Mounzer E. Agha

Background: Oral anticancer medications are standard care for cancer. Medication adherence influences health outcomes, but valid, reliable measures assessing self-reported medication adherence are limited. Psychometric properties of the PROMIS ^® Medication Adherence Scale (PMAS) were evaluated in patients prescribed oral anticancer medications for multiple myeloma. Methodology: This was a secondary analysis from a longitudinal observational study examining medication adherence, symptoms, quality of life, and financial hardship among individuals prescribed oral anticancer medications for multiple myeloma. PMAS measured self-reported medication adherence. Self-report and medical record data assessed participant characteristics and adherence correlates. Objective medication adherence indices were generated from continuous electronic event monitored data. Internal consistency reliability was estimated using Cronbach’s alpha. Dimensionality was assessed with confirmatory factor analysis. Construct validity was assessed considering adherence correlates. Spearman rank-order correlations summarized associations between PMAS and electronic event monitored data. Results: PMAS items had limited variability, with high adherence over time. Reliability of PMAS scores was adequate (T1 α = 0.82, 95% CI: 0.75, 0.87; T2 α = 0.84, 95% CI: 0.78, 0.89). Confirmatory factor analysis fit was better for sub-scales (Medication Beliefs and Knowledge and Medication Taking Behaviors) than Total scale, particularly for the Medication Beliefs and Knowledge subscale. Adherence correlates were observed as expected between PMAS and age, self-reported cognitive function, symptom severity, and depression. Weak/moderate positive associations were found between PMAS and electronic event monitored data. Conclusions: Reliability, two-factor dimensionality, and construct validity were supported, with some evidence of concurrent criterion validity with electronic event monitored adherence data. Additional validation testing is needed to support findings. Evidence supports the feasibility of longitudinal oral anticancer medication adherence assessment monitoring using PMAS.

Ciltacabtagene autoleucel (cilta-cel) is a Chimeric antigen receptor T-cell therapy with the potential for long-term disease control in heavily pre-treated patients with relapsed/refractory multiple myeloma (RRMM). As cilta-cel was assessed in the single-arm CARTITUDE-1 clinical trial, we used an external cohort of patients from the Therapie Monitor registry fulfilling the CARTITUDE-1 inclusion criteria to evaluate the effectiveness of cilta-cel for overall survival (OS) and time to next treatment (TTNT) vs. real-world clinical practice. Individual patient data allowed us to adjust the comparisons between both cohorts, using the inverse probability of treatment weighting (IPW; average treatment effect in the treated population (ATT) and overlap population (ATO) weights) and multivariable Cox proportional hazards regression. Outcomes were compared in intention-to-treat (HR, IPW-ATT: TTNT: 0.13 (95% CI: 0.07, 0.24); OS: 0.14 (95% CI: 0.07, 0.25); IPW-ATO: TTNT: 0.24 (95% CI: 0.12, 0.49); OS: 0.26 (95% CI: 0.13, 0.54)) and modified intention-to-treat (HR, IPW-ATT: TTNT: 0.24 (95% CI: 0.09, 0.67); OS: 0.26 (95% CI: 0.08, 0.84); IPW-ATO: TTNT: 0.26 (95% CI: 0.11, 0.59); OS: 0.31 (95% CI: 0.12, 0.79)) populations. All the comparisons were statistically significant in favor of cilta-cel. These results highlight cilta-cel’s potential as a novel, effective treatment to address unmet needs in patients with RRMM.

A blastic plasmacytoid dendritic-cell neoplasm is a rare tumor with very aggressive clinical behavior and no established treatment.¹ Available treatment options include aggressive multiagent chemotherapeutic regimens. Treatment for relapsed disease is largely ineffective, and the prognosis for patients with refractory disease is poor.