KEY TAKEAWAYS
- The CARTITUDE-2 phase 2 study aimed to validate the MySIm-Q Symptom score and enhance its utility for assessing MM symptoms and impacts.
- Researchers noticed that the MySIm-Q shows reliability and validity in measuring MM symptoms; further investigation is ongoing.
MySIm-Q is a validated disease-specific PRO instrument that measures symptoms and impacts experienced by patients with active Multiple Myeloma (MM).
Mateos MV and the team aimed to assess the psychometric properties of the MySIm-Q Symptom score using data from the CARTITUDE-2 study.
They performed an inclusive analysis in the CARTITUDE-2 study, which evaluates ciltacabtagene autoleucel (cilta-cel) in MM patients across various disease settings. Pooled data from cohorts A, B, and C were examined, encompassing lenalidomide-refractory MM, early relapse after initial treatment, and relapsed/refractory MM with prior treatments.
Assessments, including MySIm-Q, EORTC QLQ-C30, PGIS, and PGIC, were conducted at multiple time points post cilta-cel administration, following FDA-recommended frameworks for evaluating PRO measurement properties. Internal consistency, test-retest reliability, concurrent validity, known-group validity, and meaningful within-patient change in MySIm-Q Symptom score analyses were conducted.
They performed an inclusive analysis in the CARTITUDE-2 study, assessing 82 patients (cohorts A/B/C, n=43/19/20) who completed MySIm-Q assessments. Internal consistency of the MySIm-Q symptom score was deemed acceptable (Cronbach’s α-coefficient, 0.89), surpassing the predefined threshold (≥0.70). Test-retest reliability was adequate (2-way random intraclass correlation coefficient [ICC(2,1)], 0.81), meeting the minimally acceptable ICC(2,1) of 0.70.
MySIm-Q Symptom score showed acceptable concurrent validity with existing measures from the EORTC QLQ-C30, with a high proportion of correlations exceeding │0.60│. Known-groups validity was confirmed, as evidenced by discrimination across disease severity groups based on PGIS and EORTC QLQ-C30 items. The PGIC anchor-based meaningful within-patient change deterioration threshold and the average distribution-based clinical significance threshold was well-aligned.
The study concluded that the preliminary analysis offers compelling evidence of the MySIm-Q’s reliability and validity in detecting changes in MM symptoms. These findings strongly support its utilization as a fit-for-purpose PRO instrument in MM trials. Moreover, the forthcoming analysis of psychometric properties using data from the phase 3 CARTITUDE-4 study holds promise for further validation and application.
The study was sponsored by the Janssen Research & Development, LLC.
Source: https://www.ispor.org/heor-resources/presentations-database/presentation/intl2024-3897/136386
Clinical Trial: https://clinicaltrials.gov/study/NCT04133636
Mateos MV, Cohen AD, Cohen YC, et al. (2024). “Psychometric Properties of the Multiple Myeloma Symptom and Impact Questionnaire (MySIm-Q) in Patients with Relapsed/Refractory Multiple Myeloma (MM): Analysis of Phase 2 CARTITUDE-2 Study Cohorts A, B, and C.” Presented at ISPOR 2024.
