Online-Ressource | |
Verfasst von: | Lübke, Johannes [VerfasserIn] |
Schwaab, Juliana [VerfasserIn] | |
Naumann, Nicole [VerfasserIn] | |
Horny, Hans-Peter [VerfasserIn] | |
Weiß, Christel [VerfasserIn] | |
Metzgeroth, Georgia [VerfasserIn] | |
Kreil, Sebastian [VerfasserIn] | |
Cross, Nicholas C.P. [VerfasserIn] | |
Sotlar, Karl [VerfasserIn] | |
Fabarius, Alice [VerfasserIn] | |
Hofmann, Wolf-Karsten [VerfasserIn] | |
Valent, Peter [VerfasserIn] | |
Gotlib, Jason [VerfasserIn] | |
Jawhar, Mohamad [VerfasserIn] | |
Reiter, Andreas [VerfasserIn] | |
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 |
Verknüpfungen: | → Zeitschrift |