Scoring the Risk of Having Systemic Mastocytosis in Adult Patients with Mastocytosis in the Skin

David Fuchs; Alex Kilbertus; Karin Kofler; Nikolas von Bubnoff; Khalid Shoumariyeh; Roberta Zanotti; Patrizia Bonadonna; Luigi Scaffidi; Michael Doubek; Hanneke Oude Elberink; Lambert F.R. Span; Olivier Hermine; Chiara Elena; Pietro Benvenuti; Akif Selim Yavuz; Knut Brockow; Alexander Zink; Elisabeth Aberer; Aleksandra Gorska; Jan Romantowski; Emir Hadzijusufovic; Anna Belloni Fortina; Francesca Caroppo; Cecelia Perkins; Anja Illerhaus; Jens Panse; Vladan Vucinic; Mohamad Jawhar; Vito Sabato; Massimo Triggiani; Roberta Parente; Anna Bergström; Christine Breynaert; Jason Gotlib; Andreas Reiter; Karin Hartmann; Marek Niedoszytko; Michel Arock; Hanneke C. Kluin-Nelemans; Wolfgang R. Sperr; Rosemarie Greul; Peter Valent

doi:10.1016/j.jaip.2020.12.022

Scoring the Risk of Having Systemic Mastocytosis in Adult Patients with Mastocytosis in the Skin

David Fuchs^*, Alex Kilbertus, Karin Kofler, Nikolas von Bubnoff, Khalid Shoumariyeh, Roberta Zanotti, Patrizia Bonadonna, Luigi Scaffidi, Michael Doubek, Hanneke Oude Elberink, Lambert F.R. Span, Olivier Hermine, Chiara Elena, Pietro Benvenuti, Akif Selim Yavuz, Knut Brockow, Alexander Zink, Elisabeth Aberer, Aleksandra Gorska, Jan RomantowskiEmir Hadzijusufovic, Anna Belloni Fortina, Francesca Caroppo, Cecelia Perkins, Anja Illerhaus, Jens Panse, Vladan Vucinic, Mohamad Jawhar, Vito Sabato, Massimo Triggiani, Roberta Parente, Anna Bergström, Christine Breynaert, Jason Gotlib, Andreas Reiter, Karin Hartmann, Marek Niedoszytko, Michel Arock, Hanneke C. Kluin-Nelemans, Wolfgang R. Sperr, Rosemarie Greul, Peter Valent

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

26 Zitate (Scopus)

Abstract

Background: Mastocytosis in adults often presents with skin lesions. A bone marrow biopsy is necessary to confirm or exclude the presence of systemic mastocytosis (SM) in these cases. When a bone marrow biopsy is not performed, the provisional diagnosis is mastocytosis in the skin (MIS). No generally accepted scoring system has been established to estimate the risk of SM in these patients. Objective: To develop a risk score to predict SM in adults with MIS. Methods: We examined 1145 patients with MIS from the European Competence Network on Mastocytosis Registry who underwent a bone marrow biopsy. A total of 944 patients had SM and 201 patients had cutaneous mastocytosis; 63.7% were female, and 36.3% were male. Median age was 44 ± 13.3 years. The median serum tryptase level amounted to 29.3 ± 81.9 ng/mL. We established a multivariate regression model using the whole population of patients as a training and validation set (bootstrapping). A risk score was developed and validated with receiver-operating curves. Results: In the multivariate model, the tryptase level (P <.001), constitutional/cardiovascular symptoms (P =.014), and bone symptoms/osteoporosis (P <.001) were independent predictors of SM (P <.001; sensitivity, 90.7%; specificity, 69.1%). A 6-point risk score was established (risk, 10.7%-98.0%) and validated. Conclusions: Using a large data set of the European Competence Network on Mastocytosis Registry, we created a risk score to predict the presence of SM in patients with MIS. Although the score will need further validation in independent cohorts, our score seems to discriminate safely between patients with SM and with pure cutaneous mastocytosis.

