Comparison of efficacy and safety of thalidomide vs hydroxyurea in patients with Hb E-β thalassemia-a pilot study from a tertiary care Centre of India
Comparison of efficacy and safety of thalidomide vs hydroxyurea in patients with Hb E-β thalassemia-a pilot study from a tertiary care Centre of India
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DOI：
10.1016/j.bcmd.2021.102544
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发表时间：
2021-02-18
影响因子：
2.3
通讯作者：
De, Rajib
中科院分区：
文献类型：
--
作者：
Jain, Manisha;Chakrabarti, Prantar;De, Rajib
简介：已知血红蛋白 (Hb)-F 诱导剂可改善地中海贫血的 Hb 水平和输血依赖性。本试验研究的目的是评估 Hb-F 诱导剂沙利度胺与羟基脲 (HU) 相比，对 Hb E-β 地中海贫血患者的疗效和安全性。 方法：这是一项前瞻性介入单中心研究，45 名 Hb E-β 地中海贫血患者平均分为 I 组（沙利度胺 + 叶酸）、II 组（HU + 叶酸）和 III 组（叶酸）。在 12 个月的随访期内，以不同的时间间隔评估疗效。主要终点是 Hb、Hb-F 水平的增加和输血需求的改善；次要终点是耐受性和安全性。结果：I 组有 100% 的应答者（R：Hb 增量 >1 g/dl），主要应答者有 66.67%（MaR：Hb 增量 >2 g/dl），而 II 组和 III 组分别有 40% 和 0% 的应答者。 Hb 增量显着（p 值
Introduction: Hemoglobin (Hb)-F inducers are known to improve Hb level and transfusion dependence in thalassemia. This pilot study was conducted to assess the efficacy and safety of Hb-F inducer thalidomide compared to hydroxyurea (HU) in Hb E-beta thalassemia patients.Methods: This was a prospective interventional single-centre study with 45 Hb E-beta thalassemia patients equally divided into group-I (thalidomide+folic acid), group-II (HU + folic acid) and group-III (folic acid). Response was assessed at various time intervals with 12-months follow up period. Primary end points were increment in Hb, Hb-F level and improvement in transfusion requirement; secondary end point were tolerability and safety.Results: There was 100% responder (R: Hb-increment >1 g/dl) in group-I with 66.67% major responder (MaR: Hb-increment >2 g/dl), while there were 40% and 0% responder in group-II and III respectively. Hb-increment was significantly (p-value