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骨髓纤维化中的贫血:关注积极管理及莫洛替尼的作用

Anemia in Myelofibrosis: A Focus on Proactive Management and the Role of Momelotinib.

作者信息

Al-Ali Haifa Kathrin, Kuykendall Andrew T, Ellis Catherine E, Sampath Janardhan, Mesa Ruben

机构信息

Krukenberg Cancer Center Halle, University Hospital Halle, 06120 Halle, Germany.

Moffit Cancer Center, Tampa, FL 33612, USA.

出版信息

Cancers (Basel). 2024 Dec 4;16(23):4064. doi: 10.3390/cancers16234064.

Abstract

Anemia is a common and progressive clinical manifestation of myelofibrosis that may occur as part of the disease pathogenesis as well as due to the myelosuppressive effects of some treatments, with a substantial impact on quality of life, prognosis, and healthcare resource utilization. Despite these burdens, anemia management has traditionally been a secondary priority to spleen and symptom control, due in part to the limitations of available therapeutic approaches. With the initial regulatory approvals of momelotinib, a Janus kinase 1 (JAK1), JAK2, and activin A receptor type 1 inhibitor that provides anemia-related benefits in addition to addressing splenomegaly and symptoms, re-evaluation of anemia as an early and prominent treatment consideration is warranted. In this review, we discuss the journey of patients with myelofibrosis and anemia across various severities and clinical scenarios. Summarized are traditional approaches to anemia management and the clinical trial efficacy and safety data that support momelotinib as an option in each setting from mild to severe anemia, including in the context of co-occurring thrombocytopenia. With the availability of momelotinib and other emerging therapies directed at anemia control, early treatment of anemia to avoid progression and support improvement in eligible patients with myelofibrosis should be a primary consideration.

摘要

贫血是骨髓纤维化常见且进行性的临床表现,它可能作为疾病发病机制的一部分出现,也可能是某些治疗的骨髓抑制作用所致,对生活质量、预后及医疗资源利用有重大影响。尽管有这些负担,但传统上贫血管理相对于脾脏和症状控制而言是次要优先事项,部分原因是现有治疗方法存在局限性。随着莫洛替尼(一种同时抑制 Janus 激酶 1(JAK1)、JAK2 和激活素 A 受体 1 型的抑制剂,除了能解决脾肿大和症状外还具有与贫血相关的益处)最初获得监管批准,有必要重新将贫血视为早期且重要的治疗考量因素。在本综述中,我们讨论了不同严重程度和临床情形下骨髓纤维化合并贫血患者的情况。总结了贫血管理的传统方法以及支持莫洛替尼作为从轻度到重度贫血各情形下(包括合并血小板减少症的情况下)一种选择的临床试验疗效和安全性数据。鉴于莫洛替尼及其他针对贫血控制的新兴疗法的出现,早期治疗贫血以避免病情进展并支持符合条件的骨髓纤维化患者病情改善应成为首要考虑因素。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a2ce/11640463/7e1ebdf998b2/cancers-16-04064-g001.jpg

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