Title :

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

Author(s) :

Heiblig, M. [Auteur]
Gourguechon, C. [Auteur]
Guilpain, P. [Auteur]
Bulai-Livideanu, C. [Auteur]
Barete, S. [Auteur]
Chantran, Y. [Auteur]
Agopian, J. [Auteur]
Brenet, F. [Auteur]
Dubreuil, P. [Auteur]
Lespinasse, J. [Auteur]
Lemal, R. [Auteur]
Tournilhac, O. [Auteur]
Terriou, Louis [Auteur]
Institute for Translational Research in Inflammation - U 1286 [INFINITE]
Launay, David [Auteur]
Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286
Bouillet, L. [Auteur]
Chatain, C. [Auteur]
Damaj, G. [Auteur]
Ballul, T. [Auteur]
Greco, C. [Auteur]
Polivka, L. [Auteur]
Frenzel, L. [Auteur]
Meni, C. [Auteur]
Bouktit, H. [Auteur]
Benabou, D. [Auteur]
Gaudy-Marqueste, C. [Auteur]
Gousseff, M. [Auteur]
Le Mouel, E. [Auteur]
Neel, A. [Auteur]
Ranta, D. [Auteur]
Jaussaud, R. [Auteur]
Molina, T. J. [Auteur]
Bruneau, J. [Auteur]
Villarese, P. [Auteur]
Lhermitte, L. [Auteur]
Maouche-Chrétien, L. [Auteur]
Temple, M. [Auteur]
Kosmider, O. [Auteur]
Javier, R. M. [Auteur]
Pelletier, F. [Auteur]
Castelain, F. [Auteur]
Retornaz, F. [Auteur]
Cabrera, Q. [Auteur]
Zunic, P. [Auteur]
Gourin, M. P. [Auteur]
Wierzbicka-Hainaut, E. [Auteur]
Viallard, J. F. [Auteur]
Lavigne, C. [Auteur]
Hoarau, C. [Auteur]
Durieu, I. [Auteur]
Dimicoli-Salazar, S. [Auteur]
Torregrosa-Diaz, J. M. [Auteur]
Wemeau, M. [Auteur]
Soria, A. [Auteur]
Arock, M. [Auteur]
Bodemer, C. [Auteur]
Lortholary, O. [Auteur]
Hermine, O. [Auteur]
Rossignol, Julien [Auteur]
Hôpital Necker - Enfants Malades [AP-HP]

Journal title :

Am J Hematol

Abbreviated title :

Am J Hematol

Volume number :

99

Pages :

2127-2139

Publisher :

Wiley Online Library

Publication date :

2024-10-14

ISSN :

1096-8652

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

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, ...
Show more >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.Show less >

Language :

Anglais

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Submission date :

2024-10-26T21:02:21Z
2024-11-06T09:03:48Z