test-retest and internal consistency), construct validity (convergent, divergent, and known-groups), and sensitivity to change. Other PRO measures included Patient Global Impression of Severity/Change (PGIS/PGIC), EORTC QLQ-C30, PROMIS Physical Function Short Form 10b, and ECOG performance status.
Results: Participants (N = 195) showed high completion rates (> 93%) across 24 weeks. Moderate to strong Spearman correlation coefficients among items were mostly observed at baseline (range, 0.289-0.772) and week 24 (range, 0.391-0.829), which supported combining items into a multi-item scale and total score. Internal consistency (Cronbach's α, 0.877 at baseline and 0.903 at week 24) and test-retest reliability (intraclass correlation coefficient, > 0.829) were satisfactory across selected time intervals. Reliability was also supported by McDonald's omega (ω) coefficient (> 0.875). MFSAF moderately correlated with PRO measures of similar content, differentiated between PGIS and ECOG groups (P < .001), and was able to detect change over time.
Conclusions: The MFSAF v4.0 is a valid tool to assess MF symptom burden, supporting its use in future trials in similar populations.
Competing Interests: Declarations. Ethical approval: The current analysis is based on results of the MOMENTUM trial. MOMENTUM was done in accordance with the Declaration of Helsinki and the International Council for Harmonisation guidelines on Good Clinical Practice. Institutional review boards or independent ethics committees at each site approved the protocol. Consent to participate: Written consent was obtained from all individual participants included in the MOMENTUM study. Consent to Publish: Not applicable. All personal data are deidentified. Competing interests: Christina Daskalopoulou holds an advisory role within IQVIA and reports grants from GSK during the conduct of this study. Boris Gorsh has been employed by GSK and Daiichi Sankyo during the production of this manuscript and has stock or stock options with both companies. Gerasimos Dumi and Jean Paty are employees of IQVIA. Samineh Deheshi is an employee of GSK. Chad Gwaltney has received consulting fees from IQVIA for this MFSAF evaluation. Catherine Ellis is an employee of GSK and reports stock or stock options. Jun Kawashima is an employee of Sierra Oncology, a GSK company. Ruben Mesa reports consulting fees/honoraria from AbbVie, Blueprint, Bristol Myers Squibb, CTI, Genentech, Geron, GSK, Incyte, MorphoSys, Novartis, Sierra, Sierra Oncology, and Telios. All authors acknowledge medical writing support related to this manuscript, funded by GSK.
(© 2024. The Author(s).)
Databáze: MEDLINE
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