The term "macroglobulinemia" refers to the production of excess IgM monoclonal protein that occurs in certain clonal lymphoproliferative disorders and plasma cell dyscrasias. This broad definition includes patients with monoclonal gammopathy of undetermined significance of the IgM type (IgM MGUS), smoldering Waldenström macroglobulinemia (SWM), Waldenström macroglobulinemia (WM), and a number of related disorders in which an IgM monoclonal protein is detected, such as chronic lymphocytic leukemia (CLL) and a number of lymphoma variants, and primary amyloidosis.
WM is a distinct clinicopathologic entity demonstrating lymphoplasmacytic lymphoma (LPL) in the bone marrow with an IgM monoclonal gammopathy in the blood. Patients may present with symptoms related to the infiltration of the hematopoietic tissues or the effects of monoclonal IgM in the blood.
There is no standard therapy for the treatment of symptomatic WM, and no agents have been specifically approved by the US Food and Drug Administration (FDA) or European Medicines Agency (EMA) for this disease. Whenever possible, patients should be encouraged to participate in clinical trials. This topic review presents suggestions for the treatment of patients who are not eligible for clinical trials or who choose not to participate in clinical trials.
The treatment and prognosis of WM will be reviewed here. The clinical manifestations and diagnosis of this disorder are discussed separately. (See "Epidemiology, pathogenesis, clinical manifestations and diagnosis of Waldenström macroglobulinemia" and "Clinical manifestations, pathologic features, and diagnosis of lymphoplasmacytic lymphoma".)
The pretreatment evaluation both determines the extent of the disease and provides information about the individual's comorbidities that are likely to have an impact on treatment options. In addition to a history and physical examination, it is our practice to perform the following pretreatment studies in patients with Waldenström macroglobulinemia (WM):