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Dabrafenib–trametinib is effective in paediatric high-grade glioma

Dabrafenib–trametinib is effective in paediatric high-grade glioma Research highlights Targeted therapy https://doi.org/10.1038/s41571-023-00820-8 Dabrafenib–trametinib is effective in paediatric high-grade glioma Children with recurrent high-grade glioma typically receive combination chemo- therapy, although these regimens pro- vide limited benefit and are highly toxic. Approximately 5–10% of these patients have V600E BRAF -mutant tumours. Now, data from a phase II trial demonstrate the efficacy of the BRAF inhibitor dabrafenib combined with the MEK inhibitor trametinib in this patient subset. In a cohort of this trial, 41 patients between 1 and 18 years of age received the dabrafenib–trametinib combination. The most common histologies were glio- blastoma multiforme (n = 13) and anaplastic pleomorphic xanthoastrocytoma (n = 6). Objective response rate (ORR) was the primary end point. The ORR and complete response rate were 56.1% and 29.3%, respectively, with responses observed regardless of tumour histology. At a median follow-up duration of 25.1 months, the median duration of response was 22.2 months. Regardless of the best overall response, 84% and 60% of patients had ≥50% and 100% reductions in target lesions from baseline measure- ments, respectively. Independent review committee-assessed median progression- free survival was 9.0 months. Median overall survival was 32.8 months. Treatment-related adverse events of any grade and of grade ≥3 occurred in 82.9% and 26.8% of patients, http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Nature Reviews Clinical Oncology Springer Journals

Dabrafenib–trametinib is effective in paediatric high-grade glioma

Nature Reviews Clinical Oncology , Volume 20 (11) – Nov 1, 2023

Dabrafenib–trametinib is effective in paediatric high-grade glioma

Abstract

Research highlights Targeted therapy https://doi.org/10.1038/s41571-023-00820-8 Dabrafenib–trametinib is effective in paediatric high-grade glioma Children with recurrent high-grade glioma typically receive combination chemo- therapy, although these regimens pro- vide limited benefit and are highly toxic. Approximately 5–10% of these patients have V600E BRAF -mutant tumours. Now, data from a phase II trial demonstrate the efficacy of the BRAF inhibitor dabrafenib combined with the...
 
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References (1)

Publisher
Springer Journals
Copyright
Copyright © Springer Nature Limited 2023
ISSN
1759-4774
eISSN
1759-4782
DOI
10.1038/s41571-023-00820-8
Publisher site
See Article on Publisher Site

Abstract

Research highlights Targeted therapy https://doi.org/10.1038/s41571-023-00820-8 Dabrafenib–trametinib is effective in paediatric high-grade glioma Children with recurrent high-grade glioma typically receive combination chemo- therapy, although these regimens pro- vide limited benefit and are highly toxic. Approximately 5–10% of these patients have V600E BRAF -mutant tumours. Now, data from a phase II trial demonstrate the efficacy of the BRAF inhibitor dabrafenib combined with the MEK inhibitor trametinib in this patient subset. In a cohort of this trial, 41 patients between 1 and 18 years of age received the dabrafenib–trametinib combination. The most common histologies were glio- blastoma multiforme (n = 13) and anaplastic pleomorphic xanthoastrocytoma (n = 6). Objective response rate (ORR) was the primary end point. The ORR and complete response rate were 56.1% and 29.3%, respectively, with responses observed regardless of tumour histology. At a median follow-up duration of 25.1 months, the median duration of response was 22.2 months. Regardless of the best overall response, 84% and 60% of patients had ≥50% and 100% reductions in target lesions from baseline measure- ments, respectively. Independent review committee-assessed median progression- free survival was 9.0 months. Median overall survival was 32.8 months. Treatment-related adverse events of any grade and of grade ≥3 occurred in 82.9% and 26.8% of patients,

Journal

Nature Reviews Clinical OncologySpringer Journals

Published: Nov 1, 2023

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