Clinical features and survival of patients with indolent systemic mastocytosis defined by the updated WHO classification

Jakub Trizuljak; Wolfgang R. Sperr; Lucie Nekvindová; Hanneke O. Elberink; Karoline V. Gleixner; Aleksandra Gorska; Magdalena Lange; Karin Hartmann; Anja Illerhaus; Massimiliano Bonifacio; Cecelia Perkins; Chiara Elena; Luca Malcovati; Anna B. Fortina; Khalid Shoumariyeh; Mohamad Jawhar; Roberta Zanotti; Patrizia Bonadonna; Francesca Caroppo; Alexander Zink; Massimo Triggiani; Roberta Parente; Nikolas von Bubnoff; Akif S. Yavuz; Hans Hägglund; Mattias Mattsson; Jens Panse; Nadja Jäkel; Alex Kilbertus; Olivier Hermine; Michel Arock; David Fuchs; Vito Sabato; Knut Brockow; Agnes Bretterklieber; Marek Niedoszytko; Björn van Anrooij; Andreas Reiter; Jason Gotlib; Hanneke C. Kluin-Nelemans; Jiri Mayer; Michael Doubek; Peter Valent

doi:10.1111/all.14248

Clinical features and survival of patients with indolent systemic mastocytosis defined by the updated WHO classification

Jakub Trizuljak, Wolfgang R. Sperr, Lucie Nekvindová, Hanneke O. Elberink, Karoline V. Gleixner, Aleksandra Gorska, Magdalena Lange, Karin Hartmann, Anja Illerhaus, Massimiliano Bonifacio, Cecelia Perkins, Chiara Elena, Luca Malcovati, Anna B. Fortina, Khalid Shoumariyeh, Mohamad Jawhar, Roberta Zanotti, Patrizia Bonadonna, Francesca Caroppo, Alexander ZinkMassimo Triggiani, Roberta Parente, Nikolas von Bubnoff, Akif S. Yavuz, Hans Hägglund, Mattias Mattsson, Jens Panse, Nadja Jäkel, Alex Kilbertus, Olivier Hermine, Michel Arock, David Fuchs, Vito Sabato, Knut Brockow, Agnes Bretterklieber, Marek Niedoszytko, Björn van Anrooij, Andreas Reiter, Jason Gotlib, Hanneke C. Kluin-Nelemans, Jiri Mayer, Michael Doubek, Peter Valent

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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.

Original language	English
Pages (from-to)	1923-1934
Number of pages	12
Journal	Allergy: European Journal of Allergy and Clinical Immunology
Volume	75
Issue number	8
DOIs	https://doi.org/10.1111/all.14248
State	Published - 1 Aug 2020

Keywords

WHO classification
cutaneous mastocytosis
indolent systemic mastocytosis
prognostication
survival

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10.1111/all.14248

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Trizuljak, J., Sperr, W. R., Nekvindová, L., Elberink, H. O., Gleixner, K. V., Gorska, A., Lange, M., Hartmann, K., Illerhaus, A., Bonifacio, M., Perkins, C., Elena, C., Malcovati, L., Fortina, A. B., Shoumariyeh, K., Jawhar, M., Zanotti, R., Bonadonna, P., Caroppo, F., ... Valent, P. (2020). Clinical features and survival of patients with indolent systemic mastocytosis defined by the updated WHO classification. Allergy: European Journal of Allergy and Clinical Immunology, 75(8), 1923-1934. https://doi.org/10.1111/all.14248

@article{3c60a73b7b574554a0af1b16a05135b8,

title = "Clinical features and survival of patients with indolent systemic mastocytosis defined by the updated WHO classification",

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.",

keywords = "WHO classification, cutaneous mastocytosis, indolent systemic mastocytosis, prognostication, survival",

author = "Jakub Trizuljak and Sperr, {Wolfgang R.} and Lucie Nekvindov{\'a} and Elberink, {Hanneke O.} and Gleixner, {Karoline V.} and Aleksandra Gorska and Magdalena Lange and Karin Hartmann and Anja Illerhaus and Massimiliano Bonifacio and Cecelia Perkins and Chiara Elena and Luca Malcovati and Fortina, {Anna B.} and Khalid Shoumariyeh and Mohamad Jawhar and Roberta Zanotti and Patrizia Bonadonna and Francesca Caroppo and Alexander Zink and Massimo Triggiani and Roberta Parente and {von Bubnoff}, Nikolas and Yavuz, {Akif S.} and Hans H{\"a}gglund and Mattias Mattsson and Jens Panse and Nadja J{\"a}kel and Alex Kilbertus and Olivier Hermine and Michel Arock and David Fuchs and Vito Sabato and Knut Brockow and Agnes Bretterklieber and Marek Niedoszytko and {van Anrooij}, Bj{\"o}rn and Andreas Reiter and Jason Gotlib and Kluin-Nelemans, {Hanneke C.} and Jiri Mayer and Michael Doubek and Peter Valent",

