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Management of Advanced Systemic Mastocytosis: Clinical Challenges.

Authors :
Tremblay D
Wagner NE
Mascarenhas J
Source :
Journal of blood medicine [J Blood Med] 2024 Sep 11; Vol. 15, pp. 421-433. Date of Electronic Publication: 2024 Sep 11 (Print Publication: 2024).
Publication Year :
2024

Abstract

Advanced systemic mastocytosis (AdvSM) is a rare hematologic malignancy with organ damage and compromised life expectancy arising from organ accumulation of neoplastic mast cells. Identification of the gain-of-function KIT D816V in the majority of cases has accelerated pharmaceutical development culminating with the development of selective KIT inhibitors such as avapritinib. While the advent of these therapies has improved the quality and quantity of life in patients with AdvSM, current challenges remain in the management of this disease. In this review, we summarize the present and future therapeutics landscape of AdvSM, highlighting the development of novel KIT inhibitors including elenestinib and bezuclastinib. We also explore the continued role of additional treatment modalities including allogeneic stem cell transplantation before discussing unresolved clinical challenges in the management of AdvSM.<br />Competing Interests: DT received research funding from Sobi, Sumitomo, Cogent Biosciences and Gilead and honoraria from Sobi, Novartis, AbbVie, PharmaEssentia, Sierra Oncology, GSK and Cogent Biosciences. JM received research funding from Incyte, BMS, Novartis, AbbVie, Geron, Kartos, Karyopharm, CTI/Sobi and PharmaEssentia and honoraria from Incyte, Blueprint Medicines, Keros, DISC Medicines, BMS, AbbVie, Kartos, Geron, GSK, Roche, Merck, Pfizer, PharmaEssentia, MorphoSys, Novartis, Galecto, Sobi, Sumitomo, and Karyopharm. The authors report no other conflicts of interest in this work.<br /> (© 2024 Tremblay et al.)

Details

Language :
English
ISSN :
1179-2736
Volume :
15
Database :
MEDLINE
Journal :
Journal of blood medicine
Publication Type :
Academic Journal
Accession number :
39279879
Full Text :
https://doi.org/10.2147/JBM.S366367