Background: The addition of atezolizumab to carboplatin and etoposide (CP/ET) significantly improved progression-free and overall survival for patients with extensive-stage small-cell lung cancer (ES-SCLC) in the IMpower133 study (NCT02763579). We have evaluated adverse events (AEs) and patient-reported outcomes in IMpower133 to assess the benefiterisk profile of this regimen. Patients and methods: Patients received four 21-day cycles of CP/ET plus intravenous atezolizumab 1200 mg or placebo (induction phase), followed by atezolizumab or placebo (maintenance phase) until progression or loss of benefit. AEs were assessed and patient-reported outcomes were evaluated every 3 weeks during treatment using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire e Core 30 (QLQ-C30) and QLQ-LC13. Results: Overall, 394 patients were assessable for safety in the induction phase and 318 in the maintenance phase. The frequency of AEs, grade 3e4 AEs, and serious AEs was similar between arms in both phases. Immune-related AEs were more frequent in the atezolizumab arm during both induction (28% versus 17%; leading to atezolizumab/placebo interruption 9% versus 5%, leading to withdrawal 4% versus 0%) and maintenance (26% versus 15%; leading to atezolizumab/placebo interruption, 3% versus 2%, leading to withdrawal 1% versus 1%), most commonly rash (induction 11% versus 9%, maintenance 14% versus 4%), and hypothyroidism (induction 4.0% versus 0%, maintenance 10% versus 1%). Changes in patient-reported treatment-related symptoms commonly associated with quality of life impairment were generally similar during induction and most of the maintenance phase. Patientreported function and health-related quality of life (HRQoL) improved in both arms after initiating treatment, with more pronounced and persistent HRQoL improvements in the atezolizumab arm. Conclusions: In patients with ES-SCLC, atezolizumab plus CP/ET has a comparable safety profile to placebo plus CP/ET, and the addition of atezolizumab did not adversely impact patient-reported HRQoL. These data demonstrate the positive benefiterisk profile of first-line atezolizumab plus CP/ET in ES-SCLC and further support this regimen as a new standard of care in this setting. Clinical trials number: NCT02763579.

Purpose: To evaluate the reliability and validity of six PROMIS measures (anxiety, depression, fatigue, pain interference, physical function, sleep disturbance) telephone-administered to a diverse, population-based cohort of localized prostate cancer patients. Methods: Newly-diagnosed men were enrolled in the North Carolina Prostate cancer Comparative Effectiveness and Survivorship Study. PROMIS measures were telephone-administered pre-treatment (baseline), and at 3-months and 12-months post-treatment initiation (N=778). Reliability was evaluated using Cronbach’s alpha. Dimensionality was examined with bifactor models and explained common variance (ECV). Ordinal logistic regression models were used to detect potential differential item functioning (DIF) for key demographic groups. Convergent and discriminant validity were assessed by correlations with the legacy instruments Memorial Anxiety Scale for Prostate Cancer and SF-12v2. Known-groups validity was examined by age, race/ethnicity, comorbidity, and treatment. Results: Each PROMIS measure had high Cronbach’s alpha values (0.86 to 0.96) and was sufficiently unidimensional. Floor effects were observed for anxiety, depression, and pain interference measures; ceiling effects were observed for physical function. No DIF was detected. Convergent validity was established with moderate to strong correlations between PROMIS and legacy measures (0.41 to 0.77) of similar constructs. Discriminant validity was demonstrated with weak correlations between measures of dissimilar domains (−0.20 to −0.31). PROMIS measures detected differences across age, race/ethnicity, and comorbidity groups; no differences were found by treatment. Conclusions: This study provides support for the reliability and construct validity of six PROMIS measures in prostate cancer, as well as the utility of telephone administration for assessing HRQoL in low literacy and hard-to-reach populations.

BACKGROUND: Administrative claims contain detailed medication, diagnosis, and procedure data, but the lack of clinical outcomes for rheumatoid arthritis (RA) historically has limited their use in comparative effectiveness research. A claims-based algorithm was developed and validated to estimate effectiveness for RA from data for adherence, dosing, and treatment modifications.

When a claims-based algorithm was applied to a large commercial claims database, etanercept was categorized as the most effective and had the lowest estimated 1-year biologic cost per effectively treated patient. This proxy for effectiveness from claims databases was validated against a clinical effectiveness scale, but analyses of the second year or the year after a biologic switch were not included in the validation. Costs of other medications were not included in cost calculations.

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