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MARS: mutation-adjusted risk score for advanced systemic mastocytosis

PURPOSE: To develop a risk score for patients with advanced systemic mastocytosis (AdvSM) that integrates clinical and mutation characteristics. PATIENTS AND METHODS: The study included 383 patients with AdvSM from the German Registry on Disorders of Eosinophils and Mast Cells (training set; n = 231...

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Main Authors: Jawhar, Mohamad (Author) , Schwaab, Juliana (Author) , Álvarez-Twose, Iván (Author) , Shoumariyeh, Khalid (Author) , Naumann, Nicole (Author) , Lübke, Johannes (Author) , Perkins, Cecelia (Author) , Muñoz-Gonźalez, Javier Ignacio (Author) , Meggendorfer, Manja (Author) , Kennedy, Vanessa (Author) , Metzgeroth, Georgia (Author) , Fabarius, Alice (Author) , Pfeifer, Dietmar (Author) , Sotlar, Karl (Author) , Horny, Hans-Peter (Author) , Bubnoff, Nikolas von (Author) , Haferlach, Torsten (Author) , Cross, Nicholas C. P. (Author) , Hofmann, Wolf-Karsten (Author) , Sperr, Wolfgang Reinhard (Author) , García-Montero, Andrés C. (Author) , Valent, Peter (Author) , Gotlib, Jason (Author) , Orfao, Alberto (Author) , Reiter, Andreas (Author)
Format: Article (Journal)
Language:English
Published: September 11, 2019
In: Journal of clinical oncology
Year: 2019, Volume: 37, Issue: 31, Pages: 2846-2856
ISSN:1527-7755
DOI:10.1200/JCO.19.00640
Online Access:Verlag, Volltext: https://doi.org/10.1200/JCO.19.00640
Verlag: https://ascopubs.org/doi/full/10.1200/JCO.19.00640
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Author Notes:Mohamad Jawhar, Juliana Schwaab, Iván Álvarez-Twose, Khalid Shoumariyeh, Nicole Naumann, Johannes Lübke, Cecelia Perkins, Javier I. Muñoz-González, Manja Meggendorfer, Vanessa Kennedy, Georgia Metzgeroth, Alice Fabarius, Dietmar Pfeifer, Karl Sotlar, Hans-Peter Horny, Nikolas von Bubnoff, Torsten Haferlach, Nicholas C.P. Cross, Wolf-Karsten Hofmann, Wolfgang R. Sperr, Andrés C. García-Montero, Peter Valent, Jason Gotlib, Alberto Orfao, Andreas Reiter
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Summary:PURPOSE: To develop a risk score for patients with advanced systemic mastocytosis (AdvSM) that integrates clinical and mutation characteristics. PATIENTS AND METHODS: The study included 383 patients with AdvSM from the German Registry on Disorders of Eosinophils and Mast Cells (training set; n = 231) and several centers for mastocytosis in the United States and Europe, all within the European Competence Network on Mastocytosis (validation set; n = 152). A Cox multivariable model was used to select variables that were predictive of overall survival (OS). RESULTS: In multivariable analysis, the following risk factors were identified as being associated with OS: age greater than 60 years, anemia (hemoglobin < 10 g/dL), thrombocytopenia (platelets < 100 × 109/L), presence of one high molecular risk gene mutation (ie, in SRSF2, ASXL1, and/or RUNX1), and presence of two or more high molecular risk gene mutations. By assigning hazard ratio-weighted points to these variables, the following three risk categories were defined: low risk (median OS, not reached), intermediate risk (median OS, 3.9 years; 95% CI, 2.1 to 5.7 years), and high risk (median OS, 1.9 years; 95% CI, 1.3 to 2.6 years; P < .001). The mutation-adjusted risk score (MARS) was independent of the WHO classification and was confirmed in the independent validation set. During a median follow-up time of 2.2 years (range, 0 to 23 years), 63 (16%) of 383 patients experienced a leukemic transformation to secondary mast cell leukemia (32%) or secondary acute myeloid leukemia (68%). The MARS was also predictive for leukemia-free survival (P < .001).CONCLUSIONThe MARS is a validated, five-parameter, WHO-independent prognostic score that defines three risk groups among patients with AdvSM and may improve up-front treatment stratification for these rare hematologic neoplasms.
Item Description:Gesehen am 02.03.2020
Physical Description:Online Resource
ISSN:1527-7755
DOI:10.1200/JCO.19.00640

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