Thalidomide and Hydroxyurea in Transfusion-Dependent Thalassemia: Efficacy, Safety Profile and Impact on Quality of Life.

IF 1.2 4区 医学 Q4 BIOCHEMISTRY & MOLECULAR BIOLOGY
Hemoglobin Pub Date : 2024-05-01 Epub Date: 2024-08-02 DOI:10.1080/03630269.2024.2386076
Sukrita Bhattacharjee, Shouriyo Ghosh, Jyoti Shaw, Sunistha Bhattacharjee, Maitreyee Bhattacharyya
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引用次数: 0

Abstract

Transfusion-dependent thalassemia (TDT) is a major public health concern in India, requiring regular transfusions for survival. There is also significant morbidity caused by iron overload and transfusion related infections. Novel therapies targeting fetal hemoglobin induction are the need of the hour in resource-poor institutions for patients where transplant is not feasible for various reasons. This single arm, non-randomised prospective trial evaluated the efficacy and safety of a combination of low dose thalidomide and hydroxyurea in TDT along with the impact on quality of life (QoL). It included 41 TDT patients, who failed a reasonable trial of hydroxyurea. Complete response (CR) was defined as transfusion independence and partial response (PR) denoted at least a 50% reduction in transfusion requirement. The rest were defined as non-responders (NR). The mean age of the cohort was 20.78 years (range 12-45 years). There were 13 males and 28 females. Nineteen (46.3%), 7 (17.1%), and 15 (36.6%) patients achieved CR, PR, and no response respectively. The overall response rate (CR + PR) was 63.4%. There was a significant increase in hemoglobin levels with decrement in transfusion burden and ferritin levels. There were no significant adverse reactions. No significant predictors of response were found including amongst genetic modifiers. It improved the health related QoL amongst responders. The combination of thalidomide and hydroxyurea appear safe and effective in the reduction in transfusion requirement of TDT patients. The judicious use of these drugs can improve the quality of life and pave the way for patients not eligible for a stem cell transplant.

沙利度胺和羟基脲治疗输血依赖型地中海贫血症:疗效、安全性和对生活质量的影响。
输血依赖型地中海贫血症(TDT)是印度的一大公共卫生问题,患者需要定期输血才能存活。铁超载和输血相关感染也会导致严重的发病率。对于因各种原因无法进行移植的患者,资源匮乏的医疗机构迫切需要以胎儿血红蛋白诱导为目标的新型疗法。这项单臂、非随机前瞻性试验评估了低剂量沙利度胺和羟基脲联合治疗 TDT 的疗效和安全性,以及对生活质量(QoL)的影响。该试验纳入了 41 名未能通过羟基脲合理试验的 TDT 患者。完全应答(CR)的定义是不需要输血,部分应答(PR)的定义是输血需求至少减少 50%。其余患者被定义为无应答者(NR)。组群的平均年龄为 20.78 岁(12-45 岁不等)。其中男性 13 人,女性 28 人。分别有 19 名(46.3%)、7 名(17.1%)和 15 名(36.6%)患者达到 CR、PR 和无应答。总体反应率(CR + PR)为 63.4%。血红蛋白水平明显上升,输血负担和铁蛋白水平下降。没有出现明显的不良反应。没有发现明显的反应预测因素,包括基因修饰因子。该疗法改善了应答者的健康相关生活质量。沙利度胺和羟基脲联合用药在减少 TDT 患者的输血需求方面似乎安全有效。合理使用这些药物可提高生活质量,并为不符合干细胞移植条件的患者铺平道路。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Hemoglobin
Hemoglobin 医学-生化与分子生物学
CiteScore
1.70
自引率
10.00%
发文量
59
审稿时长
3 months
期刊介绍: Hemoglobin is a journal in the English language for the communication of research and information concerning hemoglobin in humans and other species. Hemoglobin publishes articles, reviews, points of view The journal covers topics such as: structure, function, genetics and evolution of hemoglobins biochemical and biophysical properties of hemoglobin molecules characterization of hemoglobin disorders (variants and thalassemias), consequences and treatment of hemoglobin disorders epidemiology and prevention of hemoglobin disorders (neo-natal and adult screening) modulating factors methodology used for diagnosis of hemoglobin disorders
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