Comparison of prognostic scores according to <scp>WHO</scp> classification in 170 patients with advanced mastocytosis and C‐finding treated with midostaurin

Advanced systemic mastocytosis (AdvSM) encompasses heterogeneous mastocytosis subtypes and is associated with poor outcomes. Although midostaurin was the first tyrosine kinase inhibitor to be approved for AdvSM patients, long-lasting responses are limited. The mutation-Adjusted Risk Score (MARS), the International Prognostic Scoring System for mastocytosis (IPSM) and the Global Prognostic Score for Systemic Mastocytosis (GPSM) have been established to characterize the outcomes of patients with overall AdvSM. However, given the outcome's dependency on the AdvSM subtype, prognostic characterization within each subtype is critical. We aimed to study the predictive ability using Harrell's concordance index of prognostic scores according to the AdvSM subtype. We conducted a nationwide retrospective study using the French mastocytosis reference center's registry and included all midostaurin-treated patients with C finding. Overall, 170 patients were identified: 46 aggressive SM (ASM), 11 mast cell leukemia (MCL), and 113 SM with associated hematological neoplasm (SM-AHN). All risk scores improved their discriminative value for overall survival (OS) when combined with the AdvSM subtype. The best predictive value was for adjusted MARS (C-index = 0.689), followed by GPSM (C-index = 0.677) and IPSM (C-index = 0.618). In a multivariable analysis, MARS stratification and the AdvSM subtype were both prognostic for OS. Accordingly, five subgroups of patients with AdvSM and a different median OS were identified: 9.9 months for MCL, 24 months for intermediate/high-risk SM-AHN, 33 months for intermediate/high-risk ASM, 58 months for low-risk SM-AHN and was not reached for low-risk ASM (p < 0.001). The AdvSM subtype and the MARS are the most predictive of OS and should prompt specific management.

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Journal
American Journal of Hematology
Published
2024-09-17
DOI
https://doi.org/10.1002/ajh.27478
Citations
2
Primary Topic
Mast cells and histamine
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article
Field-Weighted Citation Impact
0.48
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article

Comparison of prognostic scores according to WHO classification in 170 patients with advanced mastocytosis and C‐finding treated with midostaurin

Marie Templé, Ewa Wierzbicka‐Hainaut, F. Pelletier, Maël Heiblig et al.
2 citations
American Journal of Hematology
Mast cells and histamine
0.48
article

Comparison of prognostic scores according to WHO classification in 170 patients with advanced mastocytosis and C‐finding treated with midostaurin

Marie Templé, Ewa Wierzbicka‐Hainaut, F. Pelletier, Maël Heiblig, C. Hoarau, Patrick Villarèse, Thomas Ballul, I. Durieu, David Launay, Julie Agopian, F. Rétornaz, Sophie Dimicoli‐Salazar, Olivier Kosmider, Céline Greco, Laura Polivka, Florence Castelain, Julie Bruneau, Rose‐Marie Javier, C. Châtain, Hassiba Bouktit, A. Soria, Caroline Gaudy‐Marqueste, J. Lespinasse, Edwige Le Mouel, Michel Arock, Laurence Bouillet, Mathieu Wémeau, R. Jaussaud, Olivier Tournilhac, Marie Gousseff, Louis Terriou, Jean‐François Viallard, Patrice Dubreuil, Quentin Cabrera, C. Méni, Olivier Lortholary, Richard Lemal, Christian Lavigne, Fabienne Brenet, Gandhi Laurent Damaj, Jose Miguel Torregrosa‐Diaz, Olivier Hermine, Philippe Guilpain, Ludovic Lhermitte, Julien Rossignol, Patricia Zunic, Dana Ranta, C. Gourguechon, Thierry Jo Molina, Yannick Chantran, Laurent Frenzel, Christine Bodemer, Dina Benabou, Antoine Neel, Marie Pierre Gourin, Stéphane Barete
article en
2 citations

