Efficacy of Dabrafenib for three children with brainstem BRAFV600E positive ganglioglioma

  • Laflamme Philippe
  • Kondyli Maria
  • Aljared Tariq
  • Miconiatis Sofia
  • Saint-Martin Christine
  • Farmer Jean-Pierre
  • Roy W. Dudley
  • Perreault Sébastien
  • Jabado Nada
  • Larouche ValérieEmail author
Clinical Study



Children with unresectable brainstem-infiltrated ganglioglioma have poor progression-free survival when treated with conventional chemotherapy and radiation regimens. The BRAFV600E mutation occurs in a large number of gangliogliomas, making them amenable for targeted therapy using mutation-specific kinase inhibitors. However, limited data exists on the effectiveness and best treatment duration of these inhibitors in this tumor setting.


Retrospective description of three cases of childhood brainstem ganglioglioma with BRAFV600E mutation treated in the long-term with Dabrafenib, a specific BRAFV600E kinase inhibitor.


Dabrafenib resulted in rapid tumoral regression and significant and durable clinical and radiological improvement. However, all patients had rapid clinical and radiological relapse within days to weeks following treatment discontinuation but showed similar rapid and sustained therapeutic response when Dabrafenib was re-introduced. This targeted therapy has been well tolerated despite its long-term use of 4.8 to 5.5 years in the three patients.


Dabrafenib is effective and seemingly safe and well tolerated in our three patients. We observed sustained chemosensitivity even when re-introducing this kinase inhibitor after its discontinuation after 2 years of therapy. These cases indicate the need to re-evaluate the timing and means of Dabrafenib discontinuation in pediatric patients with BRAFV600E mutated gangliogliomas and better assess the future implications of its long-term use.


Ganglioglioma Central nervous system Dabrafenib BRAFV600E Children 


Compliance with ethical standards

Conflict of interest

The authors declare that they have no conflict of interest.


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Copyright information

© Springer Science+Business Media, LLC, part of Springer Nature 2019

Authors and Affiliations

  • Laflamme Philippe
    • 1
  • Kondyli Maria
    • 2
  • Aljared Tariq
    • 3
  • Miconiatis Sofia
    • 4
  • Saint-Martin Christine
    • 5
  • Farmer Jean-Pierre
    • 3
  • Roy W. Dudley
    • 3
  • Perreault Sébastien
    • 6
  • Jabado Nada
    • 7
  • Larouche Valérie
    • 8
    Email author
  1. 1.Department of MedicineLaval UniversityQuébecCanada
  2. 2.Department of MedicineMontreal UniversityMontrealCanada
  3. 3.Division of Neurosurgery, Department of Pediatric Surgery, Montreal Children’s HospitalMcGill University Health CenterMontrealCanada
  4. 4.Department of PathologyMcGill University Health CenterMontrealCanada
  5. 5.Department of Medical Imaging, Montreal Children’s HospitalMcGill University Health CenterMontrealCanada
  6. 6.Department of PediatricsCentre Hospitalier Universitaire Sainte-JustineMontrealCanada
  7. 7.Department of PediatricsMcGill University and McGill University Heath Centre Research InstituteMontrealCanada
  8. 8.Department of PediatricsCentre Mère-enfant Soleil du CHU de Québec-Université LavalQuébecCanada

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