Long-term safety and efficacy of hydroxyurea in patients with non-transfusion-dependent β-thalassemia: a comprehensive single-center experience
Long-term safety and efficacy of hydroxyurea in patients with non-transfusion-dependent β-thalassemia: a comprehensive single-center experience
复制标题
DOI：
10.1007/s00277-021-04627-4
复制
发表时间：
2021-08-12
影响因子：
3.5
通讯作者：
Haghpanah, Sezaneh
中科院分区：
文献类型：
--
作者：
Karimi, Mehran;Zarei, Tahereh;Haghpanah, Sezaneh
在过去的20年里，羟基脲（HU）已成为地中海贫血患者改善贫血和减少输血依赖的有效药物。我们评估了非输血依赖型β -地中海贫血（NTDT）患者HU的长期安全性和临床反应。在这项回顾性研究中，对2020年10月至12月伊朗南部181例NTDT患者的医疗记录进行了评估。不需要输血被认为是持续的输血独立性反应。所有患者定期检查并监测HU的任何不良事件（AE）的发生。HU消耗+/- SD的平均持续时间为18.2 +/- 4.0（8-22）年。总体而言，149例患者（82.3%）有持续的输血独立性反应。-珠蛋白基因突变和XmnI多态性与临床疗效无显著相关性（P < 0.05）。60例（33%）患者报告了轻度和短暂性不良事件，不需要中断药物治疗。羟基脲8 ~ 15mg /kg剂量可作为NTDT患者安全有效的治疗方法。长期耐受性良好，无严重并发症或继发恶性肿瘤。在该地区未观察到XmnI或β -珠蛋白基因突变与HU反应之间的关系。
Over the past 20 years, hydroxyurea (HU) has emerged as an effective therapeutic agent in thalassemic patients to improve anemia and decrease the transfusion dependency. We evaluated long-term safety and clinical response to HU in patients with non-transfusion-dependent beta-thalassemia (NTDT). In this retrospective study, medical records of 181 patients with NTDT were evaluated during October to December 2020 in Southern Iran. No requirement to blood transfusion was considered as sustained transfusion independence response. All patients were regularly examined and monitored for the occurrence of any adverse event (AE) of HU. The mean duration of HU consumption +/- SD was 18.2 +/- 4.0 (8-22) years. Overall, 149 patients (82.3%) had sustained transfusion independence response. beta-globin gene mutations and XmnI polymorphisms were not significantly associated with clinical response (P > 0.05). Mild and transient AEs were reported in 60 patients (33%) with no requirement to drug interruption. Hydroxyurea with the dose of 8-15 mg/kg can be used as a safe and effective treatment in NTDT patients. It was well tolerated in long term without any serious complication or secondary malignancy. No relationship between XmnI or beta-globin gene mutations with HU response was observed in this geographic area of the world.