Refined diagnostic criteria for bone marrow mastocytosis: a proposal of the European competence network on mastocytosis.

Abstract

In the current classification of the World Health Organization (WHO), bone marrow mastocytosis (BMM) is a provisional variant of indolent systemic mastocytosis (ISM) defined by bone marrow involvement and absence of skin lesions. However, no additional diagnostic criteria for BMM have been proposed. Within the registry dataset of the European Competence Network on Mastocytosis, we compared characteristics and outcomes of 390 patients with BMM and 1175 patients with typical ISM. BMM patients were significantly older, predominantly male, had lower tryptase and lower burden of neoplastic mast cells, and displayed a higher frequency of allergic reactions, mainly triggered by Hymenoptera, than patients with typical ISM. The estimated 10-year progression-free survival of BMM and typical ISM was 95.9% and 92.6%, respectively. In BMM patients defined by WHO-based criteria, the presence of one B-Finding and tryptase level ≥125 ng/mL were identified as risk factors for progression in multivariate analyses. BMM patients without any of these risk factors were found to have better progression-free survival (p < 0.05) and better overall survival (p < 0.05) than other ISM patients. These data support the proposal to define BMM as a separate SM variant characterized by SM criteria, absence of skin lesions, absence of B-Findings, and tryptase levels <125 ng/mL.

• Authors: Roberta Zanotti, Massimiliano Bonifacio, Giuseppe Lucchini, Wolfgang R Sperr, Luigi Scaffidi, Björn van Anrooij, Hanneke Nc Oude Elberink, Julien Rossignol, Olivier Hermine, Aleksandra Gorska, Magdalena Lange, Emir Hadzijusufovic, Cornelius Miething, Sabine Müller, Cecelia Perkins, William Shomali, Chiara Elena, Anja Illerhaus, Mohamad Jawhar, Roberta Parente, Francesca Caroppo, Oleksii Solomianyi, Alexander Zink, Mattias Mattsson, Akif Selim Yavuz, Jens Panse, Judit Varkonyi, Michael Doubek, Vito Sabato, Christine Breynaert, Vladan Vucinic, Tanja Schug, Hans Hägglund, Friederike Wortmann, Knut Brockow, Irena Angelova-Fischer, Anna Belloni Fortina, Massimo Triggiani, Andreas Reiter, Karin Hartmann, Luca Malcovati, Jason Gotlib, Khalid Shoumariyeh, Marek Niedoszytko, Michel Arock, Hanneke C Kluin-Nelemans, Patrizia Bonadonna, Peter Valent
• Journal: Leukemia (Leukemia) Vol. 36 Issue 2 Pg. 516-524 (02 2022) ISSN: 1476-5551 [Electronic] England
• PMID: 34545185 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2021. The Author(s), under exclusive licence to Springer Nature Limited.

Chemical References

• Tryptases

Topics

• Adult
• Aged
• Aged, 80 and over
• Bone Marrow (metabolism, pathology)
• Europe (epidemiology)
• Female
• Follow-Up Studies
• Humans
• Male
• Mast Cells (metabolism, pathology)
• Mastocytosis (diagnosis, epidemiology, metabolism)
• Mastocytosis, Systemic (diagnosis, epidemiology, metabolism)
• Middle Aged
• Prognosis
• Skin Diseases (physiopathology)
• Survival Rate
• Tryptases (metabolism)