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Blood, 1 September 2006, Vol. 108, No. 5, pp. 1497-1503.
Prepublished online as a Blood First Edition Paper on May 4, 2006; DOI 10.1182/blood-2006-03-009746.


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Submitted March 13, 2006
Accepted April 23, 2006

International Working Group (IWG) consensus criteria for treatment response in myelofibrosis with myeloid metaplasia: On behalf of the IWG for myelofibrosis research and treatment (IWG-MRT)

Ayalew Tefferi*, Giovanni Barosi, Ruben A Mesa, Francisco Cervantes, H J Deeg, John T Reilly, Srdan Verstovsek, Brigitte Dupriez, Richard T Silver, Olatoyosi Odenike, Jorge Cortes, Martha Wadleigh, Lawrence A Solberg Jr., John K Camoriano, Heinz Gisslinger, Pierre Noel, Juergen Thiele, James W Vardiman, Ronald Hoffman, Nicholas C Cross, D G Gilliland, and Hagop Kantarjian

Mayo Clinic, Rochester, Minnesota, USA
Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy
Hospital Clinic, Institut d'Investigacions Biomediques August Pi i Sunyer, Barcelona, Spain
Fred Hutchinson Cancer Research Center, Seattle, Washington, USA
Royal Hallamshire Hospital, Sheffield, United Kingdom
MD Anderson Cancer Center, Houston, Texas, USA
Service d'Hematologie Clinique, Centre Hospitalier de Lens, France
Cornell Medical Center, New York, New York, USA
University of Chicago, Chicago, Illinois
Dana Farber Cancer Institute, Boston, Massachusetts, USA
Mayo Clinic, Jacksonville, Florida, USA
Mayo Clinic, Scottsdale, Arizona, USA
Department of Hematology and Blood Coagulation, Medical University of Vienna, Vienna, Austria
National Institutes of Health, Bethesda, Maryland, USA
Institute of Pathology, University of Cologne, Germany
University of Illinois, Cancer Care Center
Wessex Regional Genetics Laboratory, Salisbury, United Kingdom

* Corresponding author; email: tefferi.ayalew{at}mayo.edu.

Myelofibrosis with myeloid metaplasia (MMM) is a clinicopathologic entity characterized by stem cell-derived clonal myeloproliferation, ineffective erythropoiesis, extramedullary hematopoiesis, and bone marrow fibrosis and osteosclerosis. Patients with MMM have shortened survival and their quality of life is compromised by progressive anemia, marked hepatosplenomegaly, and severe constitutional symptoms including cachexia. After decades of frustration with ineffective therapy, patients are now being served by promising treatment approaches that include allogeneic hematopoietic stem cell transplantation and immunomodulatory drugs. Recent information regarding disease pathogenesis, including a contribution to the myeloproliferative disorder phenotype by a gain-of-function JAK2 mutation (JAK2V617F), has revived the prospect of targeted therapeutics as well as molecular monitoring of treatment response. Such progress calls for standardization of response criteria to accurately assess the value of new treatment modalities, to allow accurate comparison between studies, and to assure that the definition of " response" reflects meaningful health outcome. Accordingly, an international panel of experts recently convened and delineated three response categories; complete (CR) and partial (PR) remissions and " clinical improvement (CI)" . Bone marrow histological and hematological remissions characterize CR and CR/PR, respectively. The panel agreed that the CI response category is applicable only to patients with moderate to severe cytopenia or splenomegaly.


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