Jordan Lori C, Roberts Williams Dionna O, Rodeghier Mark J, Covert Greene Brittany V, Ponisio Maria R, Casella James F, McKinstry Robert C, Noetzel Michael J, Kirkham Fenella J, Meier Emily R, Fuh Beng, McNaull Melissa, Sarnaik Sharada, Majumdar Suvankar, McCavit Timothy L, DeBaun Michael R
Department of Pediatrics, Division of Pediatric Neurology, Vanderbilt University Medical Center, Nashville, Tennessee.
Department of Pediatrics, Division of Hematology/Oncology, Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Vanderbilt University Medical Center, Nashville, Tennessee.
Am J Hematol. 2018 Jun;93(6):760-768. doi: 10.1002/ajh.25085. Epub 2018 Mar 30.
In a prospective cohort study, we tested the hypothesis that children with sickle cell anemia (SCA) with normal transcranial Doppler ultrasound (TCD) velocities and without silent cerebral infarcts (SCIs) would have a lower incidence rate of new neurological events (strokes, seizures or transient ischemic attacks) compared to children with normal TCD measurements and SCIs, not receiving regular blood transfusions. Nonrandomized participants from the silent cerebral infarct transfusion (SIT) Trial who had screening magnetic resonance imaging (MRI) of the brain and normal TCD measurements were included. Follow-up ended at the time of first neurological event (stroke, seizure or transient ischemic attack), start of regular blood transfusion, or loss to follow-up, whichever came first. The primary endpoint was a new neurological event. Of 421 participants included, 68 had suspected SCIs. Mean follow-up was 3.6 years. Incidence rates of new neurological events in nontransfused participants with normal TCD values with SCIs and without SCIs were 1.71 and 0.47 neurological events per 100 patient-years, respectively, P = .065. The absence of SCI(s) at baseline was associated with a decreased risk of a new neurological event (hazard ratio 0.231, 95% CI 0.062-0.858; P = .029). Local pediatric neurologists examined 67 of 68 participants with suspected SCIs and identified 2 with overt strokes classified as SCIs by local hematologists; subsequently one had a seizure and the other an ischemic stroke. Children with SCA, without SCIs, and normal TCD measurements have a significantly lower rate of new neurological events when compared to those with SCIs and normal TCD measurements. Pediatric neurology assessment may assist risk stratification.
在一项前瞻性队列研究中，我们检验了以下假设：与未接受定期输血、经颅多普勒超声（TCD）测量正常但有无症状脑梗死（SCI）的儿童相比，TCD速度正常且无SCI的镰状细胞贫血（SCA）儿童发生新的神经事件（中风、癫痫或短暂性脑缺血发作）的发生率更低。纳入了来自无症状脑梗死输血（SIT）试验的非随机参与者，这些参与者进行了脑部筛查磁共振成像（MRI）且TCD测量正常。随访在首次神经事件（中风、癫痫或短暂性脑缺血发作）发生时、开始定期输血时或失访时结束，以先发生者为准。主要终点是新的神经事件。在纳入的421名参与者中，68名有疑似SCI。平均随访时间为3.6年。TCD值正常、有SCI和无SCI的未输血参与者中新发神经事件的发生率分别为每100患者年1.71次和0.47次神经事件，P = 0.065。基线时无SCI与新神经事件风险降低相关（风险比0.231，95%可信区间0.062 - 0.858；P = 0.029）。当地儿科神经科医生对68名疑似SCI的参与者中的67名进行了检查，当地血液科医生将其中2名诊断为有明显中风，归类为SCI；随后其中1名发生癫痫，另1名发生缺血性中风。与有SCI且TCD测量正常的儿童相比，无SCI且TCD测量正常的SCA儿童发生新神经事件的发生率显著更低。儿科神经学评估可能有助于风险分层。
