Promising response to vemurafenib and cobimetinib treatment for BRAF V600E mutated craniopharyngioma: a case report and literature review

Abstract

Craniopharyngiomas are tumors that arise from the remnants of Rathke's pouch along the nasopharynx to the diencephalon. Current standard of care includes maximal surgical resection versus adjuvant radiation if a maximal resection is unfeasible. Pharmacological therapy with MAPK targeted agents is an emerging therapeutic option for tumors with BRAF V600E mutations. We report a 45-year-old male with a strictly third ventricle papillary craniopharyngioma with a BRAF V600E mutation. After initial surgery with subtotal resection, the patient demonstrated durable response to targeted BRAF and MEK inhibitor therapy with vemurafenib and cobimetinib. Our report suggests that targeted therapy may reduce the need for radiation and impact surgical interventions in select cases.

Practice points

• We report a case of strictly third ventricle papillary craniopharyngioma which is a rare neoplasm accounting for 0.7–11% of all craniopharyngiomas.

• Surgical complexities and deferred radiation led to exploration of alternative therapies.

• Combination vemurafenib and cobimetinib yielded significant tumor reduction.

• Targeting the prevalent BRAF V600E mutation provides a promising alternative to traditional treatments.

• Dual BRAF/MEK inhibitors emerge as a potential adjuvant therapy post-surgery in this tumor entity.

Keywords: :

• BRAF inhibitors
• BRAF mutation
• brain tumor
• craniopharyngioma
• MEK inhibitors

Author contributions

All listed authors participated in the writing of the manuscript and have read and approved the final version.

Financial disclosure

O Aboud is supported in part by the UC Davis Paul Calabresi Career Development Award for Clinical Oncology as funded by the National Cancer Institute/National Institutes of Health through grant #2K12CA138464-11. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Competing interests disclosure

The authors have no competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Writing disclosure

No writing assistance was utilized in the production of this manuscript.

Ethical conduct of research

The authors state that they have obtained appropriate institutional review board approval (IRB ID: 2001013-1) or have followed the principles outlined in the Declaration of Helsinki for all human or animal experimental investigations.

Acknowledgments

The authors would like to thank R O'Donnell for his critical review of this manuscript.