Jain Dipty L, Sarathi Vijaya, Desai Saumil, Bhatnagar Manoj, Lodha Abhijit
Department of Paediatrics, Government Medical College, Nagpur, Maharsashtra-440009, India.
Hemoglobin. 2012;36(4):323-32. doi: 10.3109/03630269.2012.697948.
There is limited data on the efficacy of hydroxyurea (HU) in Indian sickle cell anemia patients who have severe manifestations despite high fetal hemoglobin (Hb F). Sixty sickle cell anemia children (5-18 years) with more than three episodes of vasoocclusive crises or blood transfusions per year were randomized to receive HU (n = 30) or placebo (n = 30) therapy. Fixed dose (10 mg/kg/day) of HU was administered for 18 months and the patients were followed-up monthly with clinical assessment and laboratory monitoring. In the HU group, hemoglobin (Hb) and Hb F levels increased significantly along with a significant decrease in the number of painful crises, blood transfusion requirements and hospitalizations compared to the placebo group. No major adverse events were observed in this study. In conclusion, low-fixed dose HU therapy was effective for the treatment of Indian sickle cell anemia children. However, there is a need for long-term studies to evaluate the efficacy and toxicity in a larger number of Indian sickle cell anemia patients.
对于患有严重临床表现且胎儿血红蛋白（Hb F）水平较高的印度镰状细胞贫血患者，关于羟基脲（HU）疗效的数据有限。60名镰状细胞贫血儿童（5 - 18岁），每年发生超过三次血管闭塞性危机或输血，被随机分为接受HU治疗组（n = 30）或安慰剂组（n = 30）。给予固定剂量（10 mg/kg/天）的HU治疗18个月，每月对患者进行临床评估和实验室监测随访。与安慰剂组相比，HU组的血红蛋白（Hb）和Hb F水平显著升高，同时疼痛危机次数、输血需求和住院次数显著减少。本研究未观察到重大不良事件。总之，低固定剂量的HU疗法对治疗印度镰状细胞贫血儿童有效。然而，需要进行长期研究以评估大量印度镰状细胞贫血患者的疗效和毒性。
