There are many different treatment options available for WM patients, and they are increasing as researchers discover more about the biology and genetics of the disease.
Treatment may consist of just one drug (single-agent therapy) or of two or more drugs (combination therapy). Most studies seem to indicate that combination therapies are more effective, resulting in better and/or longer-lasting responses.
Many treatment options are available to WM patients, and a full discussion of each is beyond the scope of this introduction. More information can be obtained by linking to the list of presentations and publications below or by using our online Glossary of Terms. Currently available treatment options may include one or more of the following:
supportive therapy such as transfusions or growth factors to boost red blood cells, white blood cells, and platelets;
surgical or other procedures, including splenectomy (surgical removal of the spleen), plasmapheresis to remove IgM, targeted radiation to reduce the size of lymph nodes, and stem cell transplantation;
chemotherapy with alkylating agents such as chlorambucil, cyclophosphamide, and bendamustine or with nucleoside analogs such as fludarabine and cladribine;
corticosteroids, including prednisone and dexamethasone;
biologic therapy with monoclonal antibodies such as rituximab and ofatumumab;
immunomodulatory drugs, including thalidomide and lenalidomide;
proteasome inhibitors such as bortezomib and carfilzomib;
targeted therapies to the B-cell signaling pathways, including ibrutinib and everolimus.
Treatment can usually be administered in an outpatient setting or at home and may be oral, by intramuscular or subcutaneous injection, or by intravenous therapy. Some treatments require that certain medications be taken the day before or the day of treatment in order to minimize associated side effects.
Additionally, in some cases, participation in a clinical trial can actually be the best or even the only option available - and should be seriously considered by the patient. More information regarding clinical trials and their significance to WM patients can be found here.
Laboratory tests are performed during treatment to check response to therapy and/or to monitor side effects related to it. Typical laboratory testing includes the Complete Blood Count (CBC), the Complete Metabolic Panel (CMP), and the IgM measurement by nephelometry and/or serum protein electrophoresis (SPEP). Special circumstances or conditions might require additional tests.
Treatment cycles may take several weeks to months, depending on the course of therapy chosen. It is not unusual to have a round of therapy and then wait a week or a month before another round of treatment, although some of the newer oral therapies require daily dosing instead. Hematologist-oncologists follow established protocols for treatment but may make adjustments depending on side effects or response to therapy.
The following list of presentations and publications discuss many of the treatment options available to WM patients.
- A review of recent advances in the biology of WM and the current therapeutic options for untreated and relapsed WM patients, including a discussion of prognostic factors. Jorge J. Castillo, Irene M. Ghobrial, and Steven P. Treon from Dana-Farber Cancer Institute. "Biology, Prognosis, and Therapy of Waldenström Macroglobulinemia." Cancer Treatment and Research, 2015. (Provided with permission from Dr. Castillo).
- A review of what is known in 2013/2014 about WM, its biology and treatment, with a summary of novel agents that are becoming available. Ilyas Sahin, Houry Leblebjian, Steven P. Treon, and Irene M. Ghobrial, from Dana-Farber Cancer Institute. "Waldenström macroglobulinemia: From Biology to Treatment." (Article provided with permission of Dr. Ghobrial.) Published in Expert Rev. Hematol. 2014 Jan 3.
- A downloadable (but not printable) review of recent results (as of September, 2010) reported for the use of new combinations and new drugs in patients with WM at different stages of the disease. Garcia-Sanz, Ramón and Ocio, Enrique M. "Novel treatment regimens for Waldenström's macroglobulinemia." Expert Rev. Hematol. 3(3), 339-350 (2010). (Article provided with permission of Expert Reviews.)
- A variety of articles on the biology and potential side effects of rituximab (Rituxan) that were published in the journal Seminars in Hematology, Volume 47, Issue 2, April 2010. (Articles provided with permission of Elsevier/Rightslink Copyright Clearance Center.)
- A guide to the details behind autologous stem cell collection, storage, and transplantation – including the step-by-step process, with pictures and diagrams. This document was developed by the European Group for Blood and Marrow Transplantation as a guide for Nurses and Other Allied Health Care Professionals. NOTE: Stem cell collection, storage, and transplantation procedures will vary from country to country, and will change over time. This document is presented as a guide regarding what one may expect in a given situation. Please be sure to consult with your health care team as to the appropriate procedure that will fit your particular situation.
- Treatment Options, by Guy Sherwood, MD. This patient-friendly publication of the IWMF discusses the various treatment options available to WM patients.