LAUSR

This report examined the benefits and risks of palbociclib plus endocrine therapy (ET) in men with hormone receptor-positive (HR+)/human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer (MBC). Palbociclib was evaluated using 3 independent data sources: real-world data from pharmacy and medical claims, a de-identified real-world data source derived from electronic health records (EHRs), and a global safety database. From medical and pharmacy records, 1139 men with MBC were identified; in the first-line setting, median duration of treatment was longer with palbociclib plus ET (n=37; 8.5 months; 95% CI; 4.4-13.0) than ET alone (n=214; 4.3 months; 95% CI; 3.0-5.7) and specifically, was longer with palbociclib plus letrozole (n=26; 9.4 months; 95% CI; 4.4-14.0) than letrozole alone (n=63; 3.0 months; 95% CI; 1.8-4.8). In the EHR-derived database, 59 men received treatment for MBC; real-world response across all lines of therapy in the metastatic setting was reported in 4 of 12 patients (33.3%) in the palbociclib plus ET group, versus 1 of 8 (12.5%) patients in the ET group. Review of the global safety database did not identify any new safety signals in palbociclib-treated men. Real-world data indicated that men with MBC benefit from palbociclib plus ET, with a safety profile consistent with previous observations in women with MBC. Collective data on palbociclib in women and men in this report, including clinical trial data, real-world data, and a well-established risk/benefit profile, led to US approval of an expansion of the palbociclib indication to include men with MBC.