Table 3.

Previously published studies in pediatric patients with nonmelanoma BRAF-altered cancers.

Drug	Tumor type	Number of patients	ORR or overall response rate	Other comments	Reference
Dabrafenib plus trametinib	BRAF V600E high-grade gliomas	3	N/A	Patient 1 remained disease free for 20 months at which time he presented with disseminated disease recurrence and died 2 months later. Patient 2 has remained on therapy with a small amount of stable disease for 32 months. Patient 3 remained on therapy with stable disease for 23 months.	(48)
Dabrafenib or vemurafenib	BRAF V600E pediatric gliomas	67	80%	Poor prognostic factors in conventional therapies, such as concomitant homozygous deletion of CDKN2A, were not associated with lack of response to BRAF inhibition.	(49)
Dabrafenib	BRAF V600E pediatric low-grade glioma	32	44%		(50)
Dabrafenib plus trametinib	BRAF V600E Wilms tumor	1	N/A	The patient remains in a complete radiographic response 12 months after starting therapy and continues to receive dabrafenib and trametinib with minimal treatment-emergent toxicities.	(51)
Vemurafenib	Recurrent or progressive BRAF V600E mutant brain tumors	19	32%		(52)
Dabrafenib	BRAF V600 mutation—positive tumors	27	Not reported	In this first clinical trial in pediatric patients with pretreated BRAF V600–mutant tumors, dabrafenib was well tolerated while achieving target exposure levels; the average treatment duration was >1 year with many patients still on treatment.	(53)