Artikel
Targeted treatment of papillary craniopharyngiomas harbouring BRAF V600E mutations
ZielgerichteteTherapie der papillären Kraniopharyngeome mit einer BRAF V600E Mutation
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Veröffentlicht: | 26. Juni 2020 |
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Gliederung
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Objective: Craniopharyngiomas are surgically challenging brain tumors. After the operation, the quality of life is often significantly impaired due to neurological and endocrinological complications. Currently, FDA approved systemic treatments are not available for patients in whom craniopharyngiomas recur after surgery and radiation. Papillary craniopharyngiomas (PCP) are characterized by the presence of BRAFV600E mutations. To date, five case reports have been published on the treatment of BRAFV600E mutant papillary craniopharyngiomas with BRAF and/or MEK inhibitors.
Methods: In this study, authors from all previously published reports share their collective experience, provide updated follow-up on their patients, and thus generate an overview of all currently available information on targeted therapy in patients with BRAFV600E mutant PCP. We have also included information on an additional patient with a papillary craniopharyngioma recently treated with BRAF and MEK inhibitors after tumor biopsy alone, in the absence of recurrence, highlighting the potential for a neo-adjuvant therapeutic approach.
Results: All six cases in our series (100%) showed dramatic responses to targeted treatment with BRAF (and MEK) inhibitors. Altogether, our cases are highly promising and informative for patient treatment, although uncertainty remains with regards to the optimal timing, the specific agents (single agent or dual therapy) to be used and the duration of treatment. The ongoing multicenter phase II Alliance A071601 trial (NCT03224767) of vemurafenib and cobimetinib for patients with biopsy-proven residual or recurrent papillary craniopharyngiomas should provide additional information to help guide patient management.
Conclusion: PCPs are characterized by the presence of BRAFV600E mutations, which are emerging as a useful guide for diagnosis and treatment decision-making. The ongoing multicenter phase 2 Alliance A071601 trial is evaluating the efficacy of BRAF and MEK inhibitors for patients with PCP.