Originalsprache	Englisch
Zeitschrift	Journal of Allergy and Clinical Immunology: In Practice
Jahrgang	9
Ausgabenummer	4
Seiten (von - bis)	1705-1712.e4
ISSN	2213-2198
DOIs	https://doi.org/10.1016/j.jaip.2020.12.022
Publikationsstatus	Veröffentlicht - 04.2021

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This work was supported by Deutsche Forschungsgemeinschaft (DFG; grant no. RA 2838 to A.I.) and by the Koeln Fortune Program, Faculty of Medicine , University of Cologne (grant no. 216/2016 to A.I.). P.V. and his team were supported by the Austrian Science Fund (Fonds zur Förderung der wissenschaftlichen Forschung; grant nos. F4704-B20 and P32470-B). V.S. is a senior clinical researcher of the Research Foundation Flanders/Fonds Wetenschappelijk Onderzoek (FWO: 1804518N). Conflicts of interest: D. Fuchs received honoraria from Novartis, outside the submitted work. N. von Bubnoff received honoraria from Amgen, Astra Zeneca, BMS, and Novartis, and research funding from Novartis . J. Panse reports personal fees from Alexion, BMS, Boehringer Ingelheim, Grünenthal, MSD, Novartis, Pfizer, Chugai, Roche, Apellis, and Blueprint medicines, all outside the submitted work. M. Triggiani received fee for Advisory Board from Novartis, Deciphera, and Blueprint Medicines. H. C. Kluin-Nelemans received financial support from Novartis . W. R. Sperr received honoraria from Novartis, Pfizer, AbbVie, Daiichi Sankyo, Amgen, Thermo Fisher, Deciphera, Incyte, Celgene, and Jazz. Valent received honoraria from Deciphera, Blueprint, Celgene, Novartis, Pfizer, and Incyte, all outside the submitted work.

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10.1016/j.jaip.2020.12.022

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Fuchs, D., Kilbertus, A., Kofler, K., von Bubnoff, N., Shoumariyeh, K., Zanotti, R., Bonadonna, P., Scaffidi, L., Doubek, M., Elberink, H. O., Span, L. F. R., Hermine, O., Elena, C., Benvenuti, P., Yavuz, A. S., Brockow, K., Zink, A., Aberer, E., Gorska, A., ... Valent, P. (2021). Scoring the Risk of Having Systemic Mastocytosis in Adult Patients with Mastocytosis in the Skin. Journal of Allergy and Clinical Immunology: In Practice, 9(4), 1705-1712.e4. https://doi.org/10.1016/j.jaip.2020.12.022

@article{8bd9501e48964d0daab1a4d47890a664,

title = "Scoring the Risk of Having Systemic Mastocytosis in Adult Patients with Mastocytosis in the Skin",

abstract = "Background: Mastocytosis in adults often presents with skin lesions. A bone marrow biopsy is necessary to confirm or exclude the presence of systemic mastocytosis (SM) in these cases. When a bone marrow biopsy is not performed, the provisional diagnosis is mastocytosis in the skin (MIS). No generally accepted scoring system has been established to estimate the risk of SM in these patients. Objective: To develop a risk score to predict SM in adults with MIS. Methods: We examined 1145 patients with MIS from the European Competence Network on Mastocytosis Registry who underwent a bone marrow biopsy. A total of 944 patients had SM and 201 patients had cutaneous mastocytosis; 63.7\% were female, and 36.3\% were male. Median age was 44 ± 13.3 years. The median serum tryptase level amounted to 29.3 ± 81.9 ng/mL. We established a multivariate regression model using the whole population of patients as a training and validation set (bootstrapping). A risk score was developed and validated with receiver-operating curves. Results: In the multivariate model, the tryptase level (P <.001), constitutional/cardiovascular symptoms (P =.014), and bone symptoms/osteoporosis (P <.001) were independent predictors of SM (P <.001; sensitivity, 90.7\%; specificity, 69.1\%). A 6-point risk score was established (risk, 10.7\%-98.0\%) and validated. Conclusions: Using a large data set of the European Competence Network on Mastocytosis Registry, we created a risk score to predict the presence of SM in patients with MIS. Although the score will need further validation in independent cohorts, our score seems to discriminate safely between patients with SM and with pure cutaneous mastocytosis.",

