gms | German Medical Science

71. Jahrestagung der Deutschen Gesellschaft für Neurochirurgie (DGNC)
9. Joint Meeting mit der Japanischen Gesellschaft für Neurochirurgie

Deutsche Gesellschaft für Neurochirurgie (DGNC) e. V.

21.06. - 24.06.2020

Targeted treatment of papillary craniopharyngiomas harbouring BRAF V600E mutations

ZielgerichteteTherapie der papillären Kraniopharyngeome mit einer BRAF V600E Mutation

Meeting Abstract

  • presenting/speaker Tareq Juratli - Universitätsklinikum Carl Gustav Carus Dresden, Klinik für Neurochirurgie, Dresden, Deutschland; Massachusetts General Hospital, Department of Neurosurgery, Boston, MA, United States
  • Pamela Jones - Massachusetts General Hospital, Department of Neurosurgery, Boston, MA, United States
  • Nancy Wang - Massachusetts General Hospital, Division of Neuro-Oncology, Department of Neurology, Boston, MA, United States
  • Frederick Barker - Massachusetts General Hospital, Department of Neurosurgery, Boston, MA, United States
  • Yazmin Odia - Baptist Health South Florida, Miami Cancer Institute, Miami, FL, United States
  • Elham Rostami - Uppsala University, Medical Clinic IV Endocrinology, Uppsala, Sweden
  • Daniel Cahill - Massachusetts General Hospital, Department of Neurosurgery, Boston, MA, United States
  • Sandro Santagata - Brigham and Women’s Hospital, Department of Pathology, Boston, MA, United States
  • Priscilla Brastianos - Massachusetts General Hospital, Division of Neuro-Oncology, Department of Neurology, Boston, MA, United States

Deutsche Gesellschaft für Neurochirurgie. 71. Jahrestagung der Deutschen Gesellschaft für Neurochirurgie (DGNC), 9. Joint Meeting mit der Japanischen Gesellschaft für Neurochirurgie. sine loco [digital], 21.-24.06.2020. Düsseldorf: German Medical Science GMS Publishing House; 2020. DocP128

doi: 10.3205/20dgnc413, urn:nbn:de:0183-20dgnc4134

Veröffentlicht: 26. Juni 2020

© 2020 Juratli et al.
Dieser Artikel ist ein Open-Access-Artikel und steht unter den Lizenzbedingungen der Creative Commons Attribution 4.0 License (Namensnennung). Lizenz-Angaben siehe http://creativecommons.org/licenses/by/4.0/.


Gliederung

Text

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.