Prolonged survival of a patient with metastatic leptomeningeal melanoma treated with BRAF inhibition-based therapy: a case report

Abstract

Background: Leptomeningeal metastasis of melanoma is a devastating complication with a grave prognosis, and there are no known effective standard treatments. Although selective BRAF inhibitors have demonstrated a significant clinical activity in patients with metastatic melanoma harboring a BRAF mutation, the clinical benefit of BRAF inhibitor-based therapy in leptomeningeal disease is not clear.

Case presentation: We present a case of prolonged survival of a patient with BRAF V600E-mutant leptomeningeal disease who was treated with vemurafenib followed by whole brain radiation and a combination of dabrafenib and trametinib. Both vemurafenib and the sequential treatment of radiation and dabrafenib/trametinib led to regression of the leptomeningeal disease, and the patient survived for 19 months after the diagnosis of the leptomeningeal disease.

Conclusion: This case suggests a possible clinically meaningful benefit of BRAF inhibitor-based therapy and a need for close investigation of this therapeutic approach in patients with this devastating disease.

Fig. 1

Brain MRI showing the response and the progression of the leptomeningeal disease with BRAF inhibitor-based therapy. The arrows indicate the enhancement of the leptomeninges in April 2013. After vemurafenib therapy, the follow-up images revealed initial regression of the leptomeningeal disease. He had progression of the leptomeningeal disease in October 2013 and started whole brain radiation followed by dabrafenib plus trametinib. The follow-up scans showed regressed leptomeningeal disease