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.

Allergy Unit Verona University Hospital Verona Italy

Departement of Hematology Centre National de Référence des Mastocytoses Imagine Institute INSERM U1123 Université Paris Descartes Sorbonne Paris Cité Hôpital Necker Assistance Publique des Hôpitaux de Paris Paris France

Department of Allergology Medical University of Gdańsk Gdańsk Poland

Department of Allergology University Medical Center Groningen University of Groningen Groningen the Netherlands

Department of Dermatology and Allergy Biederstein School of Medicine Technical University of Munich Munich Germany

Department of Dermatology and Venereology Kepler University Hospital Johannes Kepler University Linz Austria

Department of Dermatology and Venereology Medical University of Graz Graz Austria

Department of Dermatology University of Cologne Cologne Germany

Department of Dermatology Venereology and Allergology Medical University of Gdańsk Gdańsk Poland

Department of Hematology and Oncology University Hospital CEITEC Masaryk University Brno Czech Republic

Department of Hematology Oncology and Stem Cell Transplantation Medical Center Faculty of Medicine University of Freiburg Freiburg Germany

Department of Hematology University Medical Center Groningen University of Groningen Groningen the Netherlands

Department of Internal Medicine 1 Division of Hematology and Hemostaseology Ludwig Boltzmann Institute for Hematology and Oncology Medical University of Vienna Vienna Austria

Department of Internal Medicine 3 Hematology and Oncology Kepler University Hospital Johannes Kepler University Linz Austria

Department of Internal Medicine 4 University Hospital Halle Halle Germany

Department of Medical Sciences Uppsala University Section of Hematology Uppsala University Hospital Uppsala Sweden

Department of Medicine Section of Hematology University of Verona Verona Italy

Department of Oncology Haematology Haemostaseology and Stem Cell Transplantation University Hospital RWTH Aachen Aachen Germany

Division of Allergy and Clinical Immunology University of Salerno Salerno Italy

Division of Allergy Department of Dermatology University of Basel Basel Switzerland

Division of Hematology Department of Medicine Stanford University School of Medicine Stanford Cancer Institute Stanford CA USA

Division of Hematology Fondazione IRCCS Policlinico San Matteo and University of Pavia Pavia Italy

Division of Hematology Istanbul Medical School University of Istanbul Istanbul Turkey

Faculty of Medicine and Health Sciences Department of Immunology Allergology Rheumatology University of Antwerp and Antwerp University Hospital Antwerp Belgium

Hämatologie und Onkologie 3 Medizinische Klinik Universitätsmedizin Mannheim Universität Heidelberg Mannheim Germany

Institute of Biostatistics and Analyses Ltd Spinoff Company of Masaryk University Brno Czech Republic

Laboratory of Hematology Pitié Salpêtrière Hospital Paris France

Pediatric Dermatology Unit Department of Medicine University of Padova Padova Italy

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