author = "David Fuchs and Alex Kilbertus and Karin Kofler and \{von Bubnoff\}, Nikolas and Khalid Shoumariyeh and Roberta Zanotti and Patrizia Bonadonna and Luigi Scaffidi and Michael Doubek and Elberink, \{Hanneke Oude\} and Span, \{Lambert F.R.\} and Olivier Hermine and Chiara Elena and Pietro Benvenuti and Yavuz, \{Akif Selim\} and Knut Brockow and Alexander Zink and Elisabeth Aberer and Aleksandra Gorska and Jan Romantowski and Emir Hadzijusufovic and Fortina, \{Anna Belloni\} and Francesca Caroppo and Cecelia Perkins and Anja Illerhaus and Jens Panse and Vladan Vucinic and Mohamad Jawhar and Vito Sabato and Massimo Triggiani and Roberta Parente and Anna Bergstr{\"o}m and Christine Breynaert and Jason Gotlib and Andreas Reiter and Karin Hartmann and Marek Niedoszytko and Michel Arock and Kluin-Nelemans, \{Hanneke C.\} and Sperr, \{Wolfgang R.\} and Rosemarie Greul and Peter Valent",

note = "Funding Information: This work was supported by Deutsche Forschungsgemeinschaft (DFG; grant no. RA 2838 to A.I.) and by the Koeln Fortune Program, Faculty of Medicine , University of Cologne (grant no. 216/2016 to A.I.). P.V. and his team were supported by the Austrian Science Fund (Fonds zur F{\"o}rderung der wissenschaftlichen Forschung; grant nos. F4704-B20 and P32470-B). V.S. is a senior clinical researcher of the Research Foundation Flanders/Fonds Wetenschappelijk Onderzoek (FWO: 1804518N). Funding Information: Conflicts of interest: D. Fuchs received honoraria from Novartis, outside the submitted work. N. von Bubnoff received honoraria from Amgen, Astra Zeneca, BMS, and Novartis, and research funding from Novartis . J. Panse reports personal fees from Alexion, BMS, Boehringer Ingelheim, Gr{\"u}nenthal, MSD, Novartis, Pfizer, Chugai, Roche, Apellis, and Blueprint medicines, all outside the submitted work. M. Triggiani received fee for Advisory Board from Novartis, Deciphera, and Blueprint Medicines. H. C. Kluin-Nelemans received financial support from Novartis . W. R. Sperr received honoraria from Novartis, Pfizer, AbbVie, Daiichi Sankyo, Amgen, Thermo Fisher, Deciphera, Incyte, Celgene, and Jazz. Valent received honoraria from Deciphera, Blueprint, Celgene, Novartis, Pfizer, and Incyte, all outside the submitted work. Publisher Copyright: {\textcopyright} 2020 American Academy of Allergy, Asthma \& Immunology Copyright: Copyright 2021 Elsevier B.V., All rights reserved.",

