Response criteria for essential thrombocythemia and polycythemia vera: result of a European LeukemiaNet consensus conference
Open Access
- 14 May 2009
- journal article
- review article
- Published by American Society of Hematology in Blood
- Vol. 113 (20), 4829-4833
- https://doi.org/10.1182/blood-2008-09-176818
Abstract
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.Keywords
This publication has 22 references indexed in Scilit:
- Rapid decline of JAK2V617F levels during hydroxyurea treatment in patients with polycythemia vera and essential thrombocythemiaHaematologica, 2008
- Clinical correlates of JAK2V617F presence or allele burden in myeloproliferative neoplasms: a critical reappraisalLeukemia, 2008
- Myelofibrosis – What’s in a Name?Pathobiology, 2007
- Therapy for Polycythemia Vera and Essential Thrombocythemia Is Driven by the Cardiovascular RiskSeminars in Thrombosis and Hemostasis, 2007
- The haematocrit and platelet target in polycythemia veraBritish Journal of Haematology, 2006
- Hydroxyurea Compared with Anagrelide in High-Risk Essential ThrombocythemiaNew England Journal of Medicine, 2005
- Guidelines for the diagnosis, investigation and management of polycythaemia/erythrocytosisBritish Journal of Haematology, 2005
- The Delphi technique: a methodological discussionJournal of Advanced Nursing, 1994
- The Clinical Efficacy Assessment Project of the American Collegeof PhysiciansInternational Journal of Technology Assessment in Health Care, 1985
- VASCULAR OCCLUSIVE EPISODES AND VENOUS HÆMATOCRIT IN PRIMARY PROLIFERATIVE POLYCYTHÆMLXThe Lancet, 1978