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Blood, 30 April 2009, Vol. 113, No. 18, pp. 4163-4170. Prepublished online as a Blood First Edition Paper on February 5, 2009; DOI 10.1182/blood-2008-08-174961.
CLINICAL TRIALS AND OBSERVATIONS International prognostic scoring system for Waldenström macroglobulinemia1 Hôpital Schaffner, Lens, France; 2 French Cooperative Group Chronic Lymphocytic Leukaemia/Waldenstrom Macroglobulinemia, Paris, France; 3 Faculte de Medecine, Lille, France; 4 Fondazione IRCCS Policlinico San Matteo, University of Pavia, Italy; 5 University of Athens School of Medicine, Athens, Greece; 6 Rockefeller University, New York, NY; 7 Southwest Oncology Group, Seattle, WA; 8 Spanish Group for the Study of Waldenstrom Macroglobulinaemia, Salamanca, Spain; 9 Dana-Farber Cancer Institute, Boston, MA; 10 Hôpital Pitié Salpêtrière Université Pierre et Marie Curie, Paris, France; 11 Mayo Clinic, Rochester, MN; and 12 University of Arkansas for Medical Sciences, Little Rock Recently, many new drugs have been developed for the treatment of Waldenström macroglobulinemia (WM). To optimize the treatment according to the prognosis and to facilitate the comparison of trials, we developed an International Prognostic Scoring System for WM in a series of 587 patients with clearly defined criteria for diagnosis and for initiation of treatment. The median survival after treatment initiation was 87 months. Five adverse covariates were identified: advanced age (>65 years), hemoglobin less than or equal to 11.5 g/dL, platelet count less than or equal to 100 x 109/L, β2-microglobulin more than 3 mg/L, and serum monoclonal protein concentration more than 7.0 g/dL. Low-risk patients (27%) presented with no or 1 of the adverse characteristics and advanced age, intermediate-risk patients (38%) with 2 adverse characteristics or only advanced age, and high-risk patients (35%) with more than 2 adverse characteristics. Five-year survival rates were 87%, 68%, and 36%, respectively (P < .001). The ISSWM retained its prognostic significance in subgroups defined by age, treatment with alkylating agent, and purine analog. Thus, the ISSWM may provide a means to design risk-adapted studies. However, independent validation and new biologic markers may enhance its significance.
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