Hydroxyurea for treatment of severe sickle cell anemia: A pediatric clinical trial
Hydroxyurea for treatment of severe sickle cell anemia: A pediatric clinical trial
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DOI：
10.1182/blood.v88.6.1960.bloodjournal8861960
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发表时间：
1996-09-15
期刊：
影响因子：
20.3
通讯作者：
Sariban, E
中科院分区：
文献类型：
--
作者：
Ferster, A;Vermylen, C;Sariban, E
羟基脲（HU）可促进胎儿血红蛋白（HbF）的合成，并可改善某些成人镰状细胞贫血（SCA）患者的临床病程。在一项随机试验中，我们研究了HU在患有严重SCA的儿童和年轻人中的生物学效应和临床获益。25例患者（中位年龄9岁）随机接受HU（初始剂量为20 mg/kg/d）或安慰剂治疗6个月，然后在接下来的6个月内转换为另一组。在22例可评价患者（中位年龄8岁）中，HU治疗期间HbF和平均红细胞体积显著增加。白色血细胞和网织红细胞计数显著降低，但这些变化无临床相关性。22例患者中有16例（73%）发生了需要住院治疗的事件完全消失。与接受安慰剂的患者相比，接受HU的患者的住院天数和住院次数显著减少。我们的结论是，在儿童和年轻人的治疗与胡是可行的，耐受性良好，并改善SCA的临床过程。HU的长期影响需要进一步研究。(C)1996年，美国血液学会。
Hydroxyurea (HU) enhances the synthesis of fetal hemoglobin (HbF) and can improve the clinical course of some adult patients with sickle cell anemia (SCA). In a randomized trial, we studied the biologic effects and the clinical benefit of HU in children and young adults with severe SCA. Twenty-five patients (median age, 9 years) were randomized to receive either HU (at the initial dosage of 20 mg/kg/d) or a placebo for 6 months and were then switched to the other arm for the next 6 months. Among the 22 evaluable patients (median age, 8 years), significant increases in HbF and mean corpuscular volume occurred during the HU treatment period. The white blood cell and reticulocytes counts decreased significantly, but these changes were not clinically relevant. Sixteen of 22 patients (73%) experienced a complete disappearance of events requiring hospitalization. The number of days of hospitalization as well as the number of hospitalizations for patients on HU, as compared with those for the patients receiving placebo, were significantly reduced. We conclude that treatment with HU in children and young adults is feasible, well-tolerated, and improves the clinical course of SCA. The long-term effects of HU require further investigation. (C) 1996 by The American Society of Hematology.