Manuel Rodrigues, Vincent Servois, Pascale Mariani, Marc Pracht, Caroline Dutriaux, Franck Grillet, Thomas Ryckwaert, Agnès Ducoulombier, Eve-Marie Neidhardt, Sophie Piperno-Neumann
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A French multidisciplinary panel developed evidence-based guidelines for MUM management presented in this review. Recommendations emphasise on comprehensive diagnosis, including liver biopsy and imaging, circulating tumour DNA (ctDNA) analysis, and high-definition HLA typing for HLA-A*02:01. Local therapies are proposed for patients with limited hepatic metastases, from liver surgery to isolated hepatic perfusion and chemoembolisation for patients with more extensive hepatic involvement. Systemic therapy with tebentafusp is the standard of care for HLA-A*02:01-positive patients. For HLA-A*02:01-negative patients with extensive disease, treatment options are limited. They are encouraged to participate in a clinical trial, alternatively, percutaneous hepatic perfusion, ICI alone or in combination can be proposed. Treatment efficacy assessment includes response evaluation criteria in solid tumours (RECIST), tumour growth rate (TGR) analysis, and ctDNA dynamics. 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引用次数: 0
摘要
葡萄膜黑色素瘤是一种起源于葡萄膜黑色素细胞的罕见恶性肿瘤。尽管原发肿瘤得到有效控制,但转移性葡萄膜黑色素瘤(MUM)发生在约20-30%的患者中,主要影响肝脏,预后和总生存期(OS)较差。UM独特的分子特征,缺乏BRAF、NRAS和KIT突变,限制了靶向治疗的效果。化疗和免疫检查点抑制剂(ICIs)也显示出有限的益处,而tebentafusp已成为第一个改善OS的药物,但这种全身治疗只能用于HLA-A*02:01阳性患者。一个法国多学科小组制定了以证据为基础的MUM管理指南。建议强调全面诊断,包括肝活检和影像学,循环肿瘤DNA (ctDNA)分析,HLA- a *02:01的高清HLA分型。对于有限的肝转移患者,建议局部治疗,从肝脏手术到孤立的肝脏灌注和对更广泛的肝脏累及患者的化疗栓塞。tebentafusp是HLA-A*02:01阳性患者的标准治疗方案。对于广泛患病的HLA-A*02:01阴性患者,治疗选择有限。他们被鼓励参加临床试验,或者,经皮肝灌注,ICI单独或联合可以提出。治疗效果评估包括实体肿瘤反应评价标准(RECIST)、肿瘤生长速率(TGR)分析和ctDNA动态。这一共识为法国肿瘤学家优化MUM管理、整合局部区域干预、系统治疗和生物标志物以提高患者预后提供了实用指南。
Management of metastatic uveal melanoma: French expert consensus guidelines.
Uveal melanoma (UM) is a rare malignancy originating from uveal melanocytes. Despite effective control of the primary tumour, metastatic uveal melanoma (MUM) occurs in approximately 20-30% of patients, primarily affecting the liver, with a poor prognosis and overall survival (OS). The unique molecular profile of UM, lacking BRAF, NRAS, and KIT mutations, limits targeted therapy efficacy. Chemotherapy and immune checkpoint inhibitors (ICIs) also show limited benefits, while tebentafusp has emerged as the first drug to improve OS, but this systemic treatment can be used only in HLA-A*02:01-positive patients. A French multidisciplinary panel developed evidence-based guidelines for MUM management presented in this review. Recommendations emphasise on comprehensive diagnosis, including liver biopsy and imaging, circulating tumour DNA (ctDNA) analysis, and high-definition HLA typing for HLA-A*02:01. Local therapies are proposed for patients with limited hepatic metastases, from liver surgery to isolated hepatic perfusion and chemoembolisation for patients with more extensive hepatic involvement. Systemic therapy with tebentafusp is the standard of care for HLA-A*02:01-positive patients. For HLA-A*02:01-negative patients with extensive disease, treatment options are limited. They are encouraged to participate in a clinical trial, alternatively, percutaneous hepatic perfusion, ICI alone or in combination can be proposed. Treatment efficacy assessment includes response evaluation criteria in solid tumours (RECIST), tumour growth rate (TGR) analysis, and ctDNA dynamics. This consensus provides practical guidelines for French oncologists to optimise MUM management, integrating locoregional interventions, systemic therapies, and biomarkers to enhance patient outcomes.