Bhattacharjee Sukrita, Ghosh Shouriyo, Shaw Jyoti, Bhattacharjee Sunistha, Bhattacharyya Maitreyee
Institute of Haematology and Transfusion Medicine, Medical College and Hospital, Kolkata, India.
Tata Medical Center, Clinical Haematology and Cellular Therapies, Kolkata, India.
Hemoglobin. 2024 May;48(3):161-168. doi: 10.1080/03630269.2024.2386076. Epub 2024 Aug 2.
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.
输血依赖型地中海贫血（TDT）是印度主要的公共卫生问题，患者需要定期输血以维持生命。铁过载和输血相关感染也会导致严重的发病情况。对于因各种原因无法进行移植的患者，在资源匮乏的机构中，急需能够诱导胎儿血红蛋白生成的新型疗法。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用药对TDT患者的疗效和安全性，以及对生活质量（QoL）的影响。该试验纳入了41例对羟基脲进行过合理试验但治疗失败的TDT患者。完全缓解（CR）定义为无需输血，部分缓解（PR）表示输血需求至少减少50%。其余患者定义为无反应者（NR）。该队列的平均年龄为20.78岁（范围12 - 45岁）。其中男性13例，女性28例。分别有19例（46.3%）、7例（17.1%）和15例（36.6%）患者达到CR、PR和无反应。总缓解率（CR + PR）为63.4%。血红蛋白水平显著升高，输血负担和铁蛋白水平降低。未发现明显的不良反应。未发现包括基因修饰因子在内的显著反应预测因素。该联合用药改善了有反应患者的健康相关生活质量。沙利度胺和羟基脲联合用药在降低TDT患者输血需求方面似乎安全有效。合理使用这些药物可以改善生活质量，为不符合干细胞移植条件的患者铺平道路。
