Systemic mastocytosis: 2023 update on diagnosis and management in adults

Expert Opin Emerg Drugs. 2023 Dec;28(3):153-165. doi: 10.1080/14728214.2023.2221028. Epub 2023 Jun 6.

Abstract

Introduction: Systemic mastocytosis (SM) is a complex and heterogeneous disease, characterized by the clonal accumulation of mast cells in one or more organs. In 2022 both the World Health Organization (WHO) and the International Consensus Classification (ICC) modified the diagnostic and classification criteria of SM. Moreover, the identification of new clinical and molecular variables has improved prognostic tools and led to increasingly individualized therapeutic strategies.

Areas covered: The aim of this review is to present the updates introduced by the International Consensus Classification in diagnostic criteria of SM. In addition, we report the latest data available from the most important clinical trials in patients both with non-advanced and advanced disease, including elenestinib and bezuclastinib.

Expert opinion: Diagnosis and classification of SM has evolved over years. The most recent WHO and ICC classification improved SM diagnostic work-up, providing clinicians with a clear and simplified diagnostic scheme. New approved targeted therapies such as midostaurin and avapritinib modified the treatment paradigm in patients in advanced stage, and next-generation inhibitors actually investigated in clinical trials are expected in the next future.

Keywords: ICC; Systemic mastocytosis; avapritinib; bezuclastinib; elenestinib; tyrosine-kinase inhibitors.

Publication types

  • Review

MeSH terms

  • Adult
  • Humans
  • Mast Cells
  • Mastocytosis, Systemic* / diagnosis
  • Mastocytosis, Systemic* / drug therapy
  • Prognosis