Treatment advances for pediatric and adult onset neoplasms with monocytosis.

IF 2.7 3区 医学 Q2 HEMATOLOGY
Current Hematologic Malignancy Reports Pub Date : 2021-06-01 Epub Date: 2021-03-16 DOI:10.1007/s11899-021-00622-8
Kristen B McCullough, Alexis K Kuhn, Mrinal M Patnaik
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引用次数: 0

Abstract

Purpose of review: For decades, the management of chronic myelomonocytic leukemia (CMML) or juvenile myelomonocytic leukemia (JMML) has been largely inextricable from myelodysplastic syndromes (MDS), myeloproliferative neoplasms, and acute myeloid leukemia. Hallmarks of these diseases have been the emergence of unique genomic signatures and discouraging responses to available therapies. Here, we will critically examine the current options for management and review the rapidly developing opportunities based on advances in CMML and JMML disease biology.

Recent findings: Few clinical trials have exclusively been done in CMML, and in JMML, the rarity of the disease limits wide scale participation. Recent case series in JMML suggest that hypomethylating agents (HMAs) are a viable option for bridging to curative intent with allogeneic hematopoietic stem cell transplant or as posttransplant maintenance. Emerging evidence has demonstrated targeting the RAS-pathway via MEK inhibition may also be considered. In CMML, treatment with HMAs is largely derived from data inclusive of MDS patients, including a small number of patients with dysplastic CMML variants. Based on CMML disease biology, additional therapeutic targets being investigated include inhibitors of splicing, CD123/dendritic cell axis, inherent GM-CSF progenitor cell hypersensitivity, and targeting the JAK/STAT pathway. Current evidence is also expanding for oral HMAs. The management of CMML and JMML is rapidly evolving and clinicians must be aware of the genetic landscape and expanding treatment options to ensure these rare populations are afforded therapeutic interventions best suited to their needs.

儿童和成人单核细胞增多症发病肿瘤的治疗进展。
回顾目的:几十年来,慢性髓细胞白血病(CMML)或少年髓细胞白血病(JMML)的治疗在很大程度上与骨髓增生异常综合征(MDS)、骨髓增生性肿瘤和急性髓细胞白血病密不可分。这些疾病的特点是出现了独特的基因组特征和对现有治疗的令人沮丧的反应。在这里,我们将严格检查当前的管理选择,并根据CMML和JMML疾病生物学的进展回顾快速发展的机会。最近的发现:很少有临床试验专门针对CMML,而在JMML中,这种疾病的罕见性限制了广泛的参与。最近的JMML病例系列表明,低甲基化药物(HMAs)是异体造血干细胞移植或移植后维持治疗目的的可行选择。新出现的证据表明,也可以考虑通过MEK抑制靶向ras通路。在CMML中,HMAs的治疗主要来自MDS患者的数据,包括少数CMML变异发育不良的患者。基于CMML疾病生物学,正在研究的其他治疗靶点包括剪接抑制剂,CD123/树突状细胞轴,固有的GM-CSF祖细胞过敏,以及靶向JAK/STAT通路。目前关于口服hma的证据也在不断增加。CMML和JMML的治疗正在迅速发展,临床医生必须意识到遗传景观和扩大治疗选择,以确保这些罕见的人群得到最适合他们需要的治疗干预。
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
CiteScore
6.00
自引率
0.00%
发文量
28
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.
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