BRAF–MEK Inhibition in Newly Diagnosed Papillary Craniopharyngiomas
Authors' conclusion
In this small, single-group study involving patients with papillary craniopharyngiomas, 15 of 16 patients had a partial response or better to the BRAF-MEK inhibitor combination vemurafenib-cobimetinib.
Study context
- Population
- Patients with BRAF-mutated papillary craniopharyngiomas, no previous radiation therapy, measurable disease
- Participants
- 16 patients
- Outcome
- Objective response by centrally determined volumetric data
- Follow-up
- 4 months
Main finding
As reported in the abstract
Durable objective partial response or better
Durable objective partial response or better (of 16 patients)
15
94%; 95% CI, 70 to 100
Further findings
Tumour volume reduction
- Median reduction in tumour volume
- 91% (range, 68 to 99)
Progression-free survival · 12 and 24 months
- PFS at 12 months
- 87% (95% CI, 57 to 98)
- PFS at 24 months
- 58% (95% CI, 10 to 89)
- Disease progression during follow-up after therapy discontinued
- 3 patients
- Deaths
- 0
- Median follow-up 22 months (95% CI, 19 to 30)
Safety
Adverse events
- Grade 3 AEs at least possibly treatment-related
- 12 patients
- Grade 3 rash
- 6 patients
- Grade 4 AEs (hyperglycemia 1, increased creatine kinase 1)
- 2 patients
- Discontinued owing to AEs
- 3 patients
- Sole non-responder stopped after 8 days owing to toxic effects
- 1 patient
Original publication
BRAF–MEK Inhibition in Newly Diagnosed Papillary Craniopharyngiomas. N Engl J Med. Published 13 July 2023. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →