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

^*Korrespondierende/r Autor/-in für diese Arbeit

66 Zitate (Scopus)

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.

Originalsprache	Englisch
Zeitschrift	Allergy: European Journal of Allergy and Clinical Immunology
Jahrgang	75
Ausgabenummer	8
Seiten (von - bis)	1923-1934
Seitenumfang	12
ISSN	0105-4538
DOIs	https://doi.org/10.1111/all.14248
Publikationsstatus	Veröffentlicht - 01.08.2020

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WRS received honoraria from Novartis, Pfizer, AbbVie, Daiichi Sankyo, Amgen, Thermo Fisher, Diciphera, Incyte, Celgene, Jazz and travel grants from Pfizer and Roche. HOE received honoraria from ALK‐Abelló, Chiesi, MEDA Pharma, Novartis, Blueprint. KVG received honoraria from Novartis, Roche, BMS, Sanofi, Incyte and travel grants from Roche and AbbVie. ML received honoraria from Novartis. KH received honoraria from Novartis, ALK, Blueprint, Deciphera and research funding from Euroimmun. MB received honoraria from Amgen, Incyte, Pfizer and research funding from Novartis. CE received honoraria from Novartis and Pfizer. MJ received honoraria from Novartis, Blueprint, Deciphera. RZ is consultant Deciphera and Novartis. AZ received honoraria or participated in trials from AbbVie, Almirall, Beiersdorf Dermo Medical, Bencard Allergie, BMS, Celgene, Eli Lilly, GSK, Janssen‐Cilag, Miltenyi Biotec, Novartis, Sanofi‐Aventis, Takeda Pharma. MT received honoraria from Deciphera, Blueprint, Novartis. NB received honoraria from Astra Zeneca, Amgen, Novartis and BMS and research funding from Novartis. JP received honoraria from Alexion, BMS, Boehringer Ingelheim, Grünenthal, MSD, Novartis, Pfizer, Chugai. DF received honoraria from Novartis, Pfizer, Roche travel grants from Roche. VS received honoraria from Novartis, Termofisher, Shire, Stallergens. KB received honoraria from Novartis, Phadia (Thermo Fisher), Meda, BioMarin Pharmaceutical Inc outside. BA received honoraria from Novartis. AR received honoraria from Novartis, BMS, Deciphera, Blueprint, Baxalta/Shire and research funding from Novartis. JG received honoraria from Blueprint, Deciphera, Gilead, Incyte, Novartis and research funding from Blueprint, Celgene, CTI BioPharma, Deciphera, Gilead, Incyte, Pharmacyclics, Promedior, Seattle Genetics. JM received honoraria from for Novartis, Gilead, BMS. MD received honoraria from for Roche, AbbVie, Novartis, Gilead, AOP Pharmaceuticals, Janssen‐Cilag. PV has received honoraria from Novartis, Pfizer, Deciphera, Incyte, Blueprint, Celgene and research funds from Pfizer, Incyte, Celgene. JT, LN, AG, AI, CP, LM, ABF, FC, KS, RP, MN, PB, ASY, HH, MM, NJ, AK, OH, MA, AB, HCKN have nothing to disclose.

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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{744332597d864165a141d82f0c2577ca,

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.",

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 = "Funding Information: WRS received honoraria from Novartis, Pfizer, AbbVie, Daiichi Sankyo, Amgen, Thermo Fisher, Diciphera, Incyte, Celgene, Jazz and travel grants from Pfizer and Roche. HOE received honoraria from ALK‐Abell{\'o}, Chiesi, MEDA Pharma, Novartis, Blueprint. KVG received honoraria from Novartis, Roche, BMS, Sanofi, Incyte and travel grants from Roche and AbbVie. ML received honoraria from Novartis. KH received honoraria from Novartis, ALK, Blueprint, Deciphera and research funding from Euroimmun. MB received honoraria from Amgen, Incyte, Pfizer and research funding from Novartis. CE received honoraria from Novartis and Pfizer. MJ received honoraria from Novartis, Blueprint, Deciphera. RZ is consultant Deciphera and Novartis. AZ received honoraria or participated in trials from AbbVie, Almirall, Beiersdorf Dermo Medical, Bencard Allergie, BMS, Celgene, Eli Lilly, GSK, Janssen‐Cilag, Miltenyi Biotec, Novartis, Sanofi‐Aventis, Takeda Pharma. MT received honoraria from Deciphera, Blueprint, Novartis. NB received honoraria from Astra Zeneca, Amgen, Novartis and BMS and research funding from Novartis. JP received honoraria from Alexion, BMS, Boehringer Ingelheim, Gr{\"u}nenthal, MSD, Novartis, Pfizer, Chugai. DF received honoraria from Novartis, Pfizer, Roche travel grants from Roche. VS received honoraria from Novartis, Termofisher, Shire, Stallergens. KB received honoraria from Novartis, Phadia (Thermo Fisher), Meda, BioMarin Pharmaceutical Inc outside. BA received honoraria from Novartis. AR received honoraria from Novartis, BMS, Deciphera, Blueprint, Baxalta/Shire and research funding from Novartis. JG received honoraria from Blueprint, Deciphera, Gilead, Incyte, Novartis and research funding from Blueprint, Celgene, CTI BioPharma, Deciphera, Gilead, Incyte, Pharmacyclics, Promedior, Seattle Genetics. JM received honoraria from for Novartis, Gilead, BMS. MD received honoraria from for Roche, AbbVie, Novartis, Gilead, AOP Pharmaceuticals, Janssen‐Cilag. PV has received honoraria from Novartis, Pfizer, Deciphera, Incyte, Blueprint, Celgene and research funds from Pfizer, Incyte, Celgene. JT, LN, AG, AI, CP, LM, ABF, FC, KS, RP, MN, PB, ASY, HH, MM, NJ, AK, OH, MA, AB, HCKN have nothing to disclose. Publisher Copyright: {\textcopyright} 2020 EAACI and John Wiley and Sons A/S. Published by John Wiley and Sons Ltd. Copyright: Copyright 2020 Elsevier B.V., All rights reserved.",

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, Jg. 75, Nr. 8, S. 1923-1934. https://doi.org/10.1111/all.14248

Clinical features and survival of patients with indolent systemic mastocytosis defined by the updated WHO classification. / Trizuljak, Jakub; Sperr, Wolfgang R.; Nekvindová, Lucie et al.
in: Allergy: European Journal of Allergy and Clinical Immunology, Jahrgang 75, Nr. 8, 01.08.2020, S. 1923-1934.

Publikation: Beiträge in Fachzeitschriften › Zeitschriftenaufsätze › Forschung › Begutachtung

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

N1 - Funding Information: WRS received honoraria from Novartis, Pfizer, AbbVie, Daiichi Sankyo, Amgen, Thermo Fisher, Diciphera, Incyte, Celgene, Jazz and travel grants from Pfizer and Roche. HOE received honoraria from ALK‐Abelló, Chiesi, MEDA Pharma, Novartis, Blueprint. KVG received honoraria from Novartis, Roche, BMS, Sanofi, Incyte and travel grants from Roche and AbbVie. ML received honoraria from Novartis. KH received honoraria from Novartis, ALK, Blueprint, Deciphera and research funding from Euroimmun. MB received honoraria from Amgen, Incyte, Pfizer and research funding from Novartis. CE received honoraria from Novartis and Pfizer. MJ received honoraria from Novartis, Blueprint, Deciphera. RZ is consultant Deciphera and Novartis. AZ received honoraria or participated in trials from AbbVie, Almirall, Beiersdorf Dermo Medical, Bencard Allergie, BMS, Celgene, Eli Lilly, GSK, Janssen‐Cilag, Miltenyi Biotec, Novartis, Sanofi‐Aventis, Takeda Pharma. MT received honoraria from Deciphera, Blueprint, Novartis. NB received honoraria from Astra Zeneca, Amgen, Novartis and BMS and research funding from Novartis. JP received honoraria from Alexion, BMS, Boehringer Ingelheim, Grünenthal, MSD, Novartis, Pfizer, Chugai. DF received honoraria from Novartis, Pfizer, Roche travel grants from Roche. VS received honoraria from Novartis, Termofisher, Shire, Stallergens. KB received honoraria from Novartis, Phadia (Thermo Fisher), Meda, BioMarin Pharmaceutical Inc outside. BA received honoraria from Novartis. AR received honoraria from Novartis, BMS, Deciphera, Blueprint, Baxalta/Shire and research funding from Novartis. JG received honoraria from Blueprint, Deciphera, Gilead, Incyte, Novartis and research funding from Blueprint, Celgene, CTI BioPharma, Deciphera, Gilead, Incyte, Pharmacyclics, Promedior, Seattle Genetics. JM received honoraria from for Novartis, Gilead, BMS. MD received honoraria from for Roche, AbbVie, Novartis, Gilead, AOP Pharmaceuticals, Janssen‐Cilag. PV has received honoraria from Novartis, Pfizer, Deciphera, Incyte, Blueprint, Celgene and research funds from Pfizer, Incyte, Celgene. JT, LN, AG, AI, CP, LM, ABF, FC, KS, RP, MN, PB, ASY, HH, MM, NJ, AK, OH, MA, AB, HCKN have nothing to disclose. Publisher Copyright: © 2020 EAACI and John Wiley and Sons A/S. Published by John Wiley and Sons Ltd. Copyright: Copyright 2020 Elsevier B.V., All rights reserved.

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.

UR - https://www.scopus.com/pages/publications/85081620026

U2 - 10.1111/all.14248

DO - 10.1111/all.14248

M3 - Journal articles

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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