Molecular therapy for papillary craniopharyngioma: a multi-institutional analysis of practice patterns across the RAPID Consortium.
Publication Title
Journal of neuro-oncology
Document Type
Article
Publication Date
8-14-2026
Keywords
Humans; Craniopharyngioma; Pituitary Neoplasms; Female; Proto-Oncogene Proteins B-raf; Retrospective Studies; Male; Adult; Adolescent; Middle Aged; Young Adult; Child; Molecular Targeted Therapy; Practice Patterns, Physicians'; Aged; Mutation; Follow-Up Studies; Treatment Outcome; Child, Preschool; BRAF V600E; Craniopharyngioma; Papillary craniopharyngioma; Radiation; Targeted therapy.; california; santa monica; pni; psjhc
Abstract
PURPOSE: Targeted therapy for BRAF V600E mutant papillary craniopharyngioma (PCP) has been rapidly accepted, though given the rarity of the tumor, there is limited guidance for appropriate use. Here, we retrospectively evaluated current practice patterns for the implementation of targeted therapeutics and their outcomes in the treatment of papillary craniopharyngioma (PCP).
METHODS: Practice patterns at the institutions in the Registry for Adenomas of the Pituitary and Related Disorders (RAPID) for treatment of BRAF V600E mutated PCPs using targeted therapy were evaluated. Baseline clinical and demographic variables were retrospectively recorded. Imaging response was evaluated. Dosing, treatment course, adverse events (AEs) and other therapeutic strategies employing BRAF/MEK inhibitors were studied. Adjuvant and/or salvage therapies, including radiation and surgery were recorded.
RESULTS: Most patients demonstrated at least partial response to BRAF and/or BRAF/MEK inhibition (13/19, 72.2%). No patients progressed through therapy. Grade 0-2 AEs occurred in 79% of cases, and nine (47.4%) patients eventually discontinued therapy. Radiation-sparing regimens demonstrated similar radiographic response rates and had smaller residual tumor volume after treatment completion (23.7 vs. 612.4 mm
CONCLUSION: Critical insights into molecular therapeutic practice patterns for treatment of PCP were identified. Radiation-sparing, mono-therapeutic, and prolonged treatment duration are feasible options and warrant further study.
Area of Special Interest
Neurosciences (Brain & Spine)
Area of Special Interest
Cancer
Specialty/Research Institute
Neurosciences
Specialty/Research Institute
Oncology
Specialty/Research Institute
Pathology & Laboratory Medicine
DOI
10.1007/s11060-026-05739-5
