International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study

Wolfgang R. Sperr; Michael Kundi; Ivan Alvarez-Twose; Bjorn van Anrooij; Joanna N.G. Oude Elberink; Aleksandra Gorska; Marek Niedoszytko; Karoline V. Gleixner; Emir Hadzijusufovic; Roberta Zanotti; Patrizia Bonadonna; Massimiliano Bonifacio; Cecelia Perkins; Anja Illerhaus; Chiara Elena; Serena Merante; Khalid Shoumariyeh; Nikolas von Bubnoff; Roberta Parente; Mohamad Jawhar; Anna Belloni Fortina; Francesca Caroppo; Knut Brockow; Alexander Zink; David Fuchs; Alex J. Kilbertus; Akif Selim Yavuz; Michael Doubek; Hans Hägglund; Jens Panse; Vito Sabato; Agnes Bretterklieber; Dietger Niederwieser; Christine Breynaert; Karin Hartmann; Massimo Triggiani; Boguslaw Nedoszytko; Andreas Reiter; Alberto Orfao; Olivier Hermine; Jason Gotlib; Michel Arock; Hanneke C. Kluin-Nelemans; Peter Valent

doi:10.1016/S2352-3026(19)30166-8

International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study

Wolfgang R. Sperr, Michael Kundi, Ivan Alvarez-Twose, Bjorn van Anrooij, Joanna N.G. Oude Elberink, Aleksandra Gorska, Marek Niedoszytko, Karoline V. Gleixner, Emir Hadzijusufovic, Roberta Zanotti, Patrizia Bonadonna, Massimiliano Bonifacio, Cecelia Perkins, Anja Illerhaus, Chiara Elena, Serena Merante, Khalid Shoumariyeh, Nikolas von Bubnoff, Roberta Parente, Mohamad JawharAnna Belloni Fortina, Francesca Caroppo, Knut Brockow, Alexander Zink, David Fuchs, Alex J. Kilbertus, Akif Selim Yavuz, Michael Doubek, Hans Hägglund, Jens Panse, Vito Sabato, Agnes Bretterklieber, Dietger Niederwieser, Christine Breynaert, Karin Hartmann, Massimo Triggiani, Boguslaw Nedoszytko, Andreas Reiter, Alberto Orfao, Olivier Hermine, Jason Gotlib, Michel Arock, Hanneke C. Kluin-Nelemans, Peter Valent

27 Citations (Scopus)

Abstract

Background: The WHO classification separates mastocytosis into distinct variants, but prognostication remains a clinical challenge. The aim of this study was to improve prognostication for patients with mastocytosis. Methods: We analysed data of the registry of the European Competence Network on Mastocytosis including 1639 patients (age 17–90 years) diagnosed with mastocytosis according to WHO criteria between Jan 12, 1978, and March 16, 2017. Univariate and multivariate analyses with Cox regression were applied to identify prognostic variables predicting survival outcomes and to establish a prognostic score. We validated this International Prognostic Scoring System in Mastocytosis (IPSM) with data of 462 patients (age 17–79 years) from the Spanish network Red Española de Mastocitosis diagnosed between Jan 22, 1998, and Nov 2, 2017. Findings: The prognostic value of the WHO classification was confirmed in our study (p<0·0001). For patients with non-advanced mastocytosis (n=1380), we identified age 60 years or older (HR 10·75, 95% CI 5·68–20·32) and a concentration of alkaline phosphatase 100 U/L or higher (2·91, 1·60–5·30) as additional independent prognostic variables for overall survival. The resulting scoring system divided patients with non-advanced mastocytosis into three groups: low (no risk factors), intermediate 1 (one risk factor), and intermediate 2 (two risk factors). Overall survival and progression-free survival differed significantly among these groups (p<0·0001). In patients with advanced mastocytosis (n=259), age 60 years or older (HR 2·14, 95% CI 1·42–3·22), a concentration of tryptase 125 ng/mL or higher (1·81, 1·20–2·75), a leukocyte count of 16 × 10⁹ per L or higher (1·88, 1·27–2·79), haemoglobin of 11 g/dL or lower (1·71, 1·13–2·57), a platelet count of 100 × 10⁹ per L or lower (1·63, 1·13–2·34), and skin involvement (0·46, 0·30–0·69) were prognostic variables. Based on these variables, a separate score for advanced mastocytosis with four risk categories was established, with significantly different outcomes for overall survival and progression-free survival (p<0·0001). The prognostic value of both scores was confirmed in 413 patients with non-advanced disease and 49 with advanced mastocytosis from the validation cohort. Interpretation: The IPSM scores for patients with non-advanced and advanced mastocytosis can be used to predict survival outcomes and guide treatment decisions. However, the predictive value of the IPSM needs to be confirmed in forthcoming trials. Funding: Austrian Science Fund, Deutsche Forschungsgemeinschaft, Koeln Fortune Program, Charles and Ann Johnson Foundation, Instituto de Salud Carlos III, Fondos FEDER, Research-Foundation Flanders/Fonds Wetenschappelijk Onderzoek, Clinical Research-Fund of the University Hospitals Leuven, and Research-Foundation Flanders/Fonds Wetenschappelijk Onderzoek.

