International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) & European Competence Network on Mastocytosis (ECNM) consensus response criteria in advanced systemic mastocytosis
Year: 2013
Type: article
Abstract: Systemic mastocytosis (SM) is characterized by accumulation of neoplastic mast cells and is classified into indolent and aggressive forms. The latter include aggressive SM (ASM), mast cell leukemia (MCL), and SM associated with a myeloid neoplasm wherein 1 or both disease compartments exhibit advanced features. These variants, henceforth collectively referred to as advanced SM for the purposes of this report, are typically characterized by organ damage and shortened survival duration. In contrast to indolent SM, in which symptoms are usually managed by noncytotoxic antimediator therapy, cytoreduction is usually necessary for disease control in advanced SM. Unfortunately, current drug treatment of these patients rarely results in complete clinical and histopathologic remissions or improved survival time. Previously defined response criteria were adapted to the heterogeneous presentations of advanced SM and the limited effects of available drugs. However, recent advances in understanding the molecular pathogenesis of SM and the corresponding prospect in targeted therapy make it a priority to modify these criteria. Our current study is the product of an international group of experts and summarizes the challenges in accomplishing this task and forwards a new proposal for response criteria, which builds on prior proposals and should facilitate response evaluation in clinical trials.
Source: Blood
Authors: Jason Gotlib, Animesh Pardanani, Cem Akin, Andreas Reiter, Tracy George, Olivier Hermine, Hanneke Kluin-Nelemans, Karin Hartmann, Wolfgang R. Sperr, Knut Brockow, Lawrence B. Schwartz, Alberto Orfao, Daniel J. DeAngelo, Michel Arock, Karl Sotlar, Hans-Peter Horny, Dean D. Metcalfe, Luis Escribano, Srdan Verstovsek, Ayalew Tefferi, Peter Valent
Institutions: Stanford Medicine, Stanford University, Mayo Clinic, Brigham and Women's Hospital, Harvard University, University Medical Centre Mannheim, Hôpital Necker-Enfants Malades, Centre National de la Recherche Scientifique, Université Paris Descartes, University Medical Center Groningen, University of Cologne, Medical University of Vienna, Virginia Commonwealth University, Universidad de Salamanca, Dana-Farber Cancer Institute, École Normale Supérieure Paris-Saclay, Pitié-Salpêtrière Hospital, Laboratoire de Biologie et Pharmacologie Appliquée, Ludwig-Maximilians-Universität München, National Institutes of Health, National Institute of Allergy and Infectious Diseases, The University of Texas MD Anderson Cancer Center
Language: English
Topic: Mast cells and histamine
Subfield: Immunology
Field: Immunology and Microbiology
Domain: Life Sciences
Open Access status: green
Funder: National Institutes of Health
OpenAlex ID: W1964608118