year = "2021",

month = apr,

doi = "10.1016/j.jaip.2020.12.022",

language = "English",

volume = "9",

pages = "1705--1712.e4",

journal = "Journal of Allergy and Clinical Immunology: In Practice",

issn = "2213-2198",

publisher = "American Academy of Allergy, Asthma and Immunology",

number = "4",

}

Fuchs, D, Kilbertus, A, Kofler, K, von Bubnoff, N, Shoumariyeh, K, Zanotti, R, Bonadonna, P, Scaffidi, L, Doubek, M, Elberink, HO, Span, LFR, Hermine, O, Elena, C, Benvenuti, P, Yavuz, AS, Brockow, K, Zink, A, Aberer, E, Gorska, A, Romantowski, J, Hadzijusufovic, E, Fortina, AB, Caroppo, F, Perkins, C, Illerhaus, A, Panse, J, Vucinic, V, Jawhar, M, Sabato, V, Triggiani, M, Parente, R, Bergström, A, Breynaert, C, Gotlib, J, Reiter, A, Hartmann, K, Niedoszytko, M, Arock, M, Kluin-Nelemans, HC, Sperr, WR, Greul, R & Valent, P 2021, 'Scoring the Risk of Having Systemic Mastocytosis in Adult Patients with Mastocytosis in the Skin', Journal of Allergy and Clinical Immunology: In Practice, Jg. 9, Nr. 4, S. 1705-1712.e4. https://doi.org/10.1016/j.jaip.2020.12.022

TY - JOUR

T1 - Scoring the Risk of Having Systemic Mastocytosis in Adult Patients with Mastocytosis in the Skin

AU - Fuchs, David

AU - Kilbertus, Alex

AU - Kofler, Karin

AU - von Bubnoff, Nikolas

AU - Shoumariyeh, Khalid

AU - Zanotti, Roberta

AU - Bonadonna, Patrizia

AU - Scaffidi, Luigi

AU - Doubek, Michael

AU - Elberink, Hanneke Oude

AU - Span, Lambert F.R.

AU - Hermine, Olivier

AU - Elena, Chiara

AU - Benvenuti, Pietro

AU - Yavuz, Akif Selim

AU - Brockow, Knut

AU - Zink, Alexander

AU - Aberer, Elisabeth

AU - Gorska, Aleksandra

AU - Romantowski, Jan

AU - Hadzijusufovic, Emir

AU - Fortina, Anna Belloni

AU - Caroppo, Francesca

AU - Perkins, Cecelia

AU - Illerhaus, Anja

AU - Panse, Jens

AU - Vucinic, Vladan

AU - Jawhar, Mohamad

AU - Sabato, Vito

AU - Triggiani, Massimo

AU - Parente, Roberta

AU - Bergström, Anna

AU - Breynaert, Christine

AU - Gotlib, Jason

AU - Reiter, Andreas

AU - Hartmann, Karin

AU - Niedoszytko, Marek

AU - Arock, Michel

AU - Kluin-Nelemans, Hanneke C.

AU - Sperr, Wolfgang R.

AU - Greul, Rosemarie

AU - Valent, Peter

N1 - Funding Information: This work was supported by Deutsche Forschungsgemeinschaft (DFG; grant no. RA 2838 to A.I.) and by the Koeln Fortune Program, Faculty of Medicine , University of Cologne (grant no. 216/2016 to A.I.). P.V. and his team were supported by the Austrian Science Fund (Fonds zur Förderung der wissenschaftlichen Forschung; grant nos. F4704-B20 and P32470-B). V.S. is a senior clinical researcher of the Research Foundation Flanders/Fonds Wetenschappelijk Onderzoek (FWO: 1804518N). Funding Information: Conflicts of interest: D. Fuchs received honoraria from Novartis, outside the submitted work. N. von Bubnoff received honoraria from Amgen, Astra Zeneca, BMS, and Novartis, and research funding from Novartis . J. Panse reports personal fees from Alexion, BMS, Boehringer Ingelheim, Grünenthal, MSD, Novartis, Pfizer, Chugai, Roche, Apellis, and Blueprint medicines, all outside the submitted work. M. Triggiani received fee for Advisory Board from Novartis, Deciphera, and Blueprint Medicines. H. C. Kluin-Nelemans received financial support from Novartis . W. R. Sperr received honoraria from Novartis, Pfizer, AbbVie, Daiichi Sankyo, Amgen, Thermo Fisher, Deciphera, Incyte, Celgene, and Jazz. Valent received honoraria from Deciphera, Blueprint, Celgene, Novartis, Pfizer, and Incyte, all outside the submitted work. Publisher Copyright: © 2020 American Academy of Allergy, Asthma & Immunology Copyright: Copyright 2021 Elsevier B.V., All rights reserved.