Original language	English
Journal	The Lancet Haematology
Volume	6
Issue number	12
Pages (from-to)	e638-e649
DOIs	https://doi.org/10.1016/S2352-3026(19)30166-8
Publication status	Published - 12.2019

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10.1016/S2352-3026(19)30166-8

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Sperr, W. R., Kundi, M., Alvarez-Twose, I., van Anrooij, B., Oude Elberink, J. N. G., Gorska, A., Niedoszytko, M., Gleixner, K. V., Hadzijusufovic, E., Zanotti, R., Bonadonna, P., Bonifacio, M., Perkins, C., Illerhaus, A., Elena, C., Merante, S., Shoumariyeh, K., von Bubnoff, N., Parente, R., ... Valent, P. (2019). International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study. The Lancet Haematology, 6(12), e638-e649. https://doi.org/10.1016/S2352-3026(19)30166-8

@article{dd8f148cb93f454ea03324491bcaf9c3,

title = "International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study",

abstract = "Background: The WHO classification separates mastocytosis into distinct variants, but prognostication remains a clinical challenge. The aim of this study was to improve prognostication for patients with mastocytosis. Methods: We analysed data of the registry of the European Competence Network on Mastocytosis including 1639 patients (age 17–90 years) diagnosed with mastocytosis according to WHO criteria between Jan 12, 1978, and March 16, 2017. Univariate and multivariate analyses with Cox regression were applied to identify prognostic variables predicting survival outcomes and to establish a prognostic score. We validated this International Prognostic Scoring System in Mastocytosis (IPSM) with data of 462 patients (age 17–79 years) from the Spanish network Red Espa{\~n}ola de Mastocitosis diagnosed between Jan 22, 1998, and Nov 2, 2017. Findings: The prognostic value of the WHO classification was confirmed in our study (p<0·0001). For patients with non-advanced mastocytosis (n=1380), we identified age 60 years or older (HR 10·75, 95% CI 5·68–20·32) and a concentration of alkaline phosphatase 100 U/L or higher (2·91, 1·60–5·30) as additional independent prognostic variables for overall survival. The resulting scoring system divided patients with non-advanced mastocytosis into three groups: low (no risk factors), intermediate 1 (one risk factor), and intermediate 2 (two risk factors). Overall survival and progression-free survival differed significantly among these groups (p<0·0001). In patients with advanced mastocytosis (n=259), age 60 years or older (HR 2·14, 95% CI 1·42–3·22), a concentration of tryptase 125 ng/mL or higher (1·81, 1·20–2·75), a leukocyte count of 16 × 109 per L or higher (1·88, 1·27–2·79), haemoglobin of 11 g/dL or lower (1·71, 1·13–2·57), a platelet count of 100 × 109 per L or lower (1·63, 1·13–2·34), and skin involvement (0·46, 0·30–0·69) were prognostic variables. Based on these variables, a separate score for advanced mastocytosis with four risk categories was established, with significantly different outcomes for overall survival and progression-free survival (p<0·0001). The prognostic value of both scores was confirmed in 413 patients with non-advanced disease and 49 with advanced mastocytosis from the validation cohort. Interpretation: The IPSM scores for patients with non-advanced and advanced mastocytosis can be used to predict survival outcomes and guide treatment decisions. However, the predictive value of the IPSM needs to be confirmed in forthcoming trials. Funding: Austrian Science Fund, Deutsche Forschungsgemeinschaft, Koeln Fortune Program, Charles and Ann Johnson Foundation, Instituto de Salud Carlos III, Fondos FEDER, Research-Foundation Flanders/Fonds Wetenschappelijk Onderzoek, Clinical Research-Fund of the University Hospitals Leuven, and Research-Foundation Flanders/Fonds Wetenschappelijk Onderzoek.",

author = "Sperr, {Wolfgang R.} and Michael Kundi and Ivan Alvarez-Twose and {van Anrooij}, Bjorn and {Oude Elberink}, {Joanna N.G.} and Aleksandra Gorska and Marek Niedoszytko and Gleixner, {Karoline V.} and Emir Hadzijusufovic and Roberta Zanotti and Patrizia Bonadonna and Massimiliano Bonifacio and Cecelia Perkins and Anja Illerhaus and Chiara Elena and Serena Merante and Khalid Shoumariyeh and {von Bubnoff}, Nikolas and Roberta Parente and Mohamad Jawhar and {Belloni Fortina}, Anna and Francesca Caroppo and Knut Brockow and Alexander Zink and David Fuchs and Kilbertus, {Alex J.} and Yavuz, {Akif Selim} and Michael Doubek and Hans H{\"a}gglund and Jens Panse and Vito Sabato and Agnes Bretterklieber and Dietger Niederwieser and Christine Breynaert and Karin Hartmann and Massimo Triggiani and Boguslaw Nedoszytko and Andreas Reiter and Alberto Orfao and Olivier Hermine and Jason Gotlib and Michel Arock and Kluin-Nelemans, {Hanneke C.} and Peter Valent",

