Key clinical point: Pyrotinib+trastuzumab+docetaxel was more effective than placebo+trastuzumab+docetaxel in improving progression-free survival (PFS) outcomes and showed a manageable safety profile in patients with untreated human epidermal growth factor receptor 2-positive (HER2+) metastatic breast cancer (BC).
Major finding: PFS improved by 59% with pyrotinib+trastuzumab+docetaxel vs placebo+trastuzumab+docetaxel treatment (24.3 vs 10.4 months; hazard ratio 0.41; stratified 1-sided P < .001). The most frequently reported grade ≥3 adverse events in the pyrotinib vs placebo treatment arm were decreased neutrophil count (63% vs 65%), decreased white blood cell count (53% vs 51%), and diarrhea (46% vs 3%).
Study details: Findings are from the phase 3 PHILA trial including 590 female patients with untreated HER2+ metastatic BC who were randomly assigned to receive pyrotinib or placebo, both in combination with trastuzumab and docetaxel.
Disclosures: This study was funded by Jiangsu Hengrui Pharmaceuticals, China, and other sources. Three authors declared being employees of Jiangsu Hengrui Pharmaceuticals, and two other authors reported ties with various sources.
Source: Ma F et al, on behalf of the PHILA Investigators. Pyrotinib versus placebo in combination with trastuzumab and docetaxel as first line treatment in patients with HER2 positive metastatic breast cancer (PHILA): Randomised, double blind, multicentre, phase 3 trial. BMJ. 2023;383:e076065 (Oct 31). doi: 10.1136/bmj-2023-076065