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Blood, 15 October 2005, Vol. 106, No. 8, pp. 2849-2853.
Prepublished online as a Blood First Edition Paper on June 30, 2005; DOI 10.1182/blood-2005-04-1520.
Previous Article | Next Article 
Submitted April 14, 2005
Accepted June 12, 2005
Response criteria for myelofibrosis with myeloid metaplasia: results of an initiative of the European myelofibrosis network (EUMNET)
Giovanni Barosi*, Dominique Bordessoule, Jean Briere, Francisco Cervantes, Jean-Loup Demory, Brigitte Dupriez, Heinz Gisslinger, Martin Griesshammer, Hans Hasselbalch, Rajko Kusec, Marie-Caroline Le Bousse-Kerdiles, Nicola L Liberato, Monia Marchetti, John T Reilly, and Jurgen Thiele
Laboratory of Clinical Epidemiology, IRCCS Policlinico S. Matteo, Pavia, Italy
Service d'Hematologie Clinique, CHRU Limoges, Limoges, France
Department of Hematology, Hopital Beaujon, Clichy, France
Hematology Department, Hospital Clinic, IDIBAPS, Barcelona, Spain
Department d'Hematologie, Universite Catholique de Lille, Lille, France
Service d'Hematologie Clinique, Centre Hospitalier de Lens, Lens, France
Department of Hematology and Blood Coagulation, University of Vienna, Vienna, Austria
Department of Medicine III, Hematology, Oncology, Rheumatology and Infectious Diseases, University of Ulm, Ulm, Germany
Department of Medicine, Division of Hematology and Oncology, Roskilde Hospital, Roskilde, Denmark
Institute of Clinical Chemistry and Department of Medicine, University Hospital Merkur, Zagreb, Croatia (Hrvatska)
INSERM U268, Institut Andre Lwoff, Hopital Paul Brousse, Villejuif, France
Molecular Hematology Unit, Division of Molecular and Genetic Medicine, Royal Hallamshire Hospital, Sheffield, United Kingdom
Institute of Pathology, University of Cologne, Cologne, Germany
* Corresponding author; email: barosig{at}smatteo.pv.it.
EUMNET, an European research network on myelofibrosis with myeloid metaplasia (MMM), has developed a definition of response for the disease by using clinico-hematological, histological and cytogenetic criteria. A core set of five clinico-hematological criteria was selected out of 9 candidates on the basis of their sensitivity to change measured in 196 patients treated either during clinical trials or routine clinical practice. A Consensus Panel of 16 international experts was convened, and asked to score the level of response of 104 patient profiles as major, moderate, minor or no response according to changes of the clinico-hematological criteria. Using the experts consensus as the gold standard, the performance of 100 possible definitions of response was evaluated. Criteria for major or moderate clinico-hematological response was determined to be changes in Hb and spleen size and the presence of constitutional symptoms, while changes in platelet count and WBC count served as complementary criteria and were of value for defining minor responses. A histological response was defined by changes in bone marrow fibrosis and cellularity grades. The combined use of these response definitions should help standardize the design and reporting of future clinical studies in MMM.

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