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Verfasst von:Lübke, Johannes [VerfasserIn]   i
 Schwaab, Juliana [VerfasserIn]   i
 Naumann, Nicole [VerfasserIn]   i
 Horny, Hans-Peter [VerfasserIn]   i
 Weiß, Christel [VerfasserIn]   i
 Metzgeroth, Georgia [VerfasserIn]   i
 Kreil, Sebastian [VerfasserIn]   i
 Cross, Nicholas C.P. [VerfasserIn]   i
 Sotlar, Karl [VerfasserIn]   i
 Fabarius, Alice [VerfasserIn]   i
 Hofmann, Wolf-Karsten [VerfasserIn]   i
 Valent, Peter [VerfasserIn]   i
 Gotlib, Jason [VerfasserIn]   i
 Jawhar, Mohamad [VerfasserIn]   i
 Reiter, Andreas [VerfasserIn]   i
Titel:Superior efficacy of midostaurin over cladribine in advanced systemic mastocytosis
Titelzusatz:a registry-based analysis : original reports
Verf.angabe:Johannes Lübke, Juliana Schwaab, Nicole Naumann, Hans-Peter Horny, Christel Weiß, Georgia Metzgeroth, Sebastian Kreil, Nicholas C.P. Cross, Karl Sotlar, Alice Fabarius, Wolf-Karsten Hofmann, Peter Valent, Jason Gotlib, Mohamad Jawhar, and Andreas Reiter
E-Jahr:2022
Jahr:March 02, 2022
Umfang:13 S.
Illustrationen:Illustrationen
Fussnoten:Gesehen am 30.01.2024
Titel Quelle:Enthalten in: Journal of clinical oncology
Ort Quelle:Alexandria, Va. : American Society of Clinical Oncology, 1983
Jahr Quelle:2022
Band/Heft Quelle:40(2022), 16, Seite 1783-1794
ISSN Quelle:1527-7755
Abstract:Purpose - On the basis of data from the German Registry on Disorders of Eosinophils and Mast Cells, we compared the efficacy of midostaurin and cladribine in patients with advanced systemic mastocytosis (AdvSM). - Patients and Methods - Patients with AdvSM (n = 139) were treated with midostaurin only (n = 63, 45%), cladribine only (n = 23, 17%), or sequentially (midostaurin-cladribine, n = 30, 57%; cladribine-midostaurin, n = 23, 43%). Prognosis was assessed through the Mutation-Adjusted Risk Score (MARS). Besides the comparison of efficacy between midostaurin and cladribine on response (eg, organ dysfunction, bone marrow mast cell [MC] infiltration, and tryptase), overall survival (OS), and leukemia-free survival, we focused on the impact of treatment on involved non-MC lineages, for example, monocytes or eosinophils, and the KIT D816V expressed allele burden. - Results - Midostaurin only was superior to cladribine only with effects from responses on MC and non-MC lineages conferring on a significantly improved OS (median 4.2 v 1.9 years, P = .033) and leukemia-free survival (2.7 v 1.3 years, P = .044) on the basis of a propensity score-weighted analysis of parameters included in MARS. Midostaurin compensated the inferior efficacy of cladribine in first- and second-line treatment. On midostaurin in any line, response of eosinophilia did not improve its baseline adverse prognostic impact, whereas response of monocytosis proved to be a positive on-treatment parameter. Multivariable analysis allowed to establish three risk categories (low/intermediate/high) through the combination of MARS and the reduction of the KIT D816V expressed allele burden of ≥ 25% at month 6 (median OS not reached v 3.0 years v 1.0 year; P < .001). - Conclusion - In this registry-based analysis, midostaurin revealed superior efficacy over cladribine in patients with AdvSM. In midostaurin-treated patients, the combination of baseline MARS and molecular response provided a compelling three-tier risk categorization (MARSv2.0) for OS.
DOI:doi:10.1200/JCO.21.01849
URL:Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.

Volltext: https://doi.org/10.1200/JCO.21.01849
 Volltext: http://ascopubs.org/doi/10.1200/JCO.21.01849
 DOI: https://doi.org/10.1200/JCO.21.01849
Datenträger:Online-Ressource
Sprache:eng
K10plus-PPN:1879490781
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