note = "Publisher Copyright: {\textcopyright} 2019 Elsevier Ltd",

year = "2019",

month = dec,

doi = "10.1016/S2352-3026(19)30166-8",

language = "English",

volume = "6",

pages = "e638--e649",

journal = "The Lancet Haematology",

issn = "2352-3026",

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Sperr, WR, Kundi, M, Alvarez-Twose, I, van Anrooij, B, Oude Elberink, JNG, Gorska, A, Niedoszytko, M, Gleixner, KV, Hadzijusufovic, E, Zanotti, R, Bonadonna, P, Bonifacio, M, Perkins, C, Illerhaus, A, Elena, C, Merante, S, Shoumariyeh, K, von Bubnoff, N, Parente, R, Jawhar, M, Belloni Fortina, A, Caroppo, F, Brockow, K, Zink, A, Fuchs, D, Kilbertus, AJ, Yavuz, AS, Doubek, M, Hägglund, H, Panse, J, Sabato, V, Bretterklieber, A, Niederwieser, D, Breynaert, C, Hartmann, K, Triggiani, M, Nedoszytko, B, Reiter, A, Orfao, A, Hermine, O, Gotlib, J, Arock, M, Kluin-Nelemans, HC & Valent, P 2019, 'International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study', The Lancet Haematology, vol. 6, no. 12, pp. e638-e649. https://doi.org/10.1016/S2352-3026(19)30166-8

TY - JOUR

T1 - International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study

AU - Sperr, Wolfgang R.

AU - Kundi, Michael

AU - Alvarez-Twose, Ivan

AU - van Anrooij, Bjorn

AU - Oude Elberink, Joanna N.G.

AU - Gorska, Aleksandra

AU - Niedoszytko, Marek

AU - Gleixner, Karoline V.

AU - Hadzijusufovic, Emir

AU - Zanotti, Roberta

AU - Bonadonna, Patrizia

AU - Bonifacio, Massimiliano

AU - Perkins, Cecelia

AU - Illerhaus, Anja

AU - Elena, Chiara

AU - Merante, Serena

AU - Shoumariyeh, Khalid

AU - von Bubnoff, Nikolas

AU - Parente, Roberta

AU - Jawhar, Mohamad

AU - Belloni Fortina, Anna

AU - Caroppo, Francesca

AU - Brockow, Knut

AU - Zink, Alexander

AU - Fuchs, David

AU - Kilbertus, Alex J.

AU - Yavuz, Akif Selim

AU - Doubek, Michael

AU - Hägglund, Hans

AU - Panse, Jens

AU - Sabato, Vito

AU - Bretterklieber, Agnes

AU - Niederwieser, Dietger

AU - Breynaert, Christine

AU - Hartmann, Karin

AU - Triggiani, Massimo

AU - Nedoszytko, Boguslaw

AU - Reiter, Andreas

AU - Orfao, Alberto

AU - Hermine, Olivier

AU - Gotlib, Jason

AU - Arock, Michel

AU - Kluin-Nelemans, Hanneke C.

