Waldenström macroglobulinemia: is newer really better?

Clinical Lymphoma Myeloma and Leukemia

Agarwal

Ghobrial

2013

Waldenström’s macroglobulinemia first described by Jan Waldenström in 1944 is a lymphoplasmacytic lymphoma characterized by the presence of an immunoglobulin M (IgM) 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 Waldenström macroglobulinemia 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 may 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 combinations to treat WM. We finally focus on some agents that have shown preclinical efficacy and may be available in the near future.

“…chlorambucil, and cyclophosphamide), nucleoseide analogue (fludarabine and cladribine), the monoclonal antibody rituximab, and the proteasome inhibitor bortezomib [49-51]. …”

Section: Primary Treatment Options For Wmmentioning

confidence: 99%

Novel Treatment Options for Waldenström Macroglobulinemia

Clinical Lymphoma Myeloma and Leukemia

Agarwal

Ghobrial

2013

New insights into the pathogenesis and treatment of Waldenstrom macroglobulinemia

Issa

Current Opinion in Hematology

Roccaro

et al. 2011

Waldenström macroglobulinemia: from biology to treatment

Şahin

Expert Review of Hematology

Treon

et al. 2014

Waldenström macroglobulinemia (WM) is distinct B-cell lymphoproliferative disorder primarily characterized by bone marrow infiltration of lymphoplasmacytic cells along with production of a serum monoclonal (IgM). In this review, we describe the biology of WM, the diagnostic evaluation for WM with a discussion of other conditions that are in the differential diagnosis and clinical manifestations of the disease as well as current treatment options. Within the novel agents discussed are everolimus, perifosine, enzastaurin, panobinostat, bortezomib and carfilzomib, pomalidomide and ibrutinib. Many of the novel agents have shown good responses and have a better toxicity profile compared to traditional chemotherapeutic agents, which makes them good candidates to be used as primary therapies for WM in the future.