Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Gene-targeting therapy shown to reduce mastocytosis symptoms

Key clinical point: A novel small-molecule kinase inhibitor showed potential in treating systemic mastocytosis.

Major finding: Treated patients reported a 30% average reduction in symptoms.

Study details: Part 1 of a phase 2 randomized trial of avapritinib in 39 patients.

Disclosures: Dr. Akin, the primary investigator, receives funding from and serves as a consultant for Blueprint Medicines, which sponsored the trial. He also disclosed a financial relationship with Novartis.


Akin C et al. AAAAI 2020, Presentation L5.