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Review
. 2025 Oct 30;176(1):58.
doi: 10.1007/s11060-025-05318-0.

Efficacy and safety of BRAF-MEK dual inhibition in BRAF-V600E mutated papillary craniopharyngioma: a systematic review

Beste Gülsuna  1 , Baylee Stevens  1 , Belda Gülsuyu  2 , Ethan Wood  3 , Timurhan Aksoy  4 , Erika Santos Horta  1 , Ian F Dunn  1 , Christopher S Graffeo  5
Affiliations
Review

Efficacy and safety of BRAF-MEK dual inhibition in BRAF-V600E mutated papillary craniopharyngioma: a systematic review

Beste Gülsuna et al. J Neurooncol. .

Abstract

Purpose: Papillary craniopharyngioma (PCP) is a rare central nervous system tumor with high recurrence and significant morbidity. The BRAF V600E mutation has emerged as a potential target for treatment, with BRAF-MEK inhibitors showing promise in early studies. However, their efficacy and safety remain uncertain due to limited data from small-scale studies and case reports. This systematic review aims to evaluate the clinical outcomes of dual BRAF-MEK inhibitor therapy in BRAF V600E-mutated PCP.

Methods: A systematic search of OVID Medline, Embase, Scopus, PubMed, and Web of Science was performed following PRISMA guidelines. Studies reporting clinical outcomes of BRAF-MEK inhibitors in PCP were included. Due to the predominance of case reports, a narrative systematic review was performed without meta-analysis.

Results: Twenty-one studies involving 54 patients met inclusion criteria. Treatment was administered as neoadjuvant (n = 11), adjuvant (n = 34), or palliative (n = 9) settings. Most reports were case-based; two cohort studies (Brastianos et al., n = 16; De Alcubierre et al., n = 14) reported volumetric response rates of 93.8% and 92.9%, respectively. Common presenting symptoms included hypopituitarism (60.5%), visual changes (47.3%), and headache (44.7%). Median therapy duration was 5 months (range, 1.5-31); 6 months (3-16) for neoadjuvant, 4.5 months (1.5-21) for adjuvant and 7 months (2-31) for palliative use. Median tumor volume reduction was 89% overall (neoadjuvant 90%, adjuvant 85%, palliative 88%). Reported toxicities were generally manageable.

Conclusion: Dual BRAF-MEK inhibition demonstrates robust tumor responses and a favorable safety profile across neoadjuvant, adjuvant, and palliative settings in PCP. These preliminary findings support further investigation to define long-term efficacy, safety, and integration into clinical protocols.

Keywords: BRAF; Craniopharyngioma; Inhibitors; MEK; Papillary; Systematic review; Targeted therapy.

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Conflict of interest statement

Declarations. Human ethics and consent to participate: Not applicable. Competing interests: The authors declare no competing interests.

References

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