note = "Publisher Copyright: {\textcopyright} 2020 EAACI and John Wiley and Sons A/S. Published by John Wiley and Sons Ltd.",

year = "2020",

month = aug,

day = "1",

doi = "10.1111/all.14248",

language = "English",

volume = "75",

pages = "1923--1934",

journal = "Allergy: European Journal of Allergy and Clinical Immunology",

issn = "0105-4538",

publisher = "Wiley-Blackwell Publishing Ltd",

number = "8",

}

Trizuljak, J, Sperr, WR, Nekvindová, L, Elberink, HO, Gleixner, KV, Gorska, A, Lange, M, Hartmann, K, Illerhaus, A, Bonifacio, M, Perkins, C, Elena, C, Malcovati, L, Fortina, AB, Shoumariyeh, K, Jawhar, M, Zanotti, R, Bonadonna, P, Caroppo, F, Zink, A, Triggiani, M, Parente, R, von Bubnoff, N, Yavuz, AS, Hägglund, H, Mattsson, M, Panse, J, Jäkel, N, Kilbertus, A, Hermine, O, Arock, M, Fuchs, D, Sabato, V, Brockow, K, Bretterklieber, A, Niedoszytko, M, van Anrooij, B, Reiter, A, Gotlib, J, Kluin-Nelemans, HC, Mayer, J, Doubek, M & Valent, P 2020, 'Clinical features and survival of patients with indolent systemic mastocytosis defined by the updated WHO classification', Allergy: European Journal of Allergy and Clinical Immunology, vol. 75, no. 8, pp. 1923-1934. https://doi.org/10.1111/all.14248

TY - JOUR

T1 - Clinical features and survival of patients with indolent systemic mastocytosis defined by the updated WHO classification

AU - Trizuljak, Jakub

AU - Sperr, Wolfgang R.

AU - Nekvindová, Lucie

AU - Elberink, Hanneke O.

AU - Gleixner, Karoline V.

AU - Gorska, Aleksandra

AU - Lange, Magdalena

AU - Hartmann, Karin

AU - Illerhaus, Anja

AU - Bonifacio, Massimiliano

AU - Perkins, Cecelia

AU - Elena, Chiara

AU - Malcovati, Luca

AU - Fortina, Anna B.

AU - Shoumariyeh, Khalid

AU - Jawhar, Mohamad

AU - Zanotti, Roberta

AU - Bonadonna, Patrizia

AU - Caroppo, Francesca

AU - Zink, Alexander

AU - Triggiani, Massimo

AU - Parente, Roberta

AU - von Bubnoff, Nikolas

AU - Yavuz, Akif S.

AU - Hägglund, Hans

AU - Mattsson, Mattias

AU - Panse, Jens

AU - Jäkel, Nadja

AU - Kilbertus, Alex

AU - Hermine, Olivier

AU - Arock, Michel

AU - Fuchs, David

AU - Sabato, Vito

AU - Brockow, Knut

AU - Bretterklieber, Agnes

AU - Niedoszytko, Marek

AU - van Anrooij, Björn

AU - Reiter, Andreas

AU - Gotlib, Jason

AU - Kluin-Nelemans, Hanneke C.

AU - Mayer, Jiri

AU - Doubek, Michael

AU - Valent, Peter

PY - 2020/8/1

Y1 - 2020/8/1

N2 - 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.

AB - 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.

KW - WHO classification

KW - cutaneous mastocytosis

KW - indolent systemic mastocytosis

KW - prognostication

KW - survival

UR - http://www.scopus.com/inward/record.url?scp=85081620026&partnerID=8YFLogxK

U2 - 10.1111/all.14248

DO - 10.1111/all.14248

M3 - Article

C2 - 32108361

AN - SCOPUS:85081620026

SN - 0105-4538

VL - 75

SP - 1923

EP - 1934

JO - Allergy: European Journal of Allergy and Clinical Immunology

JF - Allergy: European Journal of Allergy and Clinical Immunology

IS - 8

ER -

Clinical features and survival of patients with indolent systemic mastocytosis defined by the updated WHO classification

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