Beneficial Effect of Low Fixed Dose of Hydroxyurea in Vaso-occlusive Crisis and Transfusion Requirements in Adult HbSS Patients: A Prospective Study in a Tertiary Care Center
Beneficial Effect of Low Fixed Dose of Hydroxyurea in Vaso-occlusive Crisis and Transfusion Requirements in Adult HbSS Patients: A Prospective Study in a Tertiary Care Center
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低固定剂量羟基脲对成人 HbSS 患者血管闭塞危机和输血需求的有益影响：三级护理中心的前瞻性研究
DOI：
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发表时间：
2018
影响因子：
0.9
通讯作者：
R. Jena
中科院分区：
文献类型：
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作者：
Sudha Sethy;Tribikram Panda;R. Jena
大多数使用25-35 mg/kg/天剂量或最大耐受剂量的羟基脲的研究记录了镰状细胞病（HbSS）患者的发病率和死亡率显著降低。但毒性，需要频繁监测，合规性和成本是重要的障碍，特别是在印度的设置。我们进行了这项研究，以了解低固定剂量羟基脲（10 mg/kg/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。前瞻性评估了2013年至2016年期间128例（82例男性，46例女性）确诊的HbSS病例（均>18岁，血管闭塞危象>2/年和/或输血率1-2单位/月），无疾病相关终末器官损伤。他们开始接受10 mg/kg/天的羟基脲沿着其他支持性治疗，每月随访一次，持续1年。对治疗前后的临床和实验室指标进行了回顾和比较。在92%的反复出现血管闭塞危象的病例中，VOC在随访期间完全消失，8%的病例中，我们发现其严重程度和发作频率显著降低（p < 0.01）。87%的患者在随访期间不再需要输血，13%的患者进一步降低了输血率（p < 0.01）。VOC的中位缓解时间为3个月，输血需求为5个月。胆红素葡萄球菌、乳酸脱氢酶葡萄球菌、疾病相关并发症和住院率也显著降低，Hb、MCV和MCH显著改善。HbF升高不明显，中位值为（1.5-2.4）%，5例> 5%。在研究期间，我们没有发现任何明显的药物不良反应。低固定剂量的羟基脲（10 mg/kg/天）有益于降低成人HbSS患者（阿拉伯-印度单倍型）的血管闭塞危象和输血需求。它是安全的，合适的，是一种有效的治疗模式，在资源贫乏的环境，如印度。
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 (p < 0.01). Again in 87%, no further transfusion was required during follow up and in 13%, it further reduced the rate of transfusion (p < 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.
羟基脲对狗和镰状细胞性贫血患者红细胞中血红蛋白 F 和水含量的影响。
影响因子：
20.3
作者：
Orringer,EP;Blythe,DS;Johnson,AE;PhillipsJr,G;Dover,GJ;Parker,JC
通讯作者：
Parker,JC
影响因子：
158.5
作者：
CHARACHE, S;TERRIN, ML;WACLAWIW, M
通讯作者：
WACLAWIW, M