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低剂量羟基脲可有效降低印度东部镰状细胞贫血患者疼痛性危象的发生率和输血频率。

Low dose hydroxyurea is effective in reducing the incidence of painful crisis and frequency of blood transfusion in sickle cell anemia patients from eastern India.

作者信息

Patel Dilip K, Mashon Ranjeet S, Patel Siris, Das Bhabani S, Purohit Prasanta, Bishwal Subasa C

机构信息

Department of Medicine, Veer Surendra Sai Medical College, Burla, Orissa, India.

出版信息

Hemoglobin. 2012;36(5):409-20. doi: 10.3109/03630269.2012.709897.

Abstract

There are several questions pertaining to dosage, duration and potential long-term toxicity of hydroxyurea (HU) therapy. Use of HU is extremely limited in eastern India because of its high cost and apprehension of its toxicities. We undertook this study to assess the clinical, biochemical and hematological efficacy of minimal dose HU (10 mg/kg/day) in 118 sickle cell anemia patients (27 pediatric and 91 adults). The frequency of painful crises reduced significantly in 71.5 and 92.2% in pediatric and adult cases, respectively. Ninety-five percent of the patients became transfusion independent. The baseline Hb F, total hemoglobin (Hb), MCV, MCH and MCHC levels increased significantly, whereas the WBC, platelet count and total serum bilirubin values decreased significantly. This is the first study of minimal dose HU therapy in eastern India that showed impressive improvement in clinical and hematological parameters with minimal toxicity.

摘要

关于羟基脲(HU)治疗的剂量、疗程及潜在长期毒性存在若干问题。由于成本高昂且担心其毒性,HU在印度东部的使用极为有限。我们开展了这项研究,以评估最小剂量HU(10毫克/千克/天)对118例镰状细胞贫血患者(27例儿童和91例成人)的临床、生化及血液学疗效。儿童和成人病例中,疼痛性危象的发生频率分别显著降低了71.5%和92.2%。95%的患者不再依赖输血。基线时的胎儿血红蛋白(Hb F)、总血红蛋白(Hb)、平均红细胞体积(MCV)、平均红细胞血红蛋白含量(MCH)和平均红细胞血红蛋白浓度(MCHC)水平显著升高,而白细胞、血小板计数及总血清胆红素值显著降低。这是印度东部首次关于最小剂量HU治疗的研究,该研究显示临床和血液学参数有显著改善且毒性最小。

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