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Verfasst von:Reiter, Andreas [VerfasserIn]   i
 Gotlib, Jason [VerfasserIn]   i
 Álvarez-Twose, Iván [VerfasserIn]   i
 Radia, Deepti H. [VerfasserIn]   i
 Lübke, Johannes [VerfasserIn]   i
 Bobbili, Priyanka J. [VerfasserIn]   i
 Wang, Aolin [VerfasserIn]   i
 Norregaard, Chelsea [VerfasserIn]   i
 Dimitrijevic, Saša [VerfasserIn]   i
 Sullivan, Erin [VerfasserIn]   i
 Louie-Gao, Melinda [VerfasserIn]   i
 Schwaab, Juliana [VerfasserIn]   i
 Galinsky, Ilene A. [VerfasserIn]   i
 Perkins, Cecelia [VerfasserIn]   i
 Sperr, Wolfgang R. [VerfasserIn]   i
 Sriskandarajah, Priya [VerfasserIn]   i
 Chin, Andi [VerfasserIn]   i
 Sendhil, Selvam R. [VerfasserIn]   i
 Duh, Mei Sheng [VerfasserIn]   i
 Valent, Peter [VerfasserIn]   i
 DeAngelo, Daniel J. [VerfasserIn]   i
Titel:Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis
Titelzusatz:myelodysplastic syndrome
Verf.angabe:Andreas Reiter, Jason Gotlib, Iván Álvarez-Twose, Deepti H. Radia, Johannes Lübke, Priyanka J. Bobbili, Aolin Wang, Chelsea Norregaard, Saša Dimitrijevic, Erin Sullivan, Melinda Louie-Gao, Juliana Schwaab, Ilene A. Galinsky, Cecelia Perkins, Wolfgang R. Sperr, Priya Sriskandarajah, Andi Chin, Selvam R. Sendhil, Mei Sheng Duh, Peter Valent and Daniel J. DeAngelo
E-Jahr:2022
Jahr:05 July 2022
Umfang:13 S.
Fussnoten:Gesehen am 31.10.2023
Titel Quelle:Enthalten in: Leukemia
Ort Quelle:London : Springer Nature, 1997
Jahr Quelle:2022
Band/Heft Quelle:36(2022), 8, Seite 2108-2120
ISSN Quelle:1476-5551
Abstract:Advanced systemic mastocytosis (AdvSM) is a rare myeloid neoplasm associated with poor overall survival (OS). This study (NCT04695431) compared clinical outcomes between patients with AdvSM treated with avapritinib in the Phase 1 EXPLORER (NCT0256198) and Phase 2 PATHFINDER (NCT03580655) trials (N = 176) and patients treated with best available therapy (BAT; N = 141). A multi-center, observational, retrospective chart review study was conducted at six study sites (four European, two American) to collect data from patients with AdvSM who received BAT; these data were pooled with data from EXPLORER and PATHFINDER. Comparisons between outcomes of OS, duration of treatment (DOT), and maximum reduction in serum tryptase were conducted between the treatment cohorts, with adjustment for key covariates. The results indicated that the avapritinib cohort had significantly better survival (adjusted hazard ratio (HR) (95% confidence interval (CI)): 0.48 (0.29, 0.79); p = 0.004) and significantly longer DOT (HR: 0.36 (0.26, 0.51); p < 0.001) compared to the BAT cohort. Additionally, the mean difference in percentage maximum reduction in serum tryptase levels was 60.3% greater in the avapritinib cohort (95% CI: −72.8, −47.9; p < 0.001). With no randomized controlled trials comparing avapritinib to BAT, these data offer crucial insights into the improved efficacy of avapritinib for the treatment of AdvSM.
DOI:doi:10.1038/s41375-022-01615-z
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kostenfrei: Volltext: https://doi.org/10.1038/s41375-022-01615-z
 kostenfrei: Volltext: http://www.nature.com/articles/s41375-022-01615-z
 DOI: https://doi.org/10.1038/s41375-022-01615-z
Datenträger:Online-Ressource
Sprache:eng
Sach-SW:Cancer therapy
 Targeted therapies
K10plus-PPN:1868839656
Verknüpfungen:→ Zeitschrift

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