Systemic Mastocytosis: State of the Art.

IF 2.7 3区医学 Q2 HEMATOLOGY

Current Hematologic Malignancy Reports Pub Date : 2024-10-01 Epub Date: 2024-08-27 DOI:10.1007/s11899-024-00737-8

Isabel Farmer, Deepti H Radia

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Abstract

Purpose of review: Since identification of Systemic mastocytosis (SM) as a distinct disease entity by the World Health Organisation (WHO), there has been a wealth of new research in therapeutic targeting of the pathogenic C-KIT D816V mutation.

Recent findings: Avapritinib, the first licensed drug in SM capable of disease modification alongside the increasingly potent, oral and highly selective KIT tyrosine kinase inhibitors (TKIs) Bezuclastinib and now Elenestinib have enabled the prospect of long-term remissions. Studies have shown improved survival and symptomatic control in patients with SM. Of great triumph, this has been achieved in an outpatient setting with apparent tolerable and minimal toxicity. The importance of molecular profiling is being demonstrated in administering combination therapies for SM with an associated haematological neoplasm (AHN), allowing more personalised and streamlined treatment regimes. This review focuses on current management strategies of SM, focusing on state-of-the-art directed therapies, the evidence behind their use with presentation of two clinical cases to highlight key messages.

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系统性肥大细胞增多症：艺术现状。

综述目的：自世界卫生组织（WHO）将系统性肥大细胞增多症（SM）确定为一种独特的疾病实体以来，针对致病性C-KIT D816V突变的治疗研究层出不穷：阿伐替尼（Avapritinib）是第一种能够改变 SM 疾病的获准药物，它与越来越强效的口服高选择性 KIT 酪氨酸激酶抑制剂（TKIs）贝祖拉司替尼（Bezuclastinib）以及现在的艾伦替尼（Elenestinib）一起，为长期缓解带来了希望。研究显示，SM 患者的生存率和症状控制率均有所提高。最令人欣喜的是，这种治疗是在门诊环境下实现的，而且毒性明显可耐受且极小。在对伴有血液肿瘤（AHN）的SM患者进行联合治疗时，分子图谱分析的重要性得到了证实，从而可以提供更加个性化和简化的治疗方案。本综述侧重于当前的 SM 治疗策略，重点介绍最先进的定向疗法、使用这些疗法的证据以及两个临床病例，以突出关键信息。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Current Hematologic Malignancy Reports ONCOLOGY-HEMATOLOGY

CiteScore

6.00

自引率

0.00%

发文量

审稿时长

>12 weeks

期刊介绍： his journal intends to provide clear, insightful, balanced contributions by international experts that review the most important, recently published clinical findings related to the diagnosis, treatment, management, and prevention of hematologic malignancy. We accomplish this aim by appointing international authorities to serve as Section Editors in key subject areas, such as leukemia, lymphoma, myeloma, and T-cell and other lymphoproliferative malignancies. Section Editors, in turn, select topics for which leading experts contribute comprehensive review articles that emphasize new developments and recently published papers of major importance, highlighted by annotated reference lists. An international Editorial Board reviews the annual table of contents, suggests articles of special interest to their country/region, and ensures that topics are current and include emerging research. Commentaries from well-known figures in the field are also provided.