Multicenter study of pertuzumab plus trastuzumab for HER2-positive, metastatic biliary tract cancer

Systemic therapies for metastatic biliary tract cancers (mBTC) are few, and patients have a median overall survival of less than one year. The multicenter MyPathway multiple basket study evaluates FDA-approved therapies in non-indicated tumors with potentially actionable molecular alterations. In a MyPathway cohort of patients with previously treated mBTC with HER2 amplification, HER2 overexpression, or both, the combination of pertuzumab and trastuzumab was well tolerated and induced partial response in 23% of patients (95% CI 11-39).