Avapritinib versus Placebo in Indolent Systemic Mastocytosis

NEJM Evid. 2023 Jun;2(6):EVIDoa2200339. doi: 10.1056/EVIDoa2200339. Epub 2023 May 23.

Abstract

Avapritinib in Indolent Systemic MastocytosisIn a randomized trial, patients with indolent systemic mastocytosis were treated with avapritinib or placebo along with supportive care. The trial primary end point was the change in mean total symptom scores at 24 weeks. Avapritinib-treated patients had a decrease in mean total symptom score of 15.6 points compared with 9.2 points in the placebo group.

Publication types

  • Randomized Controlled Trial

MeSH terms

  • Humans
  • Mastocytosis, Systemic* / diagnosis
  • Pyrazoles / therapeutic use
  • Pyrroles / therapeutic use
  • Triazines / therapeutic use

Substances

  • avapritinib
  • Pyrazoles
  • Pyrroles
  • Triazines