Comparison of prognostic scores according to WHO classification in 170 patients with advanced mastocytosis and C-finding treated with midostaurin.
Journal
American journal of hematology
ISSN: 1096-8652
Titre abrégé: Am J Hematol
Pays: United States
ID NLM: 7610369
Informations de publication
Date de publication:
17 Sep 2024
17 Sep 2024
Historique:
revised:
05
08
2024
received:
05
05
2024
accepted:
06
08
2024
medline:
17
9
2024
pubmed:
17
9
2024
entrez:
17
9
2024
Statut:
aheadofprint
Résumé
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.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2024 The Author(s). American Journal of Hematology published by Wiley Periodicals LLC.
Références
Valent P, Akin C, Hartmann K, et al. Advances in the classification and treatment of Mastocytosis: current status and outlook toward the future. Cancer Res. 2017;77(6):1261‐1270. doi:10.1158/0008‐5472.CAN‐16‐2234
Pardanani A. Systemic mastocytosis in adults: 2023 update on diagnosis, risk stratification and management. Am J Hematol. 2023;98(7):1097‐1116. doi:10.1002/ajh.26962
Arber DA, Orazi A, Hasserjian RP, et al. International consensus classification of myeloid neoplasms and acute leukemias: integrating morphologic, clinical, and genomic data. Blood. 2022;140(11):1200‐1228. doi:10.1182/blood.2022015850
Khoury JD, Solary E, Abla O, et al. The 5th edition of the World Health Organization classification of Haematolymphoid Tumours: myeloid and histiocytic/dendritic neoplasms. Leukemia. 2022;36(7):1703‐1719. doi:10.1038/s41375‐022‐01613‐1
Pardanani A, Reeder TL, Kimlinger TK, et al. Flt‐3 and c‐kit mutation studies in a spectrum of chronic myeloid disorders including systemic mast cell disease. Leuk Res. 2003;27(8):739‐742. doi:10.1016/s0145‐2126(02)00303‐x
Taylor ML, Sehgal D, Raffeld M, et al. Demonstration that mast cells, T cells, and B cells bearing the activating kit mutation D816V occur in clusters within the marrow of patients with Mastocytosis. J Mol Diagn. 2004;6(4):335‐342.
Dunphy CH. Evaluation of mast cells in myeloproliferative disorders and myelodysplastic syndromes. Arch Pathol Lab Med. 2005;129(2):219‐222. doi:10.5858/2005‐129‐219‐EOMCIM
Damaj G, Joris M, Chandesris O, et al. ASXL1 but not TET2 mutations adversely impact overall survival of patients suffering systemic mastocytosis with associated clonal hematologic non‐mast‐cell diseases. PLoS ONE. 2014;9(1):e85362. doi:10.1371/journal.pone.0085362
Wang SA, Hutchinson L, Tang G, et al. Systemic mastocytosis with associated clonal hematological non‐mast cell lineage disease: clinical significance and comparison of chomosomal abnormalities in SM and AHNMD components. Am J Hematol. 2013;88(3):219‐224. doi:10.1002/ajh.23380
Jawhar M, Schwaab J, Schnittger S, et al. Additional mutations in SRSF2, ASXL1 and/or RUNX1 identify a high‐risk group of patients with KIT D816V(+) advanced systemic mastocytosis. Leukemia. 2016;30(1):136‐143. doi:10.1038/leu.2015.284
Schwaab J, Schnittger S, Sotlar K, et al. Comprehensive mutational profiling in advanced systemic mastocytosis. Blood. 2013;122(14):2460‐2466. doi:10.1182/blood‐2013‐04‐496448
Barete S, Lortholary O, Damaj G, et al. Long‐term efficacy and safety of cladribine (2‐CdA) in adult patients with mastocytosis. Blood. 2015;126(8):1009‐1016; quiz 1050. doi:10.1182/blood‐2014‐12‐614743
Lim KH, Pardanani A, Butterfield JH, Li CY, Tefferi A. Cytoreductive therapy in 108 adults with systemic mastocytosis: outcome analysis and response prediction during treatment with interferon‐alpha, hydroxyurea, imatinib mesylate or 2‐chlorodeoxyadenosine. Am J Hematol. 2009;84(12):790‐794. doi:10.1002/ajh.21561
Tefferi A, Kittur J, Farrukh F, et al. Cladribine therapy for advanced and indolent systemic mastocytosis: Mayo Clinic experience in 42 consecutive cases. Br J Haematol. 2022;196(4):975‐983. doi:10.1111/bjh.17932
Kluin‐Nelemans HC, Oldhoff JM, Van Doormaal JJ, et al. Cladribine therapy for systemic mastocytosis. Blood. 2003;102(13):4270‐4276. doi:10.1182/blood‐2003‐05‐1699
Lübke J, Naumann N, Metzgeroth G, et al. Response and resistance to cladribine in patients with advanced systemic mastocytosis: a registry‐based analysis. Ann Hematol. 2023;102(8):2077‐2085. doi:10.1007/s00277‐023‐05180‐y
