Background

Nigeria has the highest proportion of children with sickle cell anemia (SCA) globally; an estimated 150,000 infants with SCA are born annually. Primary stroke prevention in children with SCA must include Nigeria. We describe capacity-building strategies in conjunction with two National Institutes of Health–funded primary stroke prevention trials (a feasibility trial and phase III randomized controlled trial) with initial hydroxyurea treatment for children with SCA and abnormal transcranial Doppler (TCD) velocities in Nigeria. We anticipated challenges to conducting clinical trials in a low-resource setting with a local team that had not previously been involved in clinical research and sought a sustainable strategy for primary stroke prevention.

Methods

This is a descriptive, prospective study of challenges, solutions, and research teams in two trials that enrolled a total of 679 children with SCA.

Results

As part of the capacity-building component of the trials, over eight years, 23 research personnel (physicians, nurses, research coordinators, a statistician, and a pharmacist) completed a one-month research governance and ethics training program at Vanderbilt University Medical Center, USA. A lead research coordinator for each site completed the Society of Clinical Research Professionals certification. TCD machines were donated; radiologists and nonradiologists were trained and certified to perform TCD. A scalable E-prescription was implemented to track hydroxyurea treatment. We worked with regional government officials to support ongoing TCD-based screening and funding for hydroxyurea for children with SCA at a high risk of stroke.

Conclusions

Our trials and capacity building demonstrate a sustainable strategy to initiate and maintain pediatric SCA primary stroke prevention programs in Africa.

中文翻译：

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非洲镰状细胞性贫血儿童初级中风预防团队的能力建设

背景

尼日利亚是全球镰状细胞性贫血 (SCA) 儿童比例最高的国家；估计每年有 150,000 名患有 SCA 的婴儿出生。SCA 儿童的初级卒中预防必须包括尼日利亚。我们结合美国国立卫生研究院资助的两项初级卒中预防试验（可行性试验和 III 期随机对照试验）描述了能力建设策略，这些试验在尼日利亚对患有 SCA 和异常经颅多普勒 (TCD) 速度的儿童进行了初始羟基脲治疗。我们预料到在资源匮乏的环境中与当地团队进行临床试验的挑战，该团队以前没有参与过临床研究，并寻求可持续的初级卒中预防策略。

方法

这是一项关于挑战、解决方案和研究团队的描述性前瞻性研究，涉及两项试验，共招募了 679 名 SCA 儿童。

结果

作为试验能力建设部分的一部分，八年来，23 名研究人员（医生、护士、研究协调员、统计学家和药剂师）在范德比尔特大学医学中心完成了为期一个月的研究治理和伦理培训计划， 美国。每个站点的首席研究协调员完成了临床研究专业人员协会认证。捐赠TCD机器；放射科医师和非放射科医师都经过培训并获得执行 TCD 的认证。实施了可扩展的电子处方来跟踪羟基脲治疗。我们与地区政府官员合作，支持正在进行的基于 TCD 的羟基脲筛查和资助，以治疗中风风险较高的 SCA 患儿。

结论

我们的试验和能力建设展示了在非洲启动和维持儿科 SCA 初级卒中预防计划的可持续战略。