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Ref Type Journal Article
PMID (38960393)
Authors Lochrin SE, Buonocore DJ, Young RJ, Kaley TJ, Postow MA, Wolchok JD, Shoushtari AN, Momtaz P, Betof Warner AS, Callahan MK
Title Durable complete response in a patient with leptomeningeal melanoma after treatment with dabrafenib, trametinib, and nivolumab.
Journal Vol Issue Date Pages Journal Short Title
Pigment cell & melanoma research 2024 Jul 03 Pigment Cell Melanoma Res
URL
Abstract Text Leptomeningeal disease (LMD) is a devastating complication of melanoma with a dismal prognosis. We present the case of a young man with stage IV BRAF V600E mutant melanoma with lung, lymph node, and brain metastases initially treated with ipilimumab and nivolumab, who subsequently developed LMD. Upon change to BRAF/MEK targeted therapy with nivolumab, a durable complete response was achieved and remains ongoing, off treatment, 7 years from diagnosis. Management of symptomatic LMD remains a critical unmet clinical challenge, with limited clinical trial data. This exceptional case is instructive, as the first published case of the use of the triplet, and the first durable response with therapy discontinuation, in melanoma LMD. The triple-drug regimen may be considered a viable option in fit patients. This case highlights the potential for long-term disease control and the critical and urgent need to develop clinical trials inclusive of patients with LMD to define the best treatment strategies.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
BRAF V600E skin melanoma predicted - sensitive Dabrafenib + Nivolumab + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Mekinist (trametinib), Tafinlar (dabrafenib), and Opdivo (nivolumab) resulted in a partial response in the leptomeningeal disease on the spine and the extracranial melanoma, with the intracranial lesions remaining stable after 6 months, in a patient with cutaneous melanoma harboring BRAF V600E, and a follow-up at least 7 years following diagnosis demonstrated complete remission (PMID: 38960393). 38960393