Esma Fabrizio, Salvini Marco, Troia Rossella, Boccadoro Mario, Larocca Alessandra, Pautasso Chiara
a Myeloma Unit, Division of Hematology , Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino , Torino , Italy.
Expert Opin Pharmacother. 2017 Aug;18(11):1127-1136. doi: 10.1080/14656566.2017.1349102. Epub 2017 Jul 10.
Multiple myeloma (MM) is an incurable disease characterized by clonal plasma cell proliferation and overproduction of monoclonal paraprotein, hypercalcemia, renal failure, anemia, osteolytic bone lesions, and infections. Melphalan, a nitrogen mustard, is an alkylating agent synthesized in 1953, and it has been used in multiple myeloma therapy for fifty years. Although novel agents have been introduced in the past few decades improving prognosis of the disease, melphalan still maintains a crucial role in the treatment of MM acting both as cytotoxic agent through damage to DNA, and as immunostimulatory drug by inhibiting Interleukin-6, as well as interaction with dendritic cells, and immunogenic effects in tumor microenvironment. Areas covered: This review focuses on available data about melphalan pharmacology and its role in clinical practice. Expert opinion: Melphalan remains crucial in therapy of multiple myeloma because of its good manageability, safety profile, efficacy, and economic sustainability. These characteristics make it pivotal also for new regimens in combination with novel agents.
多发性骨髓瘤（MM）是一种无法治愈的疾病，其特征为克隆性浆细胞增殖、单克隆副蛋白过度产生、高钙血症、肾衰竭、贫血、溶骨性骨病变和感染。美法仑是一种氮芥，属于烷化剂，于1953年合成，已用于多发性骨髓瘤治疗达五十年之久。尽管在过去几十年中引入了新型药物改善了该疾病的预后，但美法仑在MM治疗中仍发挥着关键作用，它既作为细胞毒性药物通过损伤DNA发挥作用，又作为免疫刺激药物通过抑制白细胞介素-6以及与树突状细胞相互作用，并在肿瘤微环境中产生免疫原性效应。涵盖领域：本综述聚焦于有关美法仑药理学及其在临床实践中作用的现有数据。专家观点：由于美法仑具有良好的可管理性、安全性、疗效和经济可持续性，它在多发性骨髓瘤治疗中仍然至关重要。这些特性使其对于与新型药物联合的新方案也至关重要。
