Recent Advances in the Therapeutic Management of Advanced Systemic Mastocytosis

被引：2

作者：

Veitch, Scott ^{[1
]}

Radia, Deepti H. ^{[1
]}

机构：

[1] Guys & St Thomas NHS Fdn Trust, Dept Haematol, London SE1 7EH, England

来源：

DIAGNOSTICS | 2024年 / 14卷 / 01期

关键词：

advanced systemic mastocytosis; tyrosine kinase inhibitors; KIT inhibitors; midostaurin; avapritinib; bezuclastinib; KINASE INHIBITOR STI571; MAST-CELL LEUKEMIA; IMATINIB MESYLATE; INTERFERON-ALPHA; WILD-TYPE; EFFICACY; SAFETY; MUTATIONS; MIDOSTAURIN; AVAPRITINIB;

D O I：

10.3390/diagnostics14010080

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 haematological neoplasm characterised by the accumulation of neoplastic mast cells (MCs) in various organs, resulting in organ dysfunction and reduced life expectancy. The subtypes include aggressive SM (ASM), SM with an associated haematological neoplasm (SM-AHN) and mast cell leukaemia (MCL). The gain of function KIT D816V mutation is present in most cases. The availability of tyrosine kinase inhibitors (TKIs) has revolutionised the treatment landscape for patients with this life-limiting disease. Patients are now able to achieve molecular remission, improved quality of life and improved overall survival. This review focuses on the targeted therapies currently available in clinical practice and within the clinical trial setting for AdvSM. This review also highlights possible future therapeutic targets and discusses therapeutic strategies for this multimutated and clinically heterogeneous disease.

引用

页数：13

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