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Ref Type Journal Article
PMID (31502039)
Authors Philippe L, Maria K, Tariq A, Sofia M, Christine SM, Jean-Pierre F, Dudley RW, Sébastien P, Nada J, Valérie L
Title Efficacy of Dabrafenib for three children with brainstem BRAFV600E positive ganglioglioma.
Journal Vol Issue Date Pages Journal Short Title
Journal of neuro-oncology 145 1 2019 Oct 135-141 J Neurooncol
URL
Abstract Text 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.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
BRAF V600E ganglioglioma predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical study, Tafinlar (dabrafenib) treatment resulted in symptomatic improvement and clinical efficacy in three pediatric patients with brainstem ganglioglioma harboring BRAF V600E, with decreased tumor volume and treatment continuing for at least 5.5 years in one patient, a significant partial response in another patient, and a partial response in a third patient with tumor regression and ongoing response for at least 2 years (PMID: 31502039). 31502039