Primary Stroke Prevention in Nigerian Children With Sickle Cell Disease (SPIN): Challenges of Conducting a Feasibility Trial
Primary Stroke Prevention in Nigerian Children With Sickle Cell Disease (SPIN): Challenges of Conducting a Feasibility Trial
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DOI：
10.1002/pbc.25289
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发表时间：
2015-03-01
影响因子：
3.2
通讯作者：
DeBaun, Michael R.
中科院分区：
文献类型：
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作者：
Galadanci, Najibah A.;Abdullahi, Shehu U.;DeBaun, Michael R.
大多数患有镰状细胞病（SCD）的儿童（约75%）出生在撒哈拉以南非洲。对于经颅多普勒（TCD）速度升高的儿童，定期输血治疗用于初级卒中预防是高收入国家的标准治疗，但在撒哈拉以南非洲并不可行。在美国国立卫生研究院（NIH）在撒哈拉以南非洲发起的第一个SCD临床试验中，我们描述了在该地区开展临床试验的方案和独特的挑战。我们正在对大脑中动脉TCD速度为200cm/sec的儿童进行羟基脲治疗的单臂先导试验。符合条件的儿童将在尼日利亚Aminu Kano教学医院接受羟脲治疗（n=40），随访3年。依从性将通过Morisky量表来衡量，不良事件将根据住院情况来确定。结果最初计划进行随机安慰剂试验；然而，安慰剂并没有得到当地伦理委员会的批准。因此，将进行羟基脲单臂试验，并对每名TCD测量正常的患者进行5名对照，以比较服用羟基脲的TCD测量异常患者的不良事件发生率。使用非nih资助，在9个多月的时间里，进行了多次面对面的研究者会议，以促进培训。结论羟脲治疗儿童SCD的试验（NCT01801423）在撒哈拉以南非洲地区是可行的；然而，需要广泛的培训和资源来建立一个具有共同目标的以患者为导向的全球多学科研究团队。儿科血癌2015；62:395-401。(c) 2014 Wiley期刊公司
BackgroundThe majority of children with sickle cell disease (SCD), approximately 75%, are born in sub-Saharan Africa. For children with elevated transcranial Doppler (TCD) velocity, regular blood transfusion therapy for primary stroke prevention is standard care in high income countries, but is not feasible in sub-Saharan Africa.ProcedureIn the first U.S. National Institute of Health (NIH) sponsored SCD clinical trial in sub-Saharan Africa, we describe the protocol and challenges unique to starting a clinical trial in this region. We are conducting a single arm pilot trial of hydroxyurea therapy in children with TCD velocity 200cm/sec in the middle cerebral arteries. Eligible children will be placed on hydroxyurea (n=40) and followed for 3 years at Aminu Kano Teaching Hospital, Nigeria. Adherence will be measured via the Morisky Scale and adverse events will be determined based on hospitalization.ResultsOriginally, a randomized placebo trial was planned; however, placebo was not approved by the local Ethics Committee. Hence a single arm trial of hydroxyurea will be conducted and five controls per patient with normal TCD measurements will be followed to compare the rate of adverse events to those with abnormal TCD measurements taking hydroxyurea. Using non-NIH funding, over 9 months, multiple face-to-face investigator meetings were conducted to facilitate training.ConclusionA hydroxyurea trial (NCT01801423) for children with SCD is feasible in sub-Saharan Africa; however, extensive training and resources are needed to build a global patient oriented multi-disciplinary research team with a common purpose. Pediatr Blood Cancer 2015;62:395-401. (c) 2014 Wiley Periodicals, Inc.