Management of Advanced Systemic Mastocytosis and Associated Myeloid Neoplasms - 26/09/23

Doi : 10.1016/j.iac.2023.04.009

Tsewang Tashi, MD ^a,^⁎ , Michael W. Deininger, MD, PhD ^b
^a Division of Hematology and Hematologic Malignancies, Huntsman Cancer Institute, University of Utah, 2000, Circle of Hope, Salt Lake City, UT 84112, USA
^b Division of Hematology and Oncology, Medical College of Wisconsin, Versiti Blood Research Institute, 8727 West Watertown Plank Road, Milwaukee, WI 53226, USA

^∗Corresponding author.

Résumé

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.

Le texte complet de cet article est disponible en PDF.

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