Impact of hydroxyurea on clinical events in the BABY HUG trial
Impact of hydroxyurea on clinical events in the BABY HUG trial
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DOI：
10.1182/blood-2012-03-419879
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发表时间：
2012-11-22
期刊：
影响因子：
20.3
通讯作者：
Wang, Winfred C.
中科院分区：
文献类型：
--
作者：
Thornburg, Courtney D.;Files, Beatrice A.;Wang, Winfred C.
小儿羟基脲III期临床试验（BABY HUG）是一项在镰状细胞贫血婴儿（9-18月龄开始）中进行的羟基脲III期多中心、随机、双盲、安慰剂对照临床试验。本研究的一个重要次要目的是比较羟基脲组和安慰剂组之间的临床事件。193例受试者被随机分配至羟基脲（20 mg/kg/d）或安慰剂组;研究期间观察值为374患者-年。羟基脲与疼痛、指炎、急性胸部综合征和住院的初始和复发率在统计学上显着降低相关;即使是在入组时无症状的婴儿，如果接受羟基脲治疗，指炎也较少，住院和输血也较少。尽管有预期的轻度骨髓抑制，但羟基脲与菌血症或严重感染的风险增加无关。这些数据提供了重要的安全性和有效性信息，临床医生考虑羟基脲治疗非常年幼的儿童镰状细胞贫血。该临床试验在美国国立卫生研究院注册（NCT 00006400，www.clinicaltrials.gov）。（血。2012;120（22）：4304-4310）
The Pediatric Hydroxyurea Phase 3 Clinical Trial (BABY HUG) was a phase 3 multicenter, randomized, double-blind, placebo-controlled clinical trial of hydroxyurea in infants (beginning at 9-18 months of age) with sickle cell anemia. An important secondary objective of this study was to compare clinical events between the hydroxyurea and placebo groups. One hundred and ninety-three subjects were randomized to hydroxyurea (20 mg/kg/d) or placebo; there were 374 patient-years of on-study observation. Hydroxyurea was associated with statistically significantly lower rates of initial and recurrent episodes of pain, dactylitis, acute chest syndrome, and hospitalization; even infants who were asymptomatic at enrollment had less dactylitis as well as fewer hospitalizations and transfusions if treated with hydroxyurea. Despite expected mild myelosuppression, hydroxyurea was not associated with an increased risk of bacteremia or serious infection. These data provide important safety and efficacy information for clinicians considering hydroxyurea therapy for very young children with sickle cell anemia. This clinical trial is registered with the National Institutes of Health (NCT00006400, www.clinicaltrials.gov). (Blood. 2012;120(22):4304-4310)