Myelodysplastic syndromes are characterised by ineffective erythropoiesis. Luspatercept (ACE-536) is a novel fusion protein that blocks transforming growth factor beta (TGF β) superfamily inhibitors of erythropoiesis, giving rise to a promising new investigative therapy. We aimed to assess the safety and efficacy of luspatercept in patients with anaemia due to lower-risk myelodysplastic syndromes.

中文翻译：

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Luspatercept用于治疗低危骨髓增生异常综合症（PACE-MDS）贫血的患者：一项多中心，开放标签的2期剂量寻找研究，并进行了长期扩展研究。

骨髓增生异常综合症的特征是无效的红细胞生成。Luspatercept（ACE-536）是一种新型融合蛋白，可阻断促红细胞生成的转化生长因子β（TGFβ）超家族抑制剂，从而产生了有希望的新研究方法。我们旨在评估因低危骨髓增生异常综合征而导致的贫血患者接受luspatercept的安全性和有效性。