Sethy Sudha, Panda Tribikram, Jena Rabindra Kumar
1Department of Clinical Hematology, S.C.B Medical College and Hospital, Cuttack, India.
2P.G Department of General Medicine, S.C.B Medical College and Hospital, Cuttack, India.
Indian J Hematol Blood Transfus. 2018 Apr;34(2):294-298. doi: 10.1007/s12288-017-0869-x. Epub 2017 Sep 4.
Significant reduction in morbidity and mortality have been documented in patients with sickle cell disease (HbSS) by most of the studies using hydroxyurea at a dose of 25-35 mg/kg/day or maximum tolerated dose. But toxicities, need for frequent monitoring, compliance and cost are important hurdles particularly in Indian set up. We undertook this study to find out the efficacy, safety compliance rate of low fixed dose of hydroxyurea (10 mg/kg/day) in patients presenting to our hospital and its impact on clinical profile and laboratory parameters. A cohort of 128 (82 males, 46 females) confirmed HbSS cases (each >18 years age, vaso-occlusive crisis >2/years and/ or rate of transfusion 1-2 units/month) with no disease related end organ damage were assessed prospectively between 2013 and 2016. They were started on 10 mg/kg/day hydroxyurea along with other supportive care and followed up monthly for 1 year. Clinical and laboratory parameters before and after therapy were reviewed and compared. In 92% of cases presenting with repeated vaso-occlusive crisis, VOC disappeared completely during follow up and in 8% we found significant reduction in severity as well as frequency of attacks ( < 0.01). Again in 87%, no further transfusion was required during follow up and in 13%, it further reduced the rate of transfusion ( < 0.01). The median time of response for VOC was 3 months and in transfusion requirement was 5 months. There was also significant reduction in S.Billirubin, S.LDH, disease related complications and rate of hospitalisation with significant improvement in Hb, MCV, and MCH. There is insignificant increase in HbF with median (1.5-2.4)% and in 5 cases >5%. We did not find any remarkable adverse effect of the drug during the study period. Low fixed dose hydroxyurea (10 mg/kg/day) is beneficial in reducing the vaso-occlusive crisis and transfusion requirement in adult HbSS Patients (Arab-Indian Haplotype). It is safe, suitable and is a effective mode of treatment in resource poor setting like India.
大多数使用剂量为25 - 35毫克/千克/天或最大耐受剂量的羟基脲的研究表明，镰状细胞病（HbSS）患者的发病率和死亡率显著降低。但毒性、频繁监测的需求、依从性和成本是重要障碍，在印度的环境中尤其如此。我们开展这项研究，以了解低固定剂量羟基脲（10毫克/千克/天）在我院就诊患者中的疗效、安全性、依从率及其对临床特征和实验室参数的影响。2013年至2016年期间，对128例（82例男性，46例女性）确诊的HbSS病例（年龄均>18岁，血管闭塞性危机>2次/年和/或输血率1 - 2单位/月）且无疾病相关终末器官损害的患者进行了前瞻性评估。他们开始服用10毫克/千克/天的羟基脲并接受其他支持性治疗，每月随访1年。回顾并比较了治疗前后的临床和实验室参数。在92%反复出现血管闭塞性危机的病例中，血管闭塞性危机在随访期间完全消失，8%的病例中发作的严重程度和频率显著降低（P<0.01）。同样，87%的病例在随访期间无需进一步输血，13%的病例输血率进一步降低（P<0.01）。血管闭塞性危机的中位反应时间为3个月，输血需求的中位反应时间为5个月。血清胆红素、血清乳酸脱氢酶、疾病相关并发症和住院率也显著降低，血红蛋白、平均红细胞体积和平均红细胞血红蛋白含量显著改善。胎儿血红蛋白中位数（1.5 - 2.4）%略有增加，5例患者>5%。在研究期间，我们未发现该药物有任何明显的不良反应。低固定剂量羟基脲（10毫克/千克/天）有利于降低成年HbSS患者（阿拉伯 - 印度单倍型）的血管闭塞性危机和输血需求。在印度这样资源匮乏的环境中，它是安全、合适且有效的治疗方式。
