Pilo F, Angelucci E
Hematology and Transplant Center, Azienda Ospedaliera Brotzu, Cagliari, Italy.
Hematology and Transplant Center, IRCCS Ospedale Policlinico San Martino, Genoa, Italy.
Drugs Today (Barc). 2020 Jul;56(7):447-458. doi: 10.1358/dot.2020.56.7.3159184.
Recently, after years of research often characterized by disappointments and frustrations, finally a new drug impacting on pathological human erythropoiesis has been developed and approved. This drug, luspatercept-aamt (Reblozyl), proved to be effective in both malignant and nonmalignant disease characterized by ineffective erythropoiesis with consequent life-threatening severe anemia. Moreover, for the first time, a medication demonstrated efficacy and effectiveness in β-thalassemia where no other drug, including recombinant human erythropoietin, showed effectiveness in improving anemia. Despite recent impressive advances in understanding human normal and abnormal erythropoiesis, there are few new drugs and limited pharma research focusing on ineffective erythropoiesis. This review will discuss recent advances in understanding normal and pathological erythropoiesis that represent the background to discuss pharmacology, toxicology, efficacy, safety and effectiveness of this new drug for the treatment of human β-thalassemia.
最近，经过多年常常以失望和挫折为特征的研究，终于研发并批准了一种对人类病理性红细胞生成有影响的新药。这种药物，luspatercept-aamt（瑞布洛司他），在以无效红细胞生成并导致危及生命的严重贫血为特征的恶性和非恶性疾病中均被证明有效。此外，首次有一种药物在β地中海贫血中显示出疗效，而包括重组人促红细胞生成素在内的其他药物在改善贫血方面均未显示出效果。尽管最近在理解人类正常和异常红细胞生成方面取得了令人瞩目的进展，但针对无效红细胞生成的新药很少，制药研究也很有限。本综述将讨论在理解正常和病理性红细胞生成方面的最新进展，这些进展构成了讨论这种新药治疗人类β地中海贫血的药理学、毒理学、疗效、安全性和有效性的背景。
