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Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis: myelodysplastic syndrome

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 =...

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Main Authors: Reiter, Andreas (Author) , Gotlib, Jason (Author) , Álvarez-Twose, Iván (Author) , Radia, Deepti H. (Author) , Lübke, Johannes (Author) , Bobbili, Priyanka J. (Author) , Wang, Aolin (Author) , Norregaard, Chelsea (Author) , Dimitrijevic, Saša (Author) , Sullivan, Erin (Author) , Louie-Gao, Melinda (Author) , Schwaab, Juliana (Author) , Galinsky, Ilene A. (Author) , Perkins, Cecelia (Author) , Sperr, Wolfgang R. (Author) , Sriskandarajah, Priya (Author) , Chin, Andi (Author) , Sendhil, Selvam R. (Author) , Duh, Mei Sheng (Author) , Valent, Peter (Author) , DeAngelo, Daniel J. (Author)
Format: Article (Journal)
Language:English
Published: 05 July 2022
In: Leukemia
Year: 2022, Volume: 36, Issue: 8, Pages: 2108-2120
ISSN:1476-5551
DOI:10.1038/s41375-022-01615-z
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1038/s41375-022-01615-z
Verlag, kostenfrei, Volltext: http://www.nature.com/articles/s41375-022-01615-z
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Author Notes: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
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Summary: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.
Item Description:Gesehen am 31.10.2023
Physical Description:Online Resource
ISSN:1476-5551
DOI:10.1038/s41375-022-01615-z

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