1. Clinical features and survival of patients with indolent systemic mastocytosis defined by the updated WHO classification
- Author
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Jason Gotlib, Patrizia Bonadonna, Nadja Jäkel, Karoline V. Gleixner, Massimo Triggiani, Lucie Nekvindová, Luca Malcovati, Hanneke Oude Elberink, Akif Selim Yavuz, Mattias Mattsson, Roberta Parente, Andreas Reiter, Nikolas von Bubnoff, Karin Hartmann, Olivier Hermine, Roberta Zanotti, Michel Arock, Anja Illerhaus, Knut Brockow, Alexander Zink, Magdalena Lange, Massimiliano Bonifacio, Hanneke C. Kluin-Nelemans, Michael Doubek, Anna Belloni Fortina, Jiri Mayer, Alex Kilbertus, Khalid Shoumariyeh, Aleksandra Górska, Vito Sabato, Jens Panse, David Fuchs, Hans Hägglund, Wolfgang R. Sperr, Peter Valent, Chiara Elena, Bjorn van Anrooij, Marek Niedoszytko, Mohamad Jawhar, Francesca Caroppo, Cecelia Perkins, Jakub Trizuljak, and Agnes Bretterklieber
- Subjects
Male ,0301 basic medicine ,Oncology ,medicine.medical_specialty ,cutaneous mastocytosis ,indolent systemic mastocytosis ,prognostication ,survival ,WHO classification ,Immunology ,Disease ,World Health Organization ,DIAGNOSIS ,Article ,Organomegaly ,03 medical and health sciences ,0302 clinical medicine ,Mastocytosis, Systemic ,Bone Marrow ,Internal medicine ,medicine ,Humans ,Immunology and Allergy ,MAST-CELL DISEASE ,Mast Cells ,TRYPTASE LEVELS ,Systemic mastocytosis ,UTILITY ,Performance status ,business.industry ,Cutaneous Mastocytosis ,MUTATIONS ,Clinical course ,ADULTS ,Prognosis ,medicine.disease ,3. Good health ,030104 developmental biology ,medicine.anatomical_structure ,030228 respiratory system ,PATTERNS ,Bone marrow ,Human medicine ,medicine.symptom ,Who classification ,business ,BURDEN ,Mastocytosis - Abstract
Background: In indolent systemic mastocytosis (ISM), several risk factors of disease progression have been identified. Previous studies, performed with limited patient numbers, have also shown that the clinical course in ISM is stable and comparable to that of cutaneous mastocytosis (CM). The aim of this project was to compare the prognosis of patients with ISM with that of patients with CM.Methods: We employed a dataset of 1993 patients from the registry of the European Competence Network on Mastocytosis (ECNM) to compare outcomes of ISM and CM.Results: We found that overall survival (OS) is worse in ISM compared to CM. Moreover, in patients with typical ISM, bone marrow mastocytosis (BMM), and smoldering SM (SSM), 4.1% of disease progressions have been observed (4.9% of progressions in typical ISM group, 1.7% in BMM, and 9.4% in SSM). Progressions to advanced SM were observed in 2.9% of these patients. In contrast, six patients with CM (1.7%) converted to ISM and no definitive progression to advanced SM was found. No significant differences in OS and event-free survival (EFS) were found when comparing ISM, BMM, and SSM. Higher risk of both progression and death was significantly associated with male gender, worse performance status, and organomegaly.Conclusion: Our data confirm the clinical impact of the WHO classification that separates ISM from CM and from other SM variants.
- Published
- 2020