Adams Robert J, Brambilla Donald J, Granger Suzanne, Gallagher Dianne, Vichinsky Elliott, Abboud Miguel R, Pegelow Charles H, Woods Gerald, Rohde Elizabeth M, Nichols Fenwick T, Jones Anne, Luden Judith P, Bowman Latonya, Hagner Susan, Morales Knashawn H, Roach E Steve
Department of Neurology, Medical College of Georgia, Augusta, GA 30912-3200, USA.
Blood. 2004 May 15;103(10):3689-94. doi: 10.1182/blood-2003-08-2733. Epub 2004 Jan 29.
The Stroke Prevention Trial in Sickle Cell Anemia (STOP) was a randomized multicenter controlled trial comparing prophylactic blood transfusion with standard care in sickle cell anemia (SCA) children aged 2 to 16 years selected for high stroke risk by transcranial Doppler (TCD). More than 2000 children were screened with TCD to identify the 130 high-risk children who entered the randomized trial. A total of 5613 TCD studies from 2324 children were evaluated. We also collected information on stroke. We describe the changes in TCD with repeated testing and report the outcome without transfusion in the STOP screened cohort. Risk of stroke was higher with abnormal TCD than with normal or conditional TCD (P <.001) or inadequate TCD (P =.002), and risk with conditional TCD was higher than with normal TCD (P <.001). Repeated TCD in 1215 children showed that the condition of 9.4% of children became abnormal during observation. Younger patients and those with higher initial flow velocities were most likely to convert to abnormal TCDs. Screening in STOP confirmed the predictive value of TCD for stroke. Substantial differences in the probability of conversion to abnormal TCD were observed, with younger children and those with higher velocity more likely to have an abnormal TCD with rescreening.
镰状细胞贫血症预防中风试验(STOP)是一项随机多中心对照试验,比较了预防性输血与标准治疗方案对年龄在2至16岁、经经颅多普勒(TCD)检查确定为中风高危的镰状细胞贫血症(SCA)儿童的疗效。超过2000名儿童接受了TCD筛查,以确定130名进入随机试验的高危儿童。共评估了来自2324名儿童的5613次TCD检查。我们还收集了中风相关信息。我们描述了重复检查时TCD的变化,并报告了STOP筛查队列中未接受输血的结果。TCD异常者的中风风险高于TCD正常或条件性正常者(P<.001)或TCD检查不充分者(P =.002),条件性正常TCD者的中风风险高于正常TCD者(P<.001)。对1215名儿童进行的重复TCD检查显示,9.4%的儿童在观察期间TCD状况变为异常。年龄较小的患者和初始血流速度较高的患者最有可能转变为TCD异常。STOP试验中的筛查证实了TCD对中风的预测价值。观察到转变为TCD异常的概率存在显著差异,年龄较小及血流速度较高的儿童再次筛查时更有可能出现TCD异常。