Over 90% of papillary craniopharyngiomas have BRAF V600E mutations. Thus this phase 2 study of 16 patients with measurable tumor receiving the BRAF–MEK inhibitor combination vemurafenib–cobimetinib. After a median of 8 cycles, 15 of 16 patients had at least a partial radiographic response to treatment with a median tumor volume reduction of 90%. Progression-free survival at 2 years was 58%. Grade 3 toxicity occurred in 12 patients (75%) and grade 4 in 2 (17%). While this is an exciting new treatment option, it will be important to compare efficacy and toxicity outcomes to those with modern stereotactic radiation. | Brastianos, N Engl J Med 2023
