https://orcid.org/0000-0001-5242-1761
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Dario De Alcubierre, Grigorios Gkasdaris, Margaux Mordrel, Anthony Joncour, Claire Briet, Fabien Almairac, Julien Boetto, Celine Mouly, Delphine Larrieu-Ciron, Alexandre Vasiljevic, Chiara Villa, Camille Sergeant, François Ducray, Loic Feuvret, Philippe Chanson, Bertrand Baussart, Gerald Raverot, Emmanuel Jouanneau, BRAF and MEK inhibitor targeted therapy in papillary craniopharyngiomas: a cohort study, European Journal of Endocrinology, Volume 191, Issue 2, August 2024, Pages 251–261, https://doi.org/10.1093/ejendo/lvae091
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Abstract
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.
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.
Objective response and clinical and safety outcomes were assessed after 3 months and at the last available follow-up during TT.
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.
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.
