Comparison of prognostic scores according to WHO classification in 170 patients with advanced mastocytosis and C‐finding treated with midostaurin
2 HCL - Hospices Civils de Lyon
3 UNICANCER/CRCL - Centre de Recherche en Cancérologie de Lyon
4 CIRI-LIB - Immuno-Biologie des Lymphomes [CIRI]
5 CHU Amiens-Picardie
6 Hôpital Saint Eloi [CHU Montpellier]
7 UM - Université de Montpellier
8 CHU Toulouse - Centre Hospitalier Universitaire de Toulouse
9 SU - Sorbonne Université
10 CHU Saint-Antoine [AP-HP]
11 CRESS (U1153 / UMR_A 1125) - Centre for Research in Epidemiology and Statistics | Centre de Recherche Épidémiologie et Statistiques
12 CHU Pitié-Salpêtrière [AP-HP]
13 CRCM - Centre de Recherche en Cancérologie de Marseille
14 AMU - Aix Marseille Université
15 BPH - Bordeaux population health
16 UCA - Université Clermont Auvergne
17 CHELTER - Role of intra-Clonal Heterogeneity and Leukemic environment in ThErapy Resistance of chronic leukemias
18 CHU Clermont-Ferrand
19 CHU Estaing [Clermont-Ferrand]
20 CIC 1405 - Centre d'Investigation Clinique [CHU Clermont-Ferrand]
21 CHRU Lille - Centre Hospitalier Régional Universitaire [CHU Lille]
22 Centre de référence des maladies auto-immunes systémiques rares d'Île-de-France / National Reference Center for Rare Systemic Autoimmune Diseases
23 UGA UFRM - Université Grenoble Alpes - UFR Médecine
24 TIMC-T-RAIG - Translational Research in Autoimmunity and Inflammation Group
25 TIMC - Translational Innovation in Medicine and Complexity / Recherche Translationnelle et Innovation en Médecine et Complexité - UMR 5525
26 CHU Caen
27 Imagine - U1163 - Imagine - Institut des maladies génétiques (IHU)
28 Hôpital Necker - Enfants Malades [AP-HP]
29 iPSC - Plateforme IPS = Imagine Institute iPSC core facility
30 CEREMAST - Centre de référence des mastocytoses
31 AMU SMPM MED - Aix-Marseille Université - École de médecine
32 APHM - Assistance Publique - Hôpitaux de Marseille
33 CHBA - Centre hospitalier Bretagne Atlantique (Morbihan)
34 Centre Hospitalier Universitaire de Rennes [CHU Rennes] = Rennes University Hospital [Pontchaillou]
35 CHU Nantes - Centre Hospitalier Universitaire de Nantes = Nantes University Hospital
36 Service d'Hématologie [CHRU Nancy]
37 Internal Medicine and Clinical Immunology, Vandoeuvre-lès-Nancy, France
38 Département de Pathologie [CHU Necker]
39 Service d'hématologie-oncologie adultes
40 IC UM3 (UMR 8104 / U1016) - Institut Cochin
41 CHU Strasbourg - Centre Hospitalier Universitaire [Strasbourg]
42 CEREMAST - Centre de référence des mastocytoses de Marseille
43 CHRU Besançon - Centre Hospitalier Régional Universitaire de Besançon
44 RIGHT - Interactions hôte-greffon-tumeur, ingénierie cellulaire et génique - UFC (UMR INSERM 1098)
45 Hôpital Européen [Fondation Ambroise Paré - Marseille]
46 CHU Sud Saint Pierre [Ile de la Réunion]
47 Hôpital Dupuytren [CHU Limoges]
48 CHU de Poitiers [La Milétrie] - Centre hospitalier universitaire de Poitiers = Poitiers University Hospital
49 CHU Angers - Centre Hospitalier Universitaire d'Angers
50 CHU Trousseau [Tours]
51 CHLS - Centre Hospitalier Lyon Sud [CHU - HCL]
52 RESHAPE - Inserm U1290 - UCBL1 - Research on Healthcare Performance
53 CHU de Bordeaux Pellegrin [Bordeaux]
54 Centre Hospitalier de Roubaix
55 CHU Tenon [AP-HP]
56 MAGEC - Centre de référence national des Maladies Génétiques à Expression Cutanée - National Reference Center for Genodermatoses and Rare Skin Diseases
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Résumé
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.
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