Pharmacy Department, Dana-Farber Cancer Institute, Boston, MA.
Clin Lymphoma Myeloma Leuk. 2013 Sep;13 Suppl 2(0 2):S310-6. doi: 10.1016/j.clml.2013.05.023.
Waldenström macroglobulinemia (WM), first described by Jan Waldenström in 1944, is a lymphoplasmacytic lymphoma characterized by the presence of an immunoglobulin M monoclonal gammopathy in the blood and monoclonal small lymphocytes and lymphoplasmacytoid cells in the bone marrow. WM is a rare and indolent disease but remains incurable. In this review we discuss the pathogenesis of WM and focus on novel treatment options that target pathways deregulated in this disease. Recent studies have helped us identify specific genetic mutations that are commonly seen in WM and might prove to be important therapeutic targets in the future. We discuss the role of epigenetics and the changes in the bone marrow microenvironment that are important in the pathogenesis of WM. The commonly used drugs are discussed with a focus on novel agents that are currently being used as single agents or in combination to treat WM. We finally focus on some agents that have shown preclinical efficacy and might be available in the near future.
瓦尔登斯特伦巨球蛋白血症（WM），于 1944 年由 Jan Waldenström 首次描述，是一种淋巴浆细胞淋巴瘤，其特征是血液中存在免疫球蛋白 M 单克隆丙种球蛋白病，骨髓中存在单克隆小淋巴细胞和淋巴浆细胞样细胞。WM 是一种罕见的惰性疾病，但仍无法治愈。在这篇综述中，我们讨论了 WM 的发病机制，并重点介绍了针对该疾病中失调途径的新型治疗选择。最近的研究帮助我们确定了 WM 中常见的特定基因突变，这些突变可能在未来成为重要的治疗靶点。我们讨论了表观遗传学的作用以及在 WM 发病机制中重要的骨髓微环境变化。我们讨论了常用药物，并重点介绍了目前作为单一药物或联合使用治疗 WM 的新型药物。最后，我们关注了一些在临床前研究中显示出疗效的药物，这些药物可能在不久的将来上市。
