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- Title
BRAF and MEK inhibitor targeted therapy in papillary craniopharyngiomas: a cohort study.
- Authors
Alcubierre, Dario De; Gkasdaris, Grigorios; Mordrel, Margaux; Joncour, Anthony; Briet, Claire; Almairac, Fabien; Boetto, Julien; Mouly, Celine; Larrieu-Ciron, Delphine; Vasiljevic, Alexandre; Villa, Chiara; Sergeant, Camille; Ducray, François; Feuvret, Loic; Chanson, Philippe; Baussart, Bertrand; Raverot, Gerald; Jouanneau, Emmanuel
- Abstract
Objective Targeted therapy (TT) with BRAF/MEK inhibitors has emerged as a potential treatment in papillary craniopharyngiomas (PCPs). However, standardized data on large cohorts are lacking. Our study aimed to assess real-life efficacy and safety of BRAF/MEK inhibition in patients with PCPs. Design Retrospective French multicenter study involving BRAF V600E–mutated PCP patients, treated with BRAF/MEK inhibitor combination dabrafenib and trametinib, from April 2019 to July 2023. Methods Objective response and clinical and safety outcomes were assessed after 3 months and at the last available follow-up during TT. Results Sixteen patients (8 females, mean age 50.5 ± 15.75 years), receiving either neoadjuvant therapy (NEO) for non-resectable tumors (n = 6), post-surgical adjuvant therapy (ADJ; n = 8), or palliative therapy (PAL) following failure of multimodal treatment (n = 2), were included. At the last follow-up (mean 7.6 ± 5.3 months), 12 patients showed subtotal response, 3 exhibited partial response, and 1 maintained stable disease. Mean volume reduction was 88.9 ± 4.4%, 73.3 ± 23.4%, and 91.8 ± 4.3% in the NEO, ADJ, and PAL groups, respectively. Targeted therapy resolved headaches in 5/5 patients and visual impairment in 6/9; 2/3 patients had improved neurological symptoms, 1/4 presented weight loss, and 2/14 recovered endocrine function. Targeted therapy was well-tolerated in 62.5% of cases; adverse events led to treatment discontinuation in 5 patients and definitive discontinuation in 3 cases. Conclusions 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.
- Subjects
TERMINATION of treatment; NEOADJUVANT chemotherapy; COMBINED modality therapy; BRAF genes; WEIGHT loss
- Publication
European Journal of Endocrinology, 2024, Vol 191, Issue 2, p251
- ISSN
0804-4643
- Publication type
Article
- DOI
10.1093/ejendo/lvae091