Efficacy of T-DM1 for leptomeningeal and brain metastases in a HER2 positive metastatic breast cancer patient: new directions for systemic therapy - a case report and literature review

BMC Cancer. 2018 Jan 25;18(1):97. doi: 10.1186/s12885-018-3994-5.


Background: Herein, we report a complete response after whole brain radiotherapy (WBRT) and concomitant T-DM1 in a patient with HER2-positive metastatic breast cancer (MBC) and extensive brain and leptomeningeal involvement.

Case presentation: A 46 years old Caucasian woman with HER2-positive MBC and no baseline CNS involvement, started in August 2015 1st line therapy with Pertuzumab-Trastuzumab-Docetaxel, with partial response. However, in April 2016 the patient eventually progressed with emergence of brain and leptomeningeal metastases. Hence, she started in May 2016 2nd line therapy with T-DM1 and concomitant WBRT, with complete response (CR) after 3 courses of therapy, with complete resolution of neurological symptoms and no relevant toxicities. The CR is lasting over 13 months and the patient is out of corticosteroid use.

Conclusions: To the best of our knowledge, this is the first case reporting interesting antitumor activity of T-DM1 and concomitant WBRT in both brain and leptomeningeal metastases, with a favorable safety profile and prolonged extracranial disease control. Further prospective studies should confirm these findings.

Keywords: Brain metastases; HER2-positive; Leptomeningeal metastases; T-DM1; Trastuzumab Emtansine; Whole brain radiotherapy.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Ado-Trastuzumab Emtansine
  • Antibodies, Monoclonal, Humanized / administration & dosage
  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage
  • Brain Neoplasms / drug therapy*
  • Brain Neoplasms / pathology
  • Brain Neoplasms / radiotherapy
  • Brain Neoplasms / secondary
  • Breast Neoplasms / drug therapy*
  • Breast Neoplasms / genetics
  • Breast Neoplasms / pathology
  • Breast Neoplasms / radiotherapy
  • Combined Modality Therapy
  • Docetaxel
  • Female
  • Humans
  • Maytansine / administration & dosage
  • Maytansine / adverse effects
  • Maytansine / analogs & derivatives*
  • Meningeal Neoplasms / drug therapy*
  • Meningeal Neoplasms / pathology
  • Meningeal Neoplasms / radiotherapy
  • Meningeal Neoplasms / secondary
  • Middle Aged
  • Receptor, ErbB-2 / genetics
  • Taxoids / administration & dosage
  • Trastuzumab / administration & dosage*
  • Trastuzumab / adverse effects


  • Antibodies, Monoclonal, Humanized
  • Taxoids
  • Maytansine
  • Docetaxel
  • ERBB2 protein, human
  • Receptor, ErbB-2
  • pertuzumab
  • Trastuzumab
  • Ado-Trastuzumab Emtansine