BRAF and MEK inhibitor targeted therapy in papillary craniopharyngiomas: a cohort study
Authors' conclusion
In this study, 94% of patients showed partial response or better to TT. Adverse events were acceptable. Further research is needed to establish standardized protocols; however, these results advocate for a NEO approach in invasive PCPs.
Study context
- Population
- BRAF V600E-mutated papillary craniopharyngioma patients treated with dabrafenib and trametinib, France, April 2019 to July 2023; 8 females, mean age 50.5 ± 15.75 years
- Participants
- 16 patients
- Exposure
- Dabrafenib + trametinib as NEO, ADJ or PAL therapy
- NEOn = 6
- ADJn = 8
- PALn = 2
- Outcome
- Objective response
- Follow-up
- After 3 months and at the last available during TT
Methodological detailsmulticenter
- Multicenter
Main finding
As reported in the abstract
last follow-up (mean 7.6 ± 5.3 months)
Subtotal response
12 patients
Partial response
3 patients
Stable disease
1 patient
Observational data: associations, not effects.
Further findings
| Outcome | NEO | ADJ | PAL |
|---|---|---|---|
| Volume reduction · last follow-up | mean 88.9 ± 4.4 % | mean 73.3 ± 23.4 % | mean 91.8 ± 4.3 % |
Clinical outcomes
- Headaches resolved
- 5/5
- Visual impairment resolved
- 6/9
- Improved neurological symptoms
- 2/3
- Weight loss
- 1/4
- Recovered endocrine function
- 2/14
Safety
Tolerability and adverse events
- Well-tolerated
- 62.5% of cases
- Adverse events leading to treatment discontinuation
- 5 patients
- Definitive discontinuation
- 3 cases
Original publication
BRAF and MEK inhibitor targeted therapy in papillary craniopharyngiomas: a cohort study. Eur J Endocrinol. Published 19 August 2024. doi:10.
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