Efficacy and safety of trastuzumab emtansine in patients with advanced human epidermal growth factor receptor 2-positive extramammary Paget's disease: an investigator-initiated, open-label, single-arm, multicentre, prospective, phase II clinical trial (TEMENOS-2).

ABSTRACT:

Background

Extramammary Paget's disease (EMPD) is a rare cutaneous adenocarcinoma with no established standard systemic therapy owing to limited clinical evidence. Approximately 40% of metastatic EMPD cases overexpress human epidermal growth factor receptor 2 (HER2), suggesting a potential therapeutic target. However, prospective evidence for HER2-directed antibody-drug conjugates (ADCs) in advanced EMPD is lacking. This study aimed to assess the efficacy and safety of trastuzumab emtansine (T-DM1) in patients with HER2-positive advanced EMPD.

Patients and methods

This investigator-initiated, open-label, single-arm, multicentre, prospective phase II trial enrolled Japanese patients with HER2-positive metastatic EMPD who had not received prior HER2-targeted therapy. Patients

SUBMITTER: Arakawa S 

PROVIDER: S-EPMC13650354 | biostudies-literature | 2026 Sep

REPOSITORIES: biostudies-literature