In the randomized phase of the SUMMIT trial of bezuclastinib (100 mg daily) versus placebo in indolent and smoldering systemic mastocytosis, which finding did the speaker highlight beyond the primary symptom endpoint?
Symptoms improved, but tryptase, KIT D816V VAF and bone marrow mast cell burden were largely unchanged, suggesting a purely symptomatic effect.
Markers of disease burden fell profoundly, with about 55% of patients no longer meeting WHO diagnostic criteria for SM, and symptom reduction correlated with the fall in these markers.
Bezuclastinib reduced disease burden markers but did not significantly improve symptoms compared with placebo at 24 weeks.