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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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CLINICAL TRIALS AND OBSERVATIONS
International Working Group (IWG) consensus criteria for treatment response in myelofibrosis with myeloid metaplasia, for the IWG for Myelofibrosis Research and Treatment (IWG-MRT)
Ayalew Tefferi,
Giovanni Barosi,
Ruben A. Mesa,
Francisco Cervantes,
H. Joachim 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. P. Cross,
D. Gary Gilliland, and
Hagop Kantarjian
From the Mayo Clinic, Rochester, MN; Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy; Hospital Clinic, Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain; the Fred Hutchinson Cancer Research Center, Seattle, WA; the Royal Hallamshire Hospital, Sheffield, United Kingdom; the M. D. Anderson Cancer Center, Houston, TX; Service d'Hématologie Clinique, Centre Hospitalier de Lens, France; Cornell Medical Center, New York, NY; the University of Chicago, IL; the Dana Farber Cancer Institute, Boston, MA; the Mayo Clinic, Jacksonville, FL; the Mayo Clinic, Scottsdale, AZ; the Department of Hematology and Blood Coagulation, Medical University of Vienna, Austria; the National Institutes of Health, Bethesda, MD; the Institute of Pathology, University of Cologne, Germany; the University of Illinois, Chicago and the Wessex Regional Genetics Laboratory, Salisbury, United Kingdom.
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 ensure that the definition of response reflects meaningful health outcome. Accordingly, an international panel of experts recently convened and delineated 3 response categories: complete remission (CR), partial remission (PR), and clinical improvement (CI). Bone marrow histologic and hematologic 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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