Patient-reported outcomes predict survival and adverse events following anticancer medicines initiation


The prognostic value of patient-reported outcomes (PROs) has been minimally explored in advanced breast cancer, and their comparative prognostic performance against Eastern Cooperative Oncology Group performance status (ECOG PS) is largely unknown.


This study pooled individual participant data from clinical trials CLEOPATRA, EMILIA, and MARIANNE. Pre-treatment PRO associations with overall survival (OS), progression-free survival (PFS), and grade ≥ 3 adverse events were evaluated via Cox proportional hazards regression. Prognostic performance was assessed with the C-statistic (c). PRO values were collected via the functional assessment of the cancer therapy-breast (FACT-B) questionnaire. All analyses were stratified by study and treatment arms. Analyses adjusted for known prognostic variables were conducted.


The study included data from 2894 patients initiated on contemporary therapies including pertuzumab (n=765), trastuzumab (n=1173), trastuzumab emtansine (n=1225), taxanes (n= 1173), lapatinib (n= 496), and capecitabine (n=496). On univariable and adjusted analysis, patient-reported physical well-being, functional well-being, and breast cancer subscale were all identified to be associated with OS, PFS, and grade ≥ 3 adverse events (p<0.05). Patient-reported physical well-being was the most prognostic PRO for all assessed outcomes. The OS prognostic performance of physical well-being (c=0.58) was superior to ECOG PS (c=0.56) (p<0.05), with multivariable analysis indicating both provide independent information (p<0.0001).


PROs were identified as independent prognostic factors for OS, PFS, and grade ≥ 3 adverse events in patients with HER2-positive advanced breast cancer initiating contemporary treatment options. Further, patient-reported physical well-being was more prognostic of OS than ECOG PS and contained independent information. PROs have value as prognostic and stratification factors for clinical use and research trials of anti-cancer medicines in HER2-positive advanced breast cancer.