Loading
A low dose of the KIT inhibitor avapritinib eased the itching, flushing, gut and bone symptoms of indolent systemic mastocytosis more than placebo, the first drug shown to do so in a randomised trial.
Randomised double-blind phase 2 trial of 212 patients with indolent systemic mastocytosis and moderate to severe symptoms despite best supportive care, assigned to avapritinib 25 mg daily or placebo for 24 weeks.
The total symptom score fell by 15.6 points with avapritinib against 9.2 with placebo, with parallel falls in serum tryptase, KIT D816V allele burden and bone marrow mast cells; adverse events were mostly mild. The FDA approved avapritinib for indolent systemic mastocytosis in 2023.
Patients with indolent systemic mastocytosis whose symptoms persist on antihistamines and mast cell stabilisers now have a disease-modifying option.
Shares Indolent and smouldering systemic mastocytosis, Avapritinib.
Shares Indolent and smouldering systemic mastocytosis, Avapritinib.
Shares Indolent and smouldering systemic mastocytosis, Avapritinib.