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首页> 外文期刊>Pediatric blood & cancer >Real‐world experience using hydroxyurea in children with sickle cell disease in Lilongwe, Malawi
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Real‐world experience using hydroxyurea in children with sickle cell disease in Lilongwe, Malawi

机译：利龙圭，马拉维患有镰状细胞疾病的儿童羟基脲的现实世界经验

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Abstract Introduction Sickle cell disease (SCD) is among the most common inherited hematologic diseases in sub‐Saharan Africa (SSA). Historically, hydroxyurea administration in SSA has been restricted due to limited region‐specific evidence for safety and efficacy. Methods We conducted a prospective observational cohort study of pediatric patients with SCD in Malawi. From January 2015 to November 2017, hydroxyurea at doses of 10–20?mg/kg/day was administered to children with clinically severe disease (targeted use policy). From December 2017 to July 2018, hydroxyurea was prescribed to all patients (universal use policy). Results Of 187 patients with SCD, seven (3.7%) died and 23 (12.3%) were lost to follow‐up. The majority (135, 72.2%) were prescribed hydroxyurea, 59 (43.7%) under the targeted use policy and 76 (56.3%) under the universal use policy. There were no documented severe toxicities. Under the targeted use policy, children with SCD demonstrated absolute decreases in the rates of hospitalization (?4.1 per 1000 person‐days; ?7.2, ?1.0; P ?=?.004), fevers (?4.2 per 1000 person‐days; ?7.2, ?1.1; P ?=?.002), transfusions (?2.3 per 1000 person‐days; 95% confidence interval: ?4.9, 0.3; P ?=?.06), and annual school absenteeism (?51.2 per person‐year; ?60.1, ?42.3; P ??.0001) within 6 months of hydroxyurea commencement. Conclusion We successfully implemented universal administration of hydroxyurea to children with SCD at a tertiary hospital in Malawi. Similar to recently reported trials, hydroxyurea was safe and effective during routine programmatic experience, with clinical benefits particularly among high‐risk children. This highlights the importance of continued widespread scale‐up of hydroxyurea within SCD programs across SSA.
机译：摘要引言镰状细胞病（SCD）是撒哈拉以南非洲（SSA）中最常见的遗传性血液学疾病之一。历史上，由于有限的地区特异性证据，SSA在SSA中的羟基脲管理受到限制的安全性和疗效。方法对马拉维SCD的儿科患者进行了前瞻性观察队列研究。从2015年1月到2017年11月，羟基脲以10-20℃的剂量为10-20？Mg / kg /天的患儿对临床严重疾病（有针对性的使用政策）给予儿童。从2017年12月至2018年7月，羟基脲规定所有患者（普遍使用政策）。结果187例SCD患者，七（3.7％）死亡，23例（12.3％）失去了随访。大多数（135,72.2％）在目标使用政策下规定羟基脲，59（43.7％），普遍使用政策下76（56.3％）。没有记录的严重毒性。在目标使用政策下，SCD的儿童表现出绝对减少住院费率（？每1000人的4.1天;？7.2，？1.0; P？=Δ004），Frees（？4.2每1000人 - 天; ？7.2，？1.1; p？= = 002），输血（？每1000人的2.3天; 95％置信区间：？4.9,0.3; p？=？06）和年度学校缺勤（每年人年;α60.1，？42.3; p？＆ 0001）在羟基脲开始6个月内。结论我们成功地将羟基脲施用与SCD在马拉维第三级医院的儿童施用。与最近报道的试验类似，羟基脲在常规方案经验期间安全有效，特别是在高危儿童中临床效益。这突出了SSA在SCD程序中持续广泛扩大的羟基脲持续扩大的重要性。

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