Abstract

Advanced systemic mastocytosis (AdvSM) encompasses heterogeneous mastocytosis subtypes and is associated with poor outcomes. Although midostaurin was the first tyrosine kinase inhibitor to be approved for AdvSM patients, long-lasting responses are limited. The mutation-Adjusted Risk Score (MARS), the International Prognostic Scoring System for mastocytosis (IPSM) and the Global Prognostic Score for Systemic Mastocytosis (GPSM) have been established to characterize the outcomes of patients with overall AdvSM. However, given the outcome's dependency on the AdvSM subtype, prognostic characterization within each subtype is critical. We aimed to study the predictive ability using Harrell's concordance index of prognostic scores according to the AdvSM subtype. We conducted a nationwide retrospective study using the French mastocytosis reference center's registry and included all midostaurin-treated patients with C finding. Overall, 170 patients were identified: 46 aggressive SM (ASM), 11 mast cell leukemia (MCL), and 113 SM with associated hematological neoplasm (SM-AHN). All risk scores improved their discriminative value for overall survival (OS) when combined with the AdvSM subtype. The best predictive value was for adjusted MARS (C-index = 0.689), followed by GPSM (C-index = 0.677) and IPSM (C-index = 0.618). In a multivariable analysis, MARS stratification and the AdvSM subtype were both prognostic for OS. Accordingly, five subgroups of patients with AdvSM and a different median OS were identified: 9.9 months for MCL, 24 months for intermediate/high-risk SM-AHN, 33 months for intermediate/high-risk ASM, 58 months for low-risk SM-AHN and was not reached for low-risk ASM (p < 0.001). The AdvSM subtype and the MARS are the most predictive of OS and should prompt specific management.

American Journal of HematologyVol. 99(11)
Université Claude Bernard Lyon 1 (FR), Institut polytechnique de Grenoble (FR), Centre National de la Recherche Scientifique (FR), Inserm (FR), Université Clermont Auvergne (FR), Université de Montpellier (FR), Université Paris Cité (FR), Aix-Marseille Université (FR), Centre Hospitalier Universitaire de Grenoble (FR), Centre Hospitalier Universitaire de Bordeaux (FR), Centre Hospitalier Universitaire de Lille (FR), Centre Hospitalier Universitaire de Toulouse (FR), Sorbonne Université (FR), Institut Necker Enfants Malades (FR), Centre Hospitalier de Roubaix (FR), Centre Hospitalier Universitaire de Besançon (FR), Institut National de Recherche pour l'Agriculture, l'Alimentation et l'Environnement (FR), Sorbonne Paris Cité (FR), Hôpital Cochin (FR), Centre Hospitalier Universitaire de Montpellier (FR), Centre Hospitalier Universitaire de Tours (FR), Centre Hospitalier Universitaire Amiens-Picardie (FR), Assistance Publique – Hôpitaux de Paris (FR), Centre Hospitalier Régional et Universitaire de Nancy (FR), Association Francaise contre les Myopathies (FR), Hôpital Saint-Antoine (FR), VetAgro Sup (FR), Délégation Centre-Est (FR), Centre hospitalier Bretagne Atlantique (FR), Hôpital Lyon Sud (FR), Hôpital Cardiologique du Haut-Lévêque (FR), Pitié-Salpêtrière Hospital (FR), Lille Inflammation Research International Center (FR), Centre de Recherche Saint-Antoine (FR), Institut Cochin (FR), Centre de Recherche en Cancérologie de Marseille (FR), Centre Hospitalier Universitaire de Rennes (FR), Hôpital Dupuytren (FR), Institut des Maladies Génétiques Imagine (FR), Hôpital Tenon (FR), Institute for Translational Research in Inflammation (FR), Centre de Recherche Épidémiologie et Statistique (FR), Université d'Angers (FR), Université de Strasbourg (FR), Université Grenoble Alpes (FR), Université de Franche-Comté (FR)
Reduced inequalities
Openalex Percentile: Top 35%
Mast cells and histamine
0.48
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