Novel agents in Waldenstrom Macroglobulinemia.

Waldenstrom macroglobulinemia (WM) is a B-cell disorder characterized by the infiltration of the bone marrow with lymphoplasmacytic cells and the detection of an IgM monoclonal gammopathy in the serum. WM is considered an incurable disease, with a median overall survival of 87 months. The success of targeted therapy in multiple myeloma has led to the development and investigation of more than 30 new compounds in this disease and in other plasma cell dyscrasias, including WM, both in the preclinical settings and as part of clinical trials. Among therapeutic options, first-line therapies have been based on single-agent or combination regimens with alkylator agents, nucleoside analogues and the monoclonal antibody anti-CD20. Based on the understanding of the complex interaction between WM tumor cells and the bone marrow microenvironment, and the signaling pathways that are deregulated in WM pathogenesis, a number of novel therapeutic agents are now available and have demonstrated significant efficacy in WM. ...