Avapritinib versus Placebo in Indolent Systemic Mastocytosis

Key Findings

• ●PIONEER trial: avapritinib superior to placebo in ISM
• ●TSS decrease: -15.6 vs -9.2 points (P<0.003)
• ●54% achieved >=50% tryptase reduction vs 0% placebo
• ●First FDA-approved treatment for ISM (May 2023)

Referenced in (1 disease)