肥大细胞白血病:综合文献综述与当前的见解和最新的管理。

IF 1.3 Q4 HEMATOLOGY
Journal of hematology Pub Date : 2025-08-25 eCollection Date: 2025-08-01 DOI:10.14740/jh2104
Muralidhar Idamakanti, Ala Ebaid, Rani Indrani Bijjam, Alexei Bakhirev
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引用次数: 0

摘要

肥大细胞白血病(MCL)是一种非常罕见和侵袭性的系统性肥大细胞增多症(SM)。MCL分为原发、无既往肥大细胞(MC)疾病、继发、既往SM、急性侵袭性、c -表现提示器官损伤或慢性惰性、无器官损伤。在这些病例中,60-65%为白血病,外周血循环MCs < 10%,其余为白血病,外周血循环MCs < 10%。诊断通常通过骨髓活检证实,在涂片中发现超过20%的非典型或未成熟的MCs。针对MCL的特异性治疗是有限的,用于SM和急性髓性白血病(AML)的多种治疗方式已经在MCL中进行了试验,但成功率有限,生存获益也不确定。自从发现受体酪氨酸激酶III型(KIT) D816V突变在SM和MCL的发病机制中起作用以来,治疗取得了显著进展。批准用于MCL的两种靶向治疗方法是midostoin(一种多激酶抑制剂)和avapritinib(一种选择性KIT D816V靶向突变的酪氨酸激酶抑制剂)。治疗MCL的多种药物正在临床试验中进行评估。MCL预后较差,中位总生存期(OS)约为1.5年。进一步的进展和研究对于开发可能提高中位生存期的治疗方法至关重要。在这篇文章中,我们对MCL进行了全面而简单的回顾,主要关注其临床表现和最新的治疗进展。我们还确定了需要进一步研究的领域,并强调了与该疾病相关的侵袭性和不良预后。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Mast Cell Leukemia: Comprehensive Review of Literature With Current Insights and Updates on Management.

Mast cell leukemia (MCL) is an exceedingly rare and aggressive variant of systemic mastocytosis (SM). MCL is classified as primary, occurring de novo without prior mast cell (MC) disorders or secondary, from a pre-existing SM, and acute aggressive form with C-findings that indicate organ damage or chronic indolent form without organ damage. Of the cases, 60-65% are aleukemic with < 10% circulating MCs in the peripheral blood, and the rest of the cases are leukemic with > 10% MCs. Diagnosis is typically confirmed by bone marrow biopsy revealing greater than 20% atypical or immature MCs in the smear. Specific MCL-targeted treatments are limited, and multiple treatment modalities used for SM and acute myeloid leukemia (AML) have been tried in MCL with limited success and variable survival benefit. The management has significantly advanced since the implication of the receptor tyrosine kinase type III (KIT) D816V mutation in the pathogenesis of SM and MCL. The two targeted therapies approved for MCL are midostaurin, a multikinase inhibitor, and avapritinib, a selective KIT D816V mutation-targeted tyrosine kinase inhibitor. Multiple drugs are being evaluated in clinical trials for managing MCL. MCL has a poor prognosis with a median overall survival (OS) of around 1.5 years. Further advancements and research are essential to develop treatments that may enhance median OS. In this article, we conducted a comprehensive yet simplified review of MCL, focusing primarily on its clinical manifestations and recent updates on management. We also identified the areas that require further research and emphasized the aggressive nature and poor prognosis associated with this disease.

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来源期刊
Journal of hematology
Journal of hematology HEMATOLOGY-
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