Case Reports:

Regression of BRAFV600E mutant adult glioblastoma after primary combined BRAF-MEK inhibitor targeted therapy: a report of two cases

Peter Y.M. Woo, Tai-Chung Lam, Jenny K.S. Pu, Lai-Fung Li, Roland C.Y. Leung, Jason M.K. Ho, James T.F. Zhung, Belinda Wong, Timonthy S.K. Chan, Herbert H.F. Loong and Ho-Keung Ng

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Oncotarget. 2019; 10:3818-3826. https://doi.org/10.18632/oncotarget.26932

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Peter Y.M. Woo1, Tai-Chung Lam2, Jenny K.S. Pu3, Lai-Fung Li3, Roland C.Y. Leung4, Jason M.K. Ho1, James T.F. Zhung5, Belinda Wong6, Timonthy S.K. Chan7, Herbert H.F. Loong8 and Ho-Keung Ng9

1 Department of Neurosurgery, Kwong Wah Hospital, Hong Kong

2 Department of Clinical Oncology, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong

3 Department of Neurosurgery, Queen Mary Hospital, Hong Kong

4 Department of Medicine, Queen Mary Hospital, Hong Kong

5 Department of Neurosurgery, Kwong Wah Hospital, Hong Kong

6 Pharmacy and Medical Therapeutics, Kwong Wah Hospital, Hong Kong

7 Department of Pathology, Kwong Wah Hospital, Hong Kong

8 Department of Clinical Oncology, The Chinese University of Hong Kong, Hong Kong

9 Department of Anatomical and Cellular Pathology, The Chinese University of Hong Kong, Hong Kong

Correspondence to:

Tai-Chung Lam,email: [email protected]

Keywords: glioblastoma; BRAFV600E mutation; targeted therapies; BRAF-MEK inhibitors

Received: August 20, 2018     Accepted: April 03, 2019     Published: June 04, 2019



Up to 15% of young adults with glioblastoma have the activating oncogenic BRAFV600E mutation, an actionable target of the MAPK signal transduction pathway governing tumor cell proliferation. Small molecule inhibitors of BRAF and MEK, a downstream protein immediately following BRAF, have been shown to confer a survival advantage for patients with BRAFV600E mutant advanced melanoma. We describe our experience using this combined target therapy for two patients with BRAFV600E mutant glioblastoma (GBM) as primary treatment due to extenuating clinical circumstances that prohibited the prescription of standard treatment.

Case Presentation

The two patients were both 22 years old on presentation. After the initial tumor resection, they both developed rapid deterioration in performance status within a few weeks due to leptomeningeal metastases. In view of the critical condition, BRAF and MEK inhibitors were prescribed as first line treatment. The two patients both achieved dramatic clinical response, which was parallel to the impressive radiological regression of the disease. Unfortunately, the duration of disease control was short as drug resistance developed rapidly. The two patients died 7 and 7.5 month after initial diagnosis of GBM.


Primary treatment with inhibitors of BRAF and MEK can lead to tumor regression for patients with BRAFV600E mutant glioblastoma. We therefore recommend that all young GBM patients should undergo BRAFV600E mutation testing, especially for those with unusual aggressive clinical course.

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