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

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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Published in:Leukemia 2022-08, Vol.36 (8), p.2108-2120
Main Authors: Reiter, Andreas, Gotlib, Jason, Álvarez-Twose, Iván, Radia, Deepti H., Lübke, Johannes, Bobbili, Priyanka J., Wang, Aolin, Norregaard, Chelsea, Dimitrijevic, Saša, Sullivan, Erin, Louie-Gao, Melinda, Schwaab, Juliana, Galinsky, Ilene A., Perkins, Cecelia, Sperr, Wolfgang R., Sriskandarajah, Priya, Chin, Andi, Sendhil, Selvam R., Duh, Mei Sheng, Valent, Peter, DeAngelo, Daniel J.
Format: Article
Language:English
Subjects:
692/699/67/1059
692/699/67/1059/602
Cancer
Cancer Research
Clinical outcomes
Clinical trials
Critical Care Medicine
Data collection
Drug dosages
Hematology
Hospitals
Intensive
Internal Medicine
Leukemia
Mastocytosis
Medical prognosis
Medicine
Medicine & Public Health
Oncology
Patients
Reduction
Survival
Tryptase
Tumors
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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  
ISSN:0887-6924
1476-5551
DOI:10.1038/s41375-022-01615-z