Schank Timo E, Hassel Jessica C
Department of Dermatology, University Hospital Heidelberg, 69120 Heidelberg, Germany.
National Center for Tumor Diseases, University Hospital Heidelberg, 69120 Heidelberg, Germany.
Cancers (Basel). 2019 Jul 24;11(8):1048. doi: 10.3390/cancers11081048.
Uveal melanoma is the most common primary intraocular malignancy among adults. It is, nevertheless, a rare disease, with an incidence of approximately one case per 100,000 individuals per year in Europe. Approximately half of tumors will eventually metastasize, and the liver is the organ usually affected. No standard-of-care treatment exists for metastasized uveal melanoma. Chemotherapies or liver-directed treatments do not usually result in long-term tumor control. Immunotherapies are currently the most promising therapy option available.
We reviewed both relevant recent literature on PubMed concerning the treatment of uveal melanoma with immunotherapies, and currently investigated drugs on ClinicalTrials.gov. Our own experiences with immune checkpoint blockers are included in a case series of 20 patients.
Because few clinical trials have been conducted for metastasized uveal melanoma, no definitive treatment strategy exists for this rare disease. The outcomes of most immunotherapies are poor, especially compared with cutaneous melanoma. However, encouraging results have been found for some very recently investigated agents such as the bispecific tebentafusp, for which a remarkably increased one-year overall survival rate, and similarly increased disease control rate, were observed in early phase studies.
The treatment of metastatic uveal melanoma remains challenging, and almost all patients still die from the disease. Long-term responses might be achievable by means of new immunological strategies. Patients should therefore be referred to large medical centers where they can take part in controlled clinical studies.
葡萄膜黑色素瘤是成年人中最常见的原发性眼内恶性肿瘤。然而，它是一种罕见疾病，在欧洲每年发病率约为每10万人中有1例。大约一半的肿瘤最终会发生转移，肝脏是通常受累的器官。对于转移性葡萄膜黑色素瘤不存在标准治疗方案。化疗或针对肝脏的治疗通常不能实现长期肿瘤控制。免疫疗法是目前最有前景的治疗选择。
我们检索了PubMed上近期有关免疫疗法治疗葡萄膜黑色素瘤的相关文献，以及ClinicalTrials.gov上目前正在研究的药物。我们自身使用免疫检查点阻滞剂的经验包含在一个20例患者的病例系列中。
由于针对转移性葡萄膜黑色素瘤开展的临床试验很少，对于这种罕见疾病不存在明确的治疗策略。大多数免疫疗法的结果不佳，尤其是与皮肤黑色素瘤相比。然而，对于一些最近研究的药物，如双特异性tebentafusp，已经发现了令人鼓舞的结果，在早期研究中观察到其一年总生存率显著提高，疾病控制率也同样提高。
转移性葡萄膜黑色素瘤的治疗仍然具有挑战性，几乎所有患者最终仍死于该疾病。通过新的免疫策略可能实现长期缓解。因此，患者应被转诊至大型医疗中心，在那里他们可以参加对照临床研究。
