Modern Cancer Hospital Guangzhou

There are limited data about DS-8201 (trastuzumab deruxtecan) in Chinese patients with advanced human epidermal growth factor receptor 2 (HER2)-positive breast cancer previously treated with trastuzumab emtansine (T-DM1). This study aimed to evaluate the efficacy and safety of DS-8201 compared to the treatment of physician's choice (TPC) in this population. In this retrospective cohort study, 185 eligible patients treated between December 2022 and March 2025 were assigned to either the DS-8201 group (n=83) or the TPC group (n=102), which received regimens such as capecitabine combined with anti-HER2 therapy. Progression-free survival (PFS), overall survival (OS), objective response rate (ORR), duration of response (DoR), disease control rate (DCR), quality of life (QoL), and safety were assessed. The DS-8201 group demonstrated significantly superior 12- and 24-month PFS (62.65% vs. 42.16%; 50.60% vs. 23.53%) and OS (90.36% vs. 60.78%; 75.90% vs. 54.90%) compared to the TPC group (all P<0.05). ORR (72.29% vs. 45.10%), median DoR (20.00 vs. 9.00 months), and DCR (80.72% vs. 60.78%) were also significantly improved (all P<0.05). Patients receiving DS-8201 reported better QoL and delayed time to first hospitalization (all P<0.05). The overall incidence of treatment-emergent adverse events (TEAEs) was similar between groups (P>0.05). However, DS-8201 was associated with higher rates of nausea, alopecia, and drug-induced interstitial lung disease, while the TPC group had more diarrhea and palmar-plantar erythrodysesthesia (all P<0.05). DS-8201 showed significantly better efficacy and similar overall safety compared to TPC in patients with T-DM1-pretreated HER2-positive advanced breast cancer, supporting its use as a treatment option in this setting.