NEJM:同时对抗镰状细胞贫血和疟疾!这款药物可有效作用于非洲儿童

2018-12-04 朱玲 生物探索

镰状细胞贫血(SCA)是一种由基因突变引起的疾病,在非洲血统的人中尤为普遍。目前治疗SCA的疗法有限,尽管近年来大量研究表明,对于控制SCA和提高患者生活质量,羟基脲是一种安全有效的药物,但是这些研究大都集中在发达国家。

镰状细胞贫血(SCA)是一种由基因突变引起的疾病,在非洲血统的人中尤为普遍。目前治疗SCA的疗法有限,尽管近年来大量研究表明,对于控制SCA和提高患者生活质量,羟基脲是一种安全有效的药物,但是这些研究大都集中在发达国家。

近日,发表在权威杂志《NEJM》上的一项研究或带来了新的希望。研究表明,每天服用羟基脲药丸或可改善撒哈拉以南非洲患有SCA的儿童的症状,并且能减少疟疾的发生。

根据这项最新的研究, 共606名1-10岁的儿童接受了每日羟基脲治疗。6个月来,孩子们每天服用一颗药丸,然后根据他们的健康状况和体重变化增加每日剂量。初始剂量在每天15-20mmg/kg之间。

临床试验确定,羟基脲疗法对撒哈拉以南非洲的儿童来说是可行和安全的。让人意外的是,研究人员发现羟基脲还能帮助对抗疟疾。众所周知,疟疾在非洲是非常普遍的。


通讯作者:Russell Ware

而研究数据显示,与治疗前水平相比,羟基脲的使用与SCA患者疼痛平均降低55 %,感染降低38 %,疟疾降低51%,输血降低67%,死亡率降低70 %有关。

据悉,该临床试验的I-II期已治疗了 4个撒哈拉以南国家的儿童。研究人员计划长期跟踪这些儿童,以获得有关生长发育的其他数据,并寻找对器官功能和生育能力的任何可能影响。

原始出处:Léon Tshilolo, George Tomlinson, Thomas N. Williams, et al. Hydroxyurea for Children with Sickle Cell Anemia in Sub-Saharan Africa. NEJM. December 1, 2018

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    2018-12-04 盖伦

    厉害了,又学到了一个新的知识点

    0

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    2018-12-04 衣带渐宽

    学习

    0

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    2018-12-04 深海的鱼

    学习学习学习

    0

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