Management of Advanced Systemic Mastocytosis: Clinical Challenges

被引：1

作者：

Tremblay, Douglas ^{[1
]}

Wagner, Nicole E. ^{[2
]}

Mascarenhas, John ^{[1
]}

机构：

[1] Tisch Canc Inst, Icahn Sch Med Mt Sinai, New York, NY USA

[2] Icahn Sch Med Mt Sinai, Dept Med, New York, NY USA

来源：

JOURNAL OF BLOOD MEDICINE | 2024年 / 15卷

关键词：

systemic mastocytosis; KITD816V; avapritinib; midostaurin; HEALTH-ORGANIZATION CLASSIFICATION; KIT D816V; CELL TRANSPLANTATION; RESPONSE CRITERIA; INTERFERON-ALPHA; C-KIT; EFFICACY; SAFETY; MIDOSTAURIN; MUTATION;

D O I：

10.2147/JBM.S366367

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：421 / 433

页数：13

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