Effectiveness and Safety of Hydroxyurea in the Treatment of Sickle Cell Anaemia Children in Jos, North Central Nigeria
Effectiveness and Safety of Hydroxyurea in the Treatment of Sickle Cell Anaemia Children in Jos, North Central Nigeria
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DOI：
10.1093/tropej/fmz070
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发表时间：
2020-06-01
影响因子：
2
通讯作者：
Sagay, Atiene S.
中科院分区：
文献类型：
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作者：
Ofakunrin, Akinyemi O. D.;Oguche, Stephen;Sagay, Atiene S.
背景：羟基尿素已被证明可以积极地改变镰状细胞病的发病机制，但由于有效性和安全性的考虑，其在尼日利亚镰状细胞性贫血(SCA)患者中的使用率较低。方法：我们进行了一项准实验研究，以评估羟基尿素在54名4-17岁的SCA儿童中的有效性和安全性。比较基线和羟基尿素治疗后12个月的临床和血液学指标。参与者被监测不良事件。结果：发生两次以上疼痛危象的受试者从27例(50%)减少到2例(2.7%)(P<；0.001)，而有急性胸部综合征的受试者从6例(11.1%)减少到0例(0.0%；P<；0.001)。多次输血的风险是治疗前12个月风险的0.11倍(95%CI=0.02~0.85；P=0.016)。同样，住院7天的风险是基线风险的0.08倍(95%CI=0.02-0.24；p<；0.0001)。中位数红细胞压积和胎儿血红蛋白百分比分别从26%增加到28%和7.8%增加到14%(p<；0.0001)。在6名儿童(11.1%)中观察到剂量依赖性但可逆的白细胞减少症，否则羟基尿素在研究人群中是安全的。结论：羟基尿素对尼日利亚乔斯的SCA儿童是有效和安全的。这一发现可能会加强旨在改善SCA儿童对羟基尿素的利用的教育计划。
Background: Hydroxyurea has been shown to positively modify sickle cell disease pathogenesis, but its use is low among Nigerian sickle cell anaemia (SCA) patients because of effectiveness and safety concerns.Methods: We conducted a quasi-experimental study to evaluate the effectiveness and safety of hydroxyurea in 54 SCA children aged 4-17 years. Clinical and haematological parameters were compared at baseline and 12 months after hydroxyurea therapy. The participants were monitored for adverse events. The parameters were compared using relative risk and Wilcoxon Signed-Rank Test.Results: The number of subjects who had more than two episodes of painful crises reduced from 27 (50%) to 2 (2.7%) (p < 0.001), while those who had acute chest syndrome reduced from 6 (11.1%) to 0 (0.0%; p < 0.001). The risk of being transfused more than once was 0.11 times the risk in the 12 months period preceding therapy (95% CI = 0.02-0.85; p = 0.016). Similarly, the risk of hospital stay >7 days was 0.08 times the risk at the baseline (95% CI = 0.02-0.24; p < 0.0001). The median haematocrit and percentage foetal haemoglobin increased from 26 to 28% and 7.8 to 14%, respectively (p < 0.0001). A dose-dependent but reversible leucopenia was observed among six children (11.1%), otherwise, hydroxyurea was safe in the study population.Conclusion: Hydroxyurea is effective and safe in SCA children in Jos, Nigeria. The findings could strengthen educational programme aimed at improving the utilization of hydroxyurea among SCA children.