Ineffective erythropoiesis (IE) is the most prominent feature of transfusion-dependent beta-thalassemia (TDT), which leads to extramedullary hemopoiesis. The rejection rate in allogeneic hematopoietic stem cell transplantation (HSCT) is high in heavily transfused patients with TDT accompanied by prominent IE. Therefore, a pretransplantation treatment bridging to HSCT is often used to reduce allosensitization and IE. Ruxolitinib is a JAK-1/JAK-2 inhibitor and has showed its efficacy in suppressing IE and the immune system. A previously published study on RUX in adult patients with TDT has revealed that this treatment significantly reduces spleen size and is well tolerated.

中文翻译：

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鲁索替尼在无效红细胞生成抑制中作为儿童重型β-地中海贫血患者移植前治疗的疗效和安全性

无效红细胞生成 (IE) 是输血依赖性 β 地中海贫血 (TDT) 最突出的特征，可导致髓外造血。大量输血的 TDT 伴有显着 IE 的同种异体造血干细胞移植 (HSCT) 的排斥率很高。因此，桥接 HSCT 的移植前治疗通常用于减少同种异体致敏和 IE。Ruxolitinib 是一种 JAK-1/JAK-2 抑制剂，已显示出其抑制 IE 和免疫系统的功效。先前发表的一项针对 TDT 成年患者的 RUX 研究表明，这种治疗可显着减小脾脏大小并且耐受性良好。