International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) & European Competence Network on Mastocytosis (ECNM) consensus response criteria in advanced systemic mastocytosis

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 TefferiPeter Valent

Research output: Contribution to journalReview article

62 Scopus citations

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 internationalgroupofexpertsandsummarizes the challenges in accomplishing this task and forwards a new proposal for response criteria,whichbuildsonprior proposals and should facilitate response evaluation in clinical trials.

Original languageEnglish (US)
Pages (from-to)2393-2401
Number of pages9
JournalBlood
Volume121
Issue number13
DOIs
StatePublished - Jan 1 2013

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ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

Cite this

Gotlib, J., Pardanani, A., Akin, C., Reiter, A., George, T., Hermine, O., Kluin-Nelemans, H., Hartmann, K., Sperr, W. R., Brockow, K., Schwartz, L. B., Orfao, A., DeAngelo, D. J., Arock, M., Sotlar, K., Horny, H. P., Metcalfe, D. D., Escribano, L., Verstovsek, S., ... Valent, P. (2013). International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) & European Competence Network on Mastocytosis (ECNM) consensus response criteria in advanced systemic mastocytosis. Blood, 121(13), 2393-2401. https://doi.org/10.1182/blood-2012-09-458521