title: International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) & European Competence Network on Mastocytosis (ECNM) consensus response criteria in advanced systemic mastocytosis.
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.
authors: Stanford University School of Medicine, Stanford, CA;
authors: Jason
authors: J
authors: Gotlib
authors: Animesh
authors: A
authors: Pardanani
authors: Cem
authors: C
authors: Akin
authors: Andreas
authors: A
authors: Reiter
authors: Tracy
authors: T
authors: George
authors: Olivier
authors: O
authors: Hermine
authors: Hanneke
authors: H
authors: Kluin-Nelemans
authors: Karin
authors: K
authors: Hartmann
authors: Wolfgang R
authors: WR
authors: Sperr
authors: Knut
authors: K
authors: Brockow
authors: Lawrence B
authors: LB
authors: Schwartz
authors: Alberto
authors: A
authors: Orfao
authors: Daniel J
authors: DJ
authors: Deangelo
authors: Michel
authors: M
authors: Arock
authors: Karl
authors: K
authors: Sotlar
authors: Hans-Peter
authors: HP
authors: Horny
authors: Dean D
authors: DD
authors: Metcalfe
authors: Luis
authors: L
authors: Escribano
authors: Srdan
authors: S
authors: Verstovsek
authors: Ayalew
authors: A
authors: Tefferi
authors: Peter
authors: P
authors: Valent
mesh_terms: D002983:Clinical Competence / Q000331:legislation & jurisprudence / Q000592:standards; D002986:Clinical Trials as Topic; D019058:Community Networks / Q000331:legislation & jurisprudence / Q000458:organization & administration; D032921:Consensus; D018450:Disease Progression; D005060:Europe; D006402:Hematologic Diseases / Q000175:diagnosis / Q000209:etiology / Q000628:therapy; D006801:Humans; D007391:International Cooperation; D008415:Mastocytosis / Q000150:complications / Q000175:diagnosis / Q000628:therapy*; D034721:Mastocytosis, Systemic / Q000150:complications / Q000175:diagnosis / Q000628:therapy*; D063305:Organ Dysfunction Scores; D012955:Societies, Medical / Q000331:legislation & jurisprudence / Q000458:organization & administration