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 Blood, 26 March 2009, Vol. 113, No. 13, pp. 2895-2901.
Prepublished online as a Blood First Edition Paper on November 6, 2008; DOI 10.1182/blood-2008-07-170449.

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CLINICAL TRIALS AND OBSERVATIONS

New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment

Francisco Cervantes1, Brigitte Dupriez2, Arturo Pereira1, Francesco Passamonti3, John T. Reilly4, Enrica Morra5, Alessandro M. Vannucchi6, Ruben A. Mesa7, Jean-Loup Demory2, Giovanni Barosi8, Elisa Rumi3, and Ayalew Tefferi7

1 Hematology Department, Hospital Clínic, Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), University of Barcelona, Barcelona, Spain; 2 Centre Hospitalier, Lens and Lille, France; 3 Hematology Division, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico S Matteo, University of Pavia, Pavia, Italy; 4 Royal Hallamshire Hospital, Sheffield, United Kingdom; 5 Universita Milano-Niguarda, Milan, Italy; 6 University of Florence, Florence, Italy; 7 Division of Hematology, Mayo Clinic College of Medicine, Rochester, MN; and 8 Unit of Clinical Epidemiology, Center for the Study of Myelofibrosis, Fondazione IRCCS Policlinico S Matteo, Pavia, Italy

Therapeutic decision-making in primary myelofibrosis (PMF) is becoming more challenging because of the increasing use of allogeneic stem cell transplantation and new investigational drugs. To enhance this process by developing a highly discriminative prognostic system, 1054 patients consecutively diagnosed with PMF at 7 centers were studied. Overall median survival was 69 months (95% confidence interval [CI]: 61-76). Multivariate analysis of parameters obtained at disease diagnosis identified age greater than 65 years, presence of constitutional symptoms, hemoglobin level less than 10 g/dL, leukocyte count greater than 25 x 109/L, and circulating blast cells 1% or greater as predictors of shortened survival. Based on the presence of 0 (low risk), 1 (intermediate risk-1), 2 (intermediate risk-2) or greater than or equal to 3 (high risk) of these variables, 4 risk groups with no overlapping in their survival curves were delineated; respective median survivals were 135, 95, 48, and 27 months (P < .001). Compared with prior prognostic models, the new risk stratification system displayed higher predictive accuracy, replicability, and discriminating power. In 409 patients with assessable metaphases, cytogenetic abnormalities were associated with shorter survival, but their independent contribution to prognosis was restricted to patients in the intermediate-risk groups. JAK2V617F did not cluster with a specific risk group or affect survival.


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