On March 16, 2026,The British Medical Journal(BMJ) online published the long-term survival results of the Phase III PHILA trial. The paper was led by Academician Xu Binghe from the Cancer Hospital Chinese Academy of Medical Sciences as corresponding author, with Professor Ma Fei from the same institution and Professor Yan Min from Henan Cancer Hospital as co-first authors. In 2023, the interim progression-free survival (PFS) results of this study were already published in BMJ, and this update analysis now demonstrates a clear overall survival (OS) benefit, with a 26% reduction in mortality risk. These findings showcase "Chinese wisdom" in breast cancer treatment to the global oncology community.

HER2-positive breast cancer is characterized by rapid progression and poor prognosis. Based on the CLEOPATRA trial, the combination of pertuzumab, trastuzumab, and docetaxel has become the standard first-line regimen for HER2-positive metastatic breast cancer. However, pertuzumab targets only the HER2 dimerization domain, whereas pyrotinib is an irreversible pan-HER tyrosine kinase inhibitor that simultaneously blocks the intracellular kinase domains of HER1, HER2, and HER4. The PHILA trial was designed based on this mechanistic rationale to evaluate the efficacy and safety of the pyrotinib + trastuzumab + docetaxel (PyroHT) regimen as first-line therapy for HER2-positive advanced breast cancer.

The PHILA trial enrolled 590 patients with HER2-positive recurrent or advanced breast cancer who had not received prior systemic therapy, across 40 centers in China. Patients were randomized 1:1 to receive either pyrotinib or placebo, both in combination with trastuzumab and docetaxel. The results showed that the investigator-assessed median PFS in the pyrotinib group reached 22.1 months, compared to only 10.5 months in the placebo group (HR 0.44), representing a 56% reduction in the risk of disease progression. Updated survival data revealed that OS in the pyrotinib group was superior to that in the placebo group (HR 0.74, nominal one-sided P="0.02)," with a 26% reduction in the risk of death. The 3-, 4-, and 5-year OS rates in the pyrotinib group were 80%, 73%, and 66%, respectively, all superior to those in the placebo group (72%, 63.5%, and 58.5%).

The PyroHTregimen demonstrated significant and durable long-term survival benefits, offering not only marked improvements in PFS and OS for patients who had prior trastuzumab exposure, but also effectively delaying the occurrence of brain metastases. This long-term follow-up data, once again published in BMJ, reflects further international academic recognition of the long-term efficacy and safety of the PyroHT regimen, and marks the transition of China's homegrown breast cancer clinical research from "first breakthrough" to "sustained leadership."