Background: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.Methods:In this review, we discuss the journey of patients with myelofibrosis and anemia across various severities and clinical scenarios.Results: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.Conclusions: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.
背景：贫血是骨髓纤维化常见且进行性的临床表现，既可能作为疾病发病机制的一部分出现，也可能由某些治疗的骨髓抑制作用引起，对患者生活质量、预后及医疗资源利用产生重大影响。尽管存在这些负担，传统上贫血管理一直被视为脾脏和症状控制的次要优先事项，部分原因是现有治疗方法的局限性。随着莫莫洛替尼（一种Janus激酶1（JAK1）、JAK2和激活素A受体1型抑制剂）的初步监管批准，该药物在解决脾肿大和症状的同时还能带来贫血相关获益，因此有必要重新评估将贫血作为早期重要治疗考量因素的价值。 方法：本综述探讨了骨髓纤维化伴贫血患者在不同严重程度和临床情境下的诊疗历程。 结果：本文总结了贫血管理的传统方法，以及支持莫莫洛替尼作为从轻度到重度贫血（包括合并血小板减少症的情况）各阶段治疗选择的临床试验疗效和安全性数据。 结论：随着莫莫洛替尼及其他针对贫血控制的新兴疗法的问世，早期治疗贫血以避免疾病进展并支持符合条件的骨髓纤维化患者获得改善，应成为首要考虑的治疗策略。
Anemia in Myelofibrosis: A Focus on Proactive Management and the Role of Momelotinib
肿瘤(癌症)患者之家