Evidence map›Paper›PMID 41165932›Full record

SynthesisJournal of neuro-oncology2025

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

Beste Gülsuna, Baylee Stevens, Belda Gülsuyu, Ethan Wood, Timurhan Aksoy, Erika Santos Horta, Ian F Dunn, Christopher S Graffeo

Erratum issuedAbstract readSystematic Review
PubMed Publisher
In one paragraph

Synthesis in Journal of neuro-oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
  2. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

8 authors.

Beste GülsunaDepartment of Neurosurgery, University of Oklahoma Health Sciences Center, 1000 N Lincoln Blvd, #4000, Oklahoma City, OK, 73104, USA.
Baylee StevensDepartment of Neurosurgery, University of Oklahoma Health Sciences Center, 1000 N Lincoln Blvd, #4000, Oklahoma City, OK, 73104, USA.
Belda GülsuyuDepartment of Neurological Surgery, University of California San Francisco, San Francisco, CA, USA.
Ethan WoodCollege of Osteopathic Medicine, Oklahoma State University, Tulsa, OK, USA.
Timurhan AksoyDokuz Eylül University Faculty of Medicine, Izmir, Türkiye.
Erika Santos HortaDepartment of Neurosurgery, University of Oklahoma Health Sciences Center, 1000 N Lincoln Blvd, #4000, Oklahoma City, OK, 73104, USA.
Ian F DunnDepartment of Neurosurgery, University of Oklahoma Health Sciences Center, 1000 N Lincoln Blvd, #4000, Oklahoma City, OK, 73104, USA.
Christopher S GraffeoDepartment of Neurosurgery, University of Oklahoma Health Sciences Center, 1000 N Lincoln Blvd, #4000, Oklahoma City, OK, 73104, USA. Graffeo@gmail.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposePapillary 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.

methodsA 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.

resultsTwenty-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.

conclusionDual 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.

Indexed as

CraniopharyngiomaPituitary NeoplasmsProtein Kinase InhibitorsProto-Oncogene Proteins B-rafHumansMutationBRAF protein, humanProtein Kinase InhibitorsProto-Oncogene Proteins B-rafBRAFCraniopharyngiomaInhibitorsMEKPapillarySystematic reviewTargeted therapy

Identifiers

What Socratic holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.