PY - 2021/4

Y1 - 2021/4

N2 - Background: Mastocytosis in adults often presents with skin lesions. A bone marrow biopsy is necessary to confirm or exclude the presence of systemic mastocytosis (SM) in these cases. When a bone marrow biopsy is not performed, the provisional diagnosis is mastocytosis in the skin (MIS). No generally accepted scoring system has been established to estimate the risk of SM in these patients. Objective: To develop a risk score to predict SM in adults with MIS. Methods: We examined 1145 patients with MIS from the European Competence Network on Mastocytosis Registry who underwent a bone marrow biopsy. A total of 944 patients had SM and 201 patients had cutaneous mastocytosis; 63.7% were female, and 36.3% were male. Median age was 44 ± 13.3 years. The median serum tryptase level amounted to 29.3 ± 81.9 ng/mL. We established a multivariate regression model using the whole population of patients as a training and validation set (bootstrapping). A risk score was developed and validated with receiver-operating curves. Results: In the multivariate model, the tryptase level (P <.001), constitutional/cardiovascular symptoms (P =.014), and bone symptoms/osteoporosis (P <.001) were independent predictors of SM (P <.001; sensitivity, 90.7%; specificity, 69.1%). A 6-point risk score was established (risk, 10.7%-98.0%) and validated. Conclusions: Using a large data set of the European Competence Network on Mastocytosis Registry, we created a risk score to predict the presence of SM in patients with MIS. Although the score will need further validation in independent cohorts, our score seems to discriminate safely between patients with SM and with pure cutaneous mastocytosis.

AB - Background: Mastocytosis in adults often presents with skin lesions. A bone marrow biopsy is necessary to confirm or exclude the presence of systemic mastocytosis (SM) in these cases. When a bone marrow biopsy is not performed, the provisional diagnosis is mastocytosis in the skin (MIS). No generally accepted scoring system has been established to estimate the risk of SM in these patients. Objective: To develop a risk score to predict SM in adults with MIS. Methods: We examined 1145 patients with MIS from the European Competence Network on Mastocytosis Registry who underwent a bone marrow biopsy. A total of 944 patients had SM and 201 patients had cutaneous mastocytosis; 63.7% were female, and 36.3% were male. Median age was 44 ± 13.3 years. The median serum tryptase level amounted to 29.3 ± 81.9 ng/mL. We established a multivariate regression model using the whole population of patients as a training and validation set (bootstrapping). A risk score was developed and validated with receiver-operating curves. Results: In the multivariate model, the tryptase level (P <.001), constitutional/cardiovascular symptoms (P =.014), and bone symptoms/osteoporosis (P <.001) were independent predictors of SM (P <.001; sensitivity, 90.7%; specificity, 69.1%). A 6-point risk score was established (risk, 10.7%-98.0%) and validated. Conclusions: Using a large data set of the European Competence Network on Mastocytosis Registry, we created a risk score to predict the presence of SM in patients with MIS. Although the score will need further validation in independent cohorts, our score seems to discriminate safely between patients with SM and with pure cutaneous mastocytosis.

UR - https://www.scopus.com/pages/publications/85099805558

UR - https://www.mendeley.com/catalogue/749b8d20-a7fc-3383-855e-fd4cc4d004b1/

U2 - 10.1016/j.jaip.2020.12.022

DO - 10.1016/j.jaip.2020.12.022

M3 - Journal articles

C2 - 33346151

AN - SCOPUS:85099805558

SN - 2213-2198

VL - 9

SP - 1705-1712.e4

JO - Journal of Allergy and Clinical Immunology: In Practice

JF - Journal of Allergy and Clinical Immunology: In Practice

IS - 4

ER -

Scoring the Risk of Having Systemic Mastocytosis in Adult Patients with Mastocytosis in the Skin

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