Role of Luspatercept in the Management of Lower-Risk Myelodysplastic Syndromes.
Role of Luspatercept in the Management of Lower-Risk Myelodysplastic Syndromes.
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DOI：
10.6004/jadpro.2023.14.1.8
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发表时间：
2023-01
期刊：
影响因子：
--
通讯作者：
Ajayi O
中科院分区：
文献类型：
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作者：
Tinsley-Vance SM;Davis M;Ajayi O
低风险骨髓增生异常综合征（LR-MDS）相关贫血患者的治疗选择有限。luspatercept最近获批用于治疗极低至中等风险MDS伴环形铁粒幼细胞（RS）或骨髓增生异常/骨髓增生性肿瘤伴RS和血小板增多相关贫血，为成人患者和从业者提供了急需的新治疗选择。Luspatercept是一种一流的红细胞成熟剂，对红细胞生成的后期阶段发挥作用。在伴有RS的LR-MDS患者的III期MEDALIST试验中，luspatercept（起始剂量1 mg/kg）显示了显著的临床获益（38%的luspatercept治疗患者与13%的安慰剂治疗患者[p < .001]在治疗的前24周内实现了≥ 8周的输血独立性）和有利的安全性特征。最常见的不良事件（AE），包括疲乏、无力、头晕和腹泻，在前4个治疗周期内更常见，随后下降。本综述全面概述了luspatercept治疗给药，包括作用机制、疗效和安全性数据、给药管理以及与LR-MDS患者luspatercept治疗相关的AE。
Treatment options are limited for patients with anemia associated with lower-risk myelodysplastic syndromes (LR-MDS). The recent approval of luspatercept for the treatment of anemia associated with very low-to intermediate-risk MDS with ring sideroblasts (RS) or with myelodysplastic/myeloproliferative neoplasm with RS and thrombocytosis has provided adult patients and practitioners with a much-needed new therapeutic option. Luspatercept is a first-in-class erythroid maturation agent that exerts its effects on later stages of erythropoiesis. In the phase III MEDALIST trial of patients with LR-MDS with RS, luspatercept (starting dose 1 mg/kg) demonstrated substantial clinical benefit (38% of patients treated with luspatercept vs. 13% of those treated with placebo [p < .001] achieved transfusion independence for ≥ 8 weeks during the first 24 weeks of treatment) and a favorable safety profile. The most common adverse events (AEs), including fatigue, asthenia, dizziness, and diarrhea, were more frequent during the first 4 treatment cycles and subsequently declined. This review provides a comprehensive overview of luspatercept treatment administration, including the mechanism of action, efficacy and safety data, management of dosing, and AEs associated with luspatercept treatment of patients with LR-MDS.