镰状细胞贫血症无症状脑梗死输血对照试验。
Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia.
作者信息
DeBaun Michael R, Gordon Mae, McKinstry Robert C, Noetzel Michael J, White Desiree A, Sarnaik Sharada A, Meier Emily R, Howard Thomas H, Majumdar Suvankar, Inusa Baba P D, Telfer Paul T, Kirby-Allen Melanie, McCavit Timothy L, Kamdem Annie, Airewele Gladstone, Woods Gerald M, Berman Brian, Panepinto Julie A, Fuh Beng R, Kwiatkowski Janet L, King Allison A, Fixler Jason M, Rhodes Melissa M, Thompson Alexis A, Heiny Mark E, Redding-Lallinger Rupa C, Kirkham Fenella J, Dixon Natalia, Gonzalez Corina E, Kalinyak Karen A, Quinn Charles T, Strouse John J, Miller J Philip, Lehmann Harold, Kraut Michael A, Ball William S, Hirtz Deborah, Casella James F
机构信息
From the Department of Pediatrics, Division of Hematology-Oncology, Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Vanderbilt University School of Medicine, Nashville (M.R.D.); Department of Ophthalmology and Visual Sciences, Division of Biostatistics (M.G.), Departments of Radiology and Pediatrics (R.C.M.), Neurology and Pediatrics (M.J.N.), and Psychology (D.A.W.), the Program in Occupational Therapy and Department of Pediatrics Hematology-Oncology (A.A.K.), and the Division of Biostatistics and Department of Internal Medicine (J.P.M.), Washington University School of Medicine, St. Louis; Department of Pediatrics, Division of Hematology-Oncology, Wayne State University, Detroit (S.A.S.); Center for Cancer and Blood Disorders, Children's National Medical Center, Department of Pediatrics, George Washington University Medical Center (E.R.M.), and Department of Pediatrics, Division of Hematology-Oncology, Georgetown University Hospital (C.E.G.) - all in Washington, DC; Department of Pediatrics, Division of Hematology-Oncology, University of Alabama at Birmingham, Birmingham (T.H.H.); Department of Pediatrics, Division of Hematology-Oncology, University of Mississippi Medical Center, Jackson (S.M.); Department of Paediatrics, Evelina Children's Hospital, St. Thomas' Hospital NHS Trust (B.P.D.I.), Department of Pediatric Hematology, Royal London Hospital, Barts Health NHS Trust (P.T.T.), and the Neurosciences Unit, Institute of Child Health, University College London (F.J.K.) - all in London; Hospital for Sick Children, Department of Paediatrics, Haematology-Oncology, University of Toronto, Toronto (M.K.-A.); Division of Hematology-Oncology, Department of Pediatrics, UT Southwestern Medical Center, Dallas (T.L.M.C.); Département Pédiatrie, Hôpital Intercommunal de Creteil, Creteil, France (A.K.); Department of Pediatrics, Division of Hematology-Oncology, Baylor College of Medicine, Houston (G.A.); Department of Pediatrics, Hematology-Oncology, Univer
出版信息
N Engl J Med. 2014 Aug 21;371(8):699-710. doi: 10.1056/NEJMoa1401731.
BACKGROUND
Silent cerebral infarcts are the most common neurologic injury in children with sickle cell anemia and are associated with the recurrence of an infarct (stroke or silent cerebral infarct). We tested the hypothesis that the incidence of the recurrence of an infarct would be lower among children who underwent regular blood-transfusion therapy than among those who received standard care.
METHODS
In this randomized, single-blind clinical trial, we randomly assigned children with sickle cell anemia to receive regular blood transfusions (transfusion group) or standard care (observation group). Participants were between 5 and 15 years of age, with no history of stroke and with one or more silent cerebral infarcts on magnetic resonance imaging and a neurologic examination showing no abnormalities corresponding to these lesions. The primary end point was the recurrence of an infarct, defined as a stroke or a new or enlarged silent cerebral infarct.
RESULTS
A total of 196 children (mean age, 10 years) were randomly assigned to the observation or transfusion group and were followed for a median of 3 years. In the transfusion group, 6 of 99 children (6%) had an end-point event (1 had a stroke, and 5 had new or enlarged silent cerebral infarcts). In the observation group, 14 of 97 children (14%) had an end-point event (7 had strokes, and 7 had new or enlarged silent cerebral infarcts). The incidence of the primary end point in the transfusion and observation groups was 2.0 and 4.8 events, respectively, per 100 years at risk, corresponding to an incidence rate ratio of 0.41 (95% confidence interval, 0.12 to 0.99; P=0.04).
CONCLUSIONS
Regular blood-transfusion therapy significantly reduced the incidence of the recurrence of cerebral infarct in children with sickle cell anemia. (Funded by the National Institute of Neurological Disorders and Stroke and others; Silent Cerebral Infarct Multi-Center Clinical Trial ClinicalTrials.gov number, NCT00072761, and Current Controlled Trials number, ISRCTN52713285.).
背景
无症状脑梗死是镰状细胞贫血患儿最常见的神经损伤，且与梗死（中风或无症状脑梗死）复发相关。我们检验了这样一个假设，即接受定期输血治疗的儿童梗死复发率低于接受标准治疗的儿童。
方法
在这项随机、单盲临床试验中，我们将镰状细胞贫血患儿随机分为接受定期输血治疗组（输血组）和标准治疗组（观察组）。参与者年龄在5至15岁之间，无中风病史，磁共振成像显示有一处或多处无症状脑梗死，且神经系统检查未发现与这些病变对应的异常。主要终点是梗死复发，定义为中风或新出现或扩大的无症状脑梗死。
结果
共有196名儿童（平均年龄10岁）被随机分配至观察组或输血组，中位随访时间为3年。输血组99名儿童中有6名（6%）发生终点事件（1名中风，5名出现新的或扩大的无症状脑梗死）。观察组97名儿童中有14名（14%）发生终点事件（7名中风，7名出现新的或扩大的无症状脑梗死）。输血组和观察组每100年的风险中主要终点事件发生率分别为2.0和4.8次，发生率比为0.41（95%置信区间，0.12至0.99；P = 0.04）。
结论
定期输血治疗显著降低了镰状细胞贫血患儿脑梗死复发率。（由美国国立神经疾病和中风研究所等资助；无症状脑梗死多中心临床试验，ClinicalTrials.gov编号，NCT00072761，以及当前对照试验编号，ISRCTN52713285。）
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