Jakub Trizuljak1, Wolfgang R Sperr2, Lucie Nekvindová3, Hanneke O Elberink4, Karoline V Gleixner2, Aleksandra Gorska5, Magdalena Lange6, Karin Hartmann7,8, Anja Illerhaus8, Massimiliano Bonifacio9, Cecelia Perkins10, Chiara Elena11, Luca Malcovati11, Anna B Fortina12, Khalid Shoumariyeh13, Mohamad Jawhar14, Roberta Zanotti9, Patrizia Bonadonna15, Francesca Caroppo12, Alexander Zink16, Massimo Triggiani17, Roberta Parente17, Nikolas von Bubnoff13, Akif S Yavuz18, Hans Hägglund19, Mattias Mattsson19, Jens Panse20, Nadja Jäkel21, Alex Kilbertus22, Olivier Hermine23, Michel Arock24, David Fuchs25, Vito Sabato26, Knut Brockow16, Agnes Bretterklieber27, Marek Niedoszytko5, Björn van Anrooij4, Andreas Reiter14, Jason Gotlib10, Hanneke C Kluin-Nelemans28, Jiri Mayer1, Michael Doubek1, Peter Valent2. 1. Department of Hematology and Oncology, University Hospital, CEITEC Masaryk University, Brno, Czech Republic. 2. Department of Internal Medicine I, Division of Hematology and Hemostaseology, Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, Vienna, Austria. 3. Institute of Biostatistics and Analyses, Ltd., Spinoff Company of Masaryk University, Brno, Czech Republic. 4. Department of Allergology, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands. 5. Department of Allergology, Medical University of Gdańsk, Gdańsk, Poland. 6. Department of Dermatology, Venereology and Allergology, Medical University of Gdańsk, Gdańsk, Poland. 7. Division of Allergy, Department of Dermatology, University of Basel, Basel, Switzerland. 8. Department of Dermatology, University of Cologne, Cologne, Germany. 9. Department of Medicine, Section of Hematology, University of Verona, Verona, Italy. 10. Division of Hematology, Department of Medicine, Stanford University, School of Medicine/Stanford Cancer Institute, Stanford, CA, USA. 11. Division of Hematology, Fondazione IRCCS Policlinico San Matteo & University of Pavia, Pavia, Italy. 12. Pediatric Dermatology Unit, Department of Medicine, University of Padova, Padova, Italy. 13. Department of Hematology, Oncology and Stem Cell Transplantation, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany. 14. Hämatologie und Onkologie, III. Medizinische Klinik, Universitätsmedizin Mannheim, Universität Heidelberg, Mannheim, Germany. 15. Allergy Unit, Verona University Hospital, Verona, Italy. 16. Department of Dermatology and Allergy Biederstein, School of Medicine, Technical University of Munich, Munich, Germany. 17. Division of Allergy and Clinical Immunology, University of Salerno, Salerno, Italy. 18. Division of Hematology, Istanbul Medical School, University of Istanbul, Istanbul, Turkey. 19. Department of Medical Sciences, Uppsala University, Section of Hematology, Uppsala University Hospital, Uppsala, Sweden. 20. Department of Oncology, Haematology, Haemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen, Germany. 21. Department of Internal Medicine IV, University Hospital Halle, Halle, Germany. 22. Department of Dermatology and Venereology, Kepler University Hospital, Johannes Kepler University, Linz, Austria. 23. 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 (APHP), Paris, France. 24. Laboratory of Hematology, Pitié-Salpêtrière Hospital, Paris, France. 25. Department of Internal Medicine 3, Hematology and Oncology, Kepler University Hospital, Johannes Kepler University, Linz, Austria. 26. Faculty of Medicine and Health Sciences, Department of Immunology-Allergology-Rheumatology, University of Antwerp and Antwerp University Hospital, Antwerp, Belgium. 27. Department of Dermatology and Venereology, Medical University of Graz, Graz, Austria. 28. Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands.
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.
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.
Authors: M Jawhar; J Schwaab; S Schnittger; M Meggendorfer; M Pfirrmann; K Sotlar; H-P Horny; G Metzgeroth; S Kluger; N Naumann; C Haferlach; T Haferlach; P Valent; W-K Hofmann; A Fabarius; N C P Cross; A Reiter Journal: Leukemia Date: 2015-10-14 Impact factor: 11.528
Authors: Cristina Teodosio; Andrea Mayado; Laura Sánchez-Muñoz; José M Morgado; María Jara-Acevedo; Ivan Álvarez-Twose; Andrés C García-Montero; Almudena Matito; Caldas Caldas; Luis Escribano; Alberto Orfao Journal: J Leukoc Biol Date: 2014-11-07 Impact factor: 4.962
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Authors: G Hoermann; K V Gleixner; G E Dinu; M Kundi; G Greiner; F Wimazal; E Hadzijusufovic; G Mitterbauer; C Mannhalter; P Valent; W R Sperr Journal: Allergy Date: 2014-04-17 Impact factor: 13.146
Authors: Peter Valent; Michel Arock; Patrizia Bonadonna; Knut Brockow; Sigurd Broesby-Olsen; Luis Escribano; Karoline V Gleixner; Clive Grattan; Emir Hadzijusufovic; Hans Hägglund; Olivier Hermine; Hans-Peter Horny; Hanneke C Kluin-Nelemans; Marcus Maurer; Marek Niedoszytko; Boguslaw Nedoszytko; Gunnar Nilsson; Hanneke N G Oude-Elberink; Alberto Orfao; Deepti Radia; Andreas Reiter; Frank Siebenhaar; Karl Sotlar; Wolfgang R Sperr; Massimo Triggiani; Jaap J VanDoormaal; Judit Várkonyi; Selim Yavuz; Karin Hartmann Journal: Wien Klin Wochenschr Date: 2012-11-20 Impact factor: 1.704
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Authors: Jason Gotlib; Tracy I George; Melody C Carter; K Frank Austen; Bruce Bochner; Daniel F Dwyer; Jonathan J Lyons; Matthew J Hamilton; Joseph Butterfield; Patrizia Bonadonna; Catherine Weiler; Stephen J Galli; Lawrence B Schwartz; Hanneke Oude Elberink; Anne Maitland; Theoharis Theoharides; Celalettin Ustun; Hans-Peter Horny; Alberto Orfao; Michael Deininger; Deepti Radia; Mohamad Jawhar; Hanneke Kluin-Nelemans; Dean D Metcalfe; Michel Arock; Wolfgang R Sperr; Peter Valent; Mariana Castells; Cem Akin Journal: J Allergy Clin Immunol Date: 2021-03-11 Impact factor: 14.290
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