Concordance of real-world versus conventional progression-free survival from a phase 3 trial of endocrine therapy as first-line treatment for metastatic breast cancer

• DOI: 10.1371/journal.pone.0227256
• Publication Date: 2020-04-21T20:28:58Z
• Authors: Cynthia Huang Bar...; Jack Mardekian; Matthew James Cotter; Xin Huang; Zhe Zhang; Christina M. Parr...; Ariel Bulua Bourla
• Keywords: Letrozole; Progression-free survival

There is growing interest in leveraging real-world data to complement knowledge gained from randomized clinical trials and inform the design of prospective studies oncology. The present study compared outcomes women with metastatic breast cancer who received letrozole as first-line monotherapy oncology practices across United States versus patients letrozole-alone cohort PALOMA-2 phase 3 trial. (N = 107) was derived de-identified patient Flatiron Health electronic health record database. trial 222) comprised postmenopausal arm PALOMA-2. Patients per labeling judgment; 2.5 mg/d, continuous. Real-world survival response rates were based on evidence disease burden curated clinician notes, radiologic reports, pathology reports available record. Progression-free objective rate Response Evaluation Criteria Solid Tumors v1.1. Concordance retrospectively assessed using inverse probability treatment weighting-adjusted Cox regression analysis. Inverse results showed similar median progression-free cohorts (18.4 16.6 months, respectively): hazard ratio reference 1.04 (95% CI, 0.69–1.56). No significant difference observed rates: 41.8% vs 39.4% (odds reference: 0.91 [95% 0.57–1.44]). These findings indicate that abstracted records proper quality controls can yield meaningful information outcomes. increase confidence use assessments progression efficacy endpoints. Trial registration NCT01740427; Funding: Pfizer.