Efficacy of avapritinib versus best available therapy for advanced systemic mastocytosis

Patients with advanced systemic mastocytosis (AdvSM) have a poor prognosis. More than 90% of AdvSM patients carry a D816V mutation in the KIT gene. Avapritinib is a selective and potent inhibitor of D816V-mutated KIT. No randomize controlled trial have compared avapritinib versus best available therapy (BAT) for AdvSM. Outcomes of avapritinib treated patients in the phase 1 EXPLORER trial and the phase 2 PATHFINDER trial have been compared with retrospectively collected real-world outcomes of patients of six centers (four European, two American). The avapritinib treated patients had significantly longer overall survival and duration of treatment than the BAT group. The authors conclude that these data offer crucial insights into the efficacy of avapritinib for AdvSM.