Efficacy and safety of a pyrotinib-based regimen in non-small cell lung cancer patients harboring HER2 alterations: A real-world retrospective study

Background:Pyrotinib, a novel irreversible pan-HER tyrosine kinase inhibitor, has been approved for the treatment of HER2-positive metastatic breast cancer in China. The aim of this study was to evaluate the efficacy and safety of pyrotinib in advanced nonsmall cell lung cancer (NSCLC) patients with HER2 alterations in real-world practice.Materials and Methods:A retrospective analysis of advanced NSCLC with HER2 mutations or amplifications who received pyrotinib-based treatment at the Qilu Hospital in Shandong University was performed. The primary end points were objective response rate and safety. The secondary end points were progression-free survival, disease control rate, and overall survival.Results:Twenty three eligible patients from a single center were enrolled between June 2019 and March 2023; among them, 21 had HER2 mutation and two harbored HER2 amplification. Evaluation of the efficacy in 21 patients revealed an objective response rate of 28.6% (6/21; 95% confidence interval [CI]: 7.5%-49.6%) and disease control rate of 85.7% (18/21). The median progression-free survival and overall survival were 7.7 months (95% CI: 6.07-9.33) and 20.8 months (95% CI: 8.42-33.18), respectively. The most common adverse events (AEs) included diarrhea (n = 14, 60.9%), nausea (n = 5, 21.7%), and liver dysfunction (n = 5, 21.7%). Seven patients (7/23, 30.4%) had grade 3-4 AE; no grade 5 AE was observed. Furthermore, one patient (1/23, 4.3%) experienced dose withdrawal and two (2/23, 8.7%) presented with dose reduction symptoms.Conclusion:Pyrotinib-based therapy showed promising antitumor activity and acceptable safety in advanced NSCLC patients with HER2 alterations.