Galadanci Najibah A, Umar Abdullahi Shehu, Vance Leah D, Musa Tabari Abdulkadir, Ali Shehi, Belonwu Raymond, Salihu Auwal, Amal Galadanci Aisha, Wudil Jibir Binta, Bello-Manga Halima, Neville Kathleen, Kirkham Fenella J, Shyr Yu, Phillips Sharon, Covert Brittany V, Kassim Adetola A, Jordan Lori C, Aliyu Muktar H, DeBaun Michael R
Department of Hematology and Blood Transfusion, Bayero University/Aminu Kano Teaching Hospital, Kano, Nigeria.
Department of Pediatrics, Bayero University/Aminu Kano Teaching Hospital, Kano, Nigeria.
Am J Hematol. 2017 Aug;92(8):780-788. doi: 10.1002/ajh.24770. Epub 2017 Jun 15.
The vast majority of children with sickle cell anemia (SCA) live in Africa, where evidence-based guidelines for primary stroke prevention are lacking. In Kano, Nigeria, we conducted a feasibility trial to determine the acceptability of hydroxyurea therapy for primary stroke prevention in children with abnormal transcranial Doppler (TCD) measurements. Children with SCA and abnormal non-imaging TCD measurements (≥200 cm/s) received moderate fixed-dose hydroxyurea therapy (∼20 mg/kg/day). A comparison group of children with TCD measurements <200 cm/s was followed prospectively. Approximately 88% (330 of 375) of families agreed to be screened, while 87% (29 of 33) of those with abnormal TCD measurements, enrolled in the trial. No participant elected to withdraw from the trial. The average mean corpuscular volume increased from 85.7 fl at baseline to 95.5 fl at 24 months (not all of the children who crossed over had a 24 month visit), demonstrating adherence to hydroxyurea. The comparison group consisted of initially 210 children, of which four developed abnormal TCD measurements, and were started on hydroxyurea. None of the monthly research visits were missed (n = total 603 visits). Two and 10 deaths occurred in the treatment and comparison groups, with mortality rates of 2.69 and 1.81 per 100 patient-years, respectively (P = .67). Our results provide strong evidence, for high family recruitment, retention, and adherence rates, to undertake the first randomized controlled trial with hydroxyurea therapy for primary stroke prevention in children with SCA living in Africa.
绝大多数镰状细胞贫血（SCA）患儿生活在非洲，而当地缺乏基于证据的原发性中风预防指南。在尼日利亚的卡诺，我们开展了一项可行性试验，以确定羟基脲疗法用于预防经颅多普勒（TCD）测量异常的儿童原发性中风的可接受性。患有SCA且非成像TCD测量异常（≥200 cm/s）的儿童接受中等固定剂量的羟基脲治疗（约20 mg/kg/天）。对TCD测量值<200 cm/s的儿童对照组进行前瞻性随访。约88%（375名中的330名）家庭同意接受筛查，而TCD测量异常的家庭中87%（33名中的29名）参加了试验。没有参与者选择退出试验。平均红细胞体积从基线时的85.7 fl增加到24个月时的95.5 fl（并非所有交叉的儿童都进行了24个月的随访），表明对羟基脲治疗的依从性良好。对照组最初有210名儿童，其中4名TCD测量值出现异常，并开始接受羟基脲治疗。没有错过任何一次每月的研究随访（n = 总共603次随访）。治疗组和对照组分别有2例和10例死亡，死亡率分别为每100患者年2.69例和1.81例（P = 0.67）。我们的结果提供了有力证据，表明家庭招募、保留率和依从率很高，从而可以在非洲对患有SCA的儿童进行第一项关于羟基脲疗法预防原发性中风的随机对照试验。