AU - Valent, Peter

PY - 2019/12

Y1 - 2019/12

N2 - Background: The WHO classification separates mastocytosis into distinct variants, but prognostication remains a clinical challenge. The aim of this study was to improve prognostication for patients with mastocytosis. Methods: We analysed data of the registry of the European Competence Network on Mastocytosis including 1639 patients (age 17–90 years) diagnosed with mastocytosis according to WHO criteria between Jan 12, 1978, and March 16, 2017. Univariate and multivariate analyses with Cox regression were applied to identify prognostic variables predicting survival outcomes and to establish a prognostic score. We validated this International Prognostic Scoring System in Mastocytosis (IPSM) with data of 462 patients (age 17–79 years) from the Spanish network Red Española de Mastocitosis diagnosed between Jan 22, 1998, and Nov 2, 2017. Findings: The prognostic value of the WHO classification was confirmed in our study (p<0·0001). For patients with non-advanced mastocytosis (n=1380), we identified age 60 years or older (HR 10·75, 95% CI 5·68–20·32) and a concentration of alkaline phosphatase 100 U/L or higher (2·91, 1·60–5·30) as additional independent prognostic variables for overall survival. The resulting scoring system divided patients with non-advanced mastocytosis into three groups: low (no risk factors), intermediate 1 (one risk factor), and intermediate 2 (two risk factors). Overall survival and progression-free survival differed significantly among these groups (p<0·0001). In patients with advanced mastocytosis (n=259), age 60 years or older (HR 2·14, 95% CI 1·42–3·22), a concentration of tryptase 125 ng/mL or higher (1·81, 1·20–2·75), a leukocyte count of 16 × 109 per L or higher (1·88, 1·27–2·79), haemoglobin of 11 g/dL or lower (1·71, 1·13–2·57), a platelet count of 100 × 109 per L or lower (1·63, 1·13–2·34), and skin involvement (0·46, 0·30–0·69) were prognostic variables. Based on these variables, a separate score for advanced mastocytosis with four risk categories was established, with significantly different outcomes for overall survival and progression-free survival (p<0·0001). The prognostic value of both scores was confirmed in 413 patients with non-advanced disease and 49 with advanced mastocytosis from the validation cohort. Interpretation: The IPSM scores for patients with non-advanced and advanced mastocytosis can be used to predict survival outcomes and guide treatment decisions. However, the predictive value of the IPSM needs to be confirmed in forthcoming trials. Funding: Austrian Science Fund, Deutsche Forschungsgemeinschaft, Koeln Fortune Program, Charles and Ann Johnson Foundation, Instituto de Salud Carlos III, Fondos FEDER, Research-Foundation Flanders/Fonds Wetenschappelijk Onderzoek, Clinical Research-Fund of the University Hospitals Leuven, and Research-Foundation Flanders/Fonds Wetenschappelijk Onderzoek.

AB - Background: The WHO classification separates mastocytosis into distinct variants, but prognostication remains a clinical challenge. The aim of this study was to improve prognostication for patients with mastocytosis. Methods: We analysed data of the registry of the European Competence Network on Mastocytosis including 1639 patients (age 17–90 years) diagnosed with mastocytosis according to WHO criteria between Jan 12, 1978, and March 16, 2017. Univariate and multivariate analyses with Cox regression were applied to identify prognostic variables predicting survival outcomes and to establish a prognostic score. We validated this International Prognostic Scoring System in Mastocytosis (IPSM) with data of 462 patients (age 17–79 years) from the Spanish network Red Española de Mastocitosis diagnosed between Jan 22, 1998, and Nov 2, 2017. Findings: The prognostic value of the WHO classification was confirmed in our study (p<0·0001). For patients with non-advanced mastocytosis (n=1380), we identified age 60 years or older (HR 10·75, 95% CI 5·68–20·32) and a concentration of alkaline phosphatase 100 U/L or higher (2·91, 1·60–5·30) as additional independent prognostic variables for overall survival. The resulting scoring system divided patients with non-advanced mastocytosis into three groups: low (no risk factors), intermediate 1 (one risk factor), and intermediate 2 (two risk factors). Overall survival and progression-free survival differed significantly among these groups (p<0·0001). In patients with advanced mastocytosis (n=259), age 60 years or older (HR 2·14, 95% CI 1·42–3·22), a concentration of tryptase 125 ng/mL or higher (1·81, 1·20–2·75), a leukocyte count of 16 × 109 per L or higher (1·88, 1·27–2·79), haemoglobin of 11 g/dL or lower (1·71, 1·13–2·57), a platelet count of 100 × 109 per L or lower (1·63, 1·13–2·34), and skin involvement (0·46, 0·30–0·69) were prognostic variables. Based on these variables, a separate score for advanced mastocytosis with four risk categories was established, with significantly different outcomes for overall survival and progression-free survival (p<0·0001). The prognostic value of both scores was confirmed in 413 patients with non-advanced disease and 49 with advanced mastocytosis from the validation cohort. Interpretation: The IPSM scores for patients with non-advanced and advanced mastocytosis can be used to predict survival outcomes and guide treatment decisions. However, the predictive value of the IPSM needs to be confirmed in forthcoming trials. Funding: Austrian Science Fund, Deutsche Forschungsgemeinschaft, Koeln Fortune Program, Charles and Ann Johnson Foundation, Instituto de Salud Carlos III, Fondos FEDER, Research-Foundation Flanders/Fonds Wetenschappelijk Onderzoek, Clinical Research-Fund of the University Hospitals Leuven, and Research-Foundation Flanders/Fonds Wetenschappelijk Onderzoek.

UR - http://www.scopus.com/inward/record.url?scp=85075358050&partnerID=8YFLogxK

U2 - 10.1016/S2352-3026(19)30166-8

DO - 10.1016/S2352-3026(19)30166-8

M3 - Journal articles

C2 - 31676322

AN - SCOPUS:85075358050

SN - 2352-3026

VL - 6

SP - e638-e649

JO - The Lancet Haematology

JF - The Lancet Haematology

IS - 12

ER -

International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study

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