Abstract：
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.
摘要：
脑膜疾病（LMD）是黑色素瘤的破坏性并发症，预后不良。我们介绍了一个年轻男子IV期BRAFV600E突变黑色素瘤肺，淋巴结,最初用ipilimumab和nivolumab治疗的脑转移瘤，后来开发了LMD。改用纳武单抗进行BRAF/MEK靶向治疗后，实现了持久的全面回应，并且仍在继续，关闭治疗，诊断后7年。有症状的LMD的管理仍然是一个关键的未满足的临床挑战，临床试验数据有限。这个特例很有启发性,作为第一个使用三元组的公开案例，和治疗中断的第一个持久反应，黑色素瘤LMD。三联药物方案可以被认为是适合患者的可行选择。该案例强调了长期疾病控制的潜力，以及开发包括LMD患者在内的临床试验以确定最佳治疗策略的关键和紧迫需求。
