Prolonged disease control with MEK inhibitor in neurofibromatosis type I-associated glioblastoma

Abstract

WHAT IS KNOWN AND OBJECTIVE: Neurofibromatosis is associated with overactivation of the RAS-MAPK pathway. MEK inhibitors have been shown to be an effective treatment modality in other malignancies. CASE SUMMARY: We present a 24-year-old male with treatment-refractory neurofibromatosis-associated glioblastoma, who experienced clinical and radiological benefit from the MEK inhibitor, trametinib. WHAT IS NEW AND CONCLUSION: This case highlights the therapeutic success of a MEK inhibitor in neurofibromatosis-associated glioblastoma. As a corollary, this should prompt evaluation of MEK inhibitors in tumours associated with neurofibromatosis. It remains to be elucidated if tumours with somatic NF1..