Response criteria for essential thrombocythemia and polycythemia vera: result of a European LeukemiaNet consensus conference

Blood - Tập 113 Số 20 - Trang 4829-4833 - 2009
Giovanni Barosi1, Gunnar Birgegård2, Guido Finazzi3, Martin Grießhammer4, Claire Harrison5, Hans Carl Hasselbalch6, Jean‐Jacques Kiladjian7, Eva Lengfelder8, Mary Frances McMullin9, Francesco Passamonti10, John T. Reilly11, Alessandro M. Vannucchi12, Tiziano Barbui13
1Unit of Clinical Epidemiology and Center for the Study of Myelofibrosis, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico S. Matteo, Pavia, Italy;
2Department of Haematology, University Hospital, Uppsala, Sweden
3Division of Transfusion Medicine, Ospedali Riuniti di Bergamo, Bergamo, Italy;
4Department of Hematology and Oncology, Johannes Wesling Medical Centre Minden, Academic Hospital of the University of Hannover, Minden, Germany
5Department of Haematology, Guy’s and St Thomas’ National Health Service Foundation Trust, London, United Kingdom
6Department of Hematology L, Herlev Hospital, University of Copenhagen, Copenhagen, Denmark
7Assistance Publique-Hopitaux de Paris, Hopital Avicenne, Service d'Hematologie Clinique, Université Paris 13, Bobigny, France;
8Department of Medicine III, Faculty for Medicine Mannheim, University of Heidelberg, Heidelberg, Germany;
9Centre for Cancer Research and Cell Biology, Queen's University, Belfast, United Kingdom
10Division of Hematology, University of Pavia, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy;
11Molecular Haematology Unit, Division of Molecular and Genetic Medicine, Royal Hallamshire Hospital, Sheffield, United Kingdom;
12Unità Funzionale di Ematologia, Dipartimento di Area Critica Medico Chirurgica, Università degli Studi, Firenze, Italy; and
13Division of Hematology, Ospedali Riuniti di Bergamo, Bergamo, Italy

Tóm tắt

European experts were convened to develop a definition of response to treatment in polycythemia vera (PV) and essential thrombocythemia (ET). Clinicohematologic (CH), molecular, and histologic response categories were selected. In ET, CH complete response (CR) was: platelet count less than or equal to 400 × 109/L, no disease-related symptoms, normal spleen size, and white blood cell count less than or equal to 10 × 109/L. Platelet count less than or equal to 600 × 109/L or a decrease greater than 50% was partial response (PR). In PV, CH-CR was: hematocrit less than 45% without phlebotomy, platelet count less than or equal to 400 × 109/L, white blood cell count less than or equal to 10 × 109/L, and no disease-related symptoms. A hematocrit less than 45% without phlebotomy or response in 3 or more of the other criteria was defined as PR. In both ET and in PV, molecular CR was a reduction of any molecular abnormality to undetectable levels. Molecular PR was defined as a reduction more than or equal to 50% in patients with less than 50% mutant allele burden, or a reduction more than or equal to 25% in patients with more than 50% mutant allele burden. Bone marrow histologic response in ET was judged on megakaryocyte hyperplasia while on cellularity and reticulin fibrosis in PV. The combined use of these response definitions should help standardize the design and reporting of clinical studies.

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