Management of Advanced Systemic Mastocytosis and Associated Myeloid Neoplasms

Tsewang Tashi; Michael W Deininger

doi:10.1016/j.iac.2023.04.009

Management of Advanced Systemic Mastocytosis and Associated Myeloid Neoplasms

Immunol Allergy Clin North Am. 2023 Nov;43(4):723-741. doi: 10.1016/j.iac.2023.04.009. Epub 2023 Jun 4.

Authors

Tsewang Tashi¹, Michael W Deininger²

Affiliations

¹ Division of Hematology and Hematologic Malignancies, Huntsman Cancer Institute, University of Utah, 2000, Circle of Hope, Salt Lake City, UT 84112, USA. Electronic address: tsewang.tashi@utah.edu.
² Division of Hematology and Oncology, Medical College of Wisconsin, Versiti Blood Research Institute, 8727 West Watertown Plank Road, Milwaukee, WI 53226, USA.

PMID: 37758409
DOI: 10.1016/j.iac.2023.04.009

Abstract

Advanced systemic mastocytosis (AdvSM) is a heterogeneous group of disorders characterized by neoplastic mast cell-related organ damage and frequently associated with a myeloid neoplasm. The 3 clinical entities that comprise AdvSM are aggressive SM (ASM), SM-associated hematologic neoplasm, and mast cell leukemia. A gain-of-function KIT D816 V mutation is the primary oncogenic driver found in about 90% of all patients with AdvSM. Midostaurin, an oral multikinase inhibitor with activity against KIT D816V, and avapritinib, an oral selective KIT D816V inhibitor are approved for AdvSM.

Keywords: Associated hematologic neoplasm; Avapritinib; KIT D816 V; KIT inhibitors; Systemic mastocytosis; Tryptase.

Publication types

Review