Updates in prognostication and treatment of Waldenström's macroglobulinemia

Waldenström's macroglobulinemia (WM) is a rare, incurable hematologic disorder with a relatively indolent course in a majority of the patients. Despite this, a significant proportion of patients require treatment because of hypersecretion of immunoglobulin M and the invasion of bone marrow and peripheral organs by neoplastic lymphoplasmacytic lymphoma cells. Historically, there has been a dearth of research and therapeutic advancements in the field of WM, with most understanding based on other, related B-cell lymphoid malignancies, including multiple myeloma, chronic lymphocytic leukemia, and non-Hodgkin lymphoma. Recently, there has been an increase in dedicated work to better explain the pathobiology of WM, which has identified several clinical and genetic markers that serve to prognosticate disease course and patient outcomes. Furthermore, this has led to dedicated clinical trials and the development of novel drugs/regimens including the first Food and Drug Administration-approved agent for this diagnosis, ibrutinib. This review aims to document some of the recent advancements with respect to prognostic markers and therapeutic options for patients with WM, as well as certain selected novel treatments with unique mechanisms of action, that are currently under development.