Chandesris MO, Damaj G, Canioni D, et al. Midostaurin in advanced systemic Mastocytosis. N Engl J Med. 2016;374(26):2605‐2607. doi:10.1056/NEJMc1515403
DeAngelo DJ, George TI, Linder A, et al. Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10‐year median follow‐up of a phase II trial. Leukemia. 2018;32(2):470‐478. doi:10.1038/leu.2017.234
Gotlib J, Kluin‐Nelemans HC, George TI, et al. Efficacy and safety of Midostaurin in advanced systemic Mastocytosis. N Engl J Med. 2016;374(26):2530‐2541. doi:10.1056/NEJMoa1513098
Singh A, Al‐Kali A, Begna KH, et al. Midostaurin therapy for advanced systemic mastocytosis: Mayo Clinic experience in 33 consecutive cases. Am J Hematol. 2022;97(5):630‐637. doi:10.1002/ajh.26498
Reiter A, Schwaab J, DeAngelo DJ, et al. Efficacy and safety of avapritinib in previously treated patients with advanced systemic mastocytosis. Blood Adv. 2022;6(21):5750‐5762. doi:10.1182/bloodadvances.2022007539
Reiter A, Gotlib J, Álvarez‐Twose I, et al. Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis. Leukemia. 2022;36(8):2108‐2120. doi:10.1038/s41375‐022‐01615‐z
Gotlib J, Reiter A, Radia DH, et al. Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial. Nat Med. 2021;27(12):2192‐2199. doi:10.1038/s41591‐021‐01539‐8
DeAngelo DJ, Radia DH, George TI, et al. Safety and efficacy of avapritinib in advanced systemic mastocytosis: the phase 1 EXPLORER trial. Nat Med. 2021;27(12):2183‐2191. doi:10.1038/s41591‐021‐01538‐9
Sperr WR, Kundi M, Alvarez‐Twose I, et al. International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study. Lancet Haematol. 2019;6(12):e638‐e649. doi:10.1016/S2352‐3026(19)30166‐8
Jawhar M, Schwaab J, Álvarez‐Twose I, et al. MARS: mutation‐adjusted risk score for advanced systemic Mastocytosis. J Clin Oncol. 2019;37(31):2846‐2856. doi:10.1200/JCO.19.00640
Valent P, Akin C, Hartmann K, et al. Updated diagnostic criteria and classification of mast cell disorders: a consensus proposal. Hema. 2021;5(11):e646. doi:10.1097/HS9.0000000000000646
Valent P, Horny HP, Escribano L, et al. Diagnostic criteria and classification of mastocytosis: a consensus proposal. Leuk Res. 2001;25(7):603‐625. doi:10.1016/s0145‐2126(01)00038‐8
Muñoz‐González JI, Álvarez‐Twose I, Jara‐Acevedo M, et al. Proposed global prognostic score for systemic mastocytosis: a retrospective prognostic modelling study. Lancet Haematol. 2021;8(3):e194‐e204. doi:10.1016/S2352‐3026(20)30400‐2
Harrell FE Jr, Lee KL, Mark DB. Multivariable prognostic models: issues in developing models, evaluating assumptions and adequacy, and measuring and reducing errors. Stat Med. 1996;15(4):361‐387. doi:10.1002/(SICI)1097‐0258(19960229)15:4<361::AID‐SIM168>3.0.CO;2‐4
R Core Team. R: A Language and Environment for Statistical Computing. R Foundation for Statistical Computing; 2013. https://www.r-project.org/.
Lübke J, Schwaab J, Naumann N, et al. Superior efficacy of Midostaurin over cladribine in advanced systemic Mastocytosis: a registry‐based analysis. J Clin Oncol. 2022;40(16):1783‐1794. doi:10.1200/JCO.21.01849
McLornan DP, Czerw T, Damaj G, et al. Allogeneic haematopoietic cell transplantation for advanced systemic mastocytosis: best practice recommendations on behalf of the EBMT practice harmonisation and guidelines committee. Leukemia. 2024;38(4):699‐711. doi:10.1038/s41375‐024‐02182‐1
Pardanani A, Lim KH, Lasho TL, et al. Prognostically relevant breakdown of 123 patients with systemic mastocytosis associated with other myeloid malignancies. Blood. 2009;114(18):3769‐3772. doi:10.1182/blood‐2009‐05‐220145
Ustun C, Gotlib J, Popat U, et al. Consensus opinion on allogeneic hematopoietic cell transplantation in advanced systemic Mastocytosis. Biol Blood Marrow Transplant. 2016;22(8):1348‐1356. doi:10.1016/j.bbmt.2016.04.018
Ustun C, Reiter A, Scott BL, et al. Hematopoietic stem‐cell transplantation for advanced systemic Mastocytosis. J Clin Oncol. 2014;32(29):3264‐3274. doi:10.1200/JCO.2014.55.2018
Lübke J, Christen D, Schwaab J, et al. Allogeneic hematopoietic cell transplantation in advanced systemic Mastocytosis: a retrospective analysis of the DRST and GREM registries. Leukemia. 2024;38(4):810‐821. doi:10.1038/s41375‐024‐02186‐x