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Published on: March 14, 2017
Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia
Michael R DeBaun1, Mae Gordon, Robert C McKinstry
1From 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
Insights
Regular blood transfusions significantly reduced recurrent cerebral infarcts in children with sickle cell anemia. This therapy lowered the risk of stroke and silent infarcts compared to standard care.
Area of Science:
- Neurology
- Pediatrics
- Hematology
Background:
- Silent cerebral infarcts are common in children with sickle cell anemia.
- These infarcts are linked to a higher risk of recurrent cerebrovascular events.
- This study investigated the efficacy of regular blood transfusions in preventing infarct recurrence.
Purpose of the Study:
- To determine if regular blood transfusions reduce the incidence of recurrent infarcts in children with sickle cell anemia.
- To compare the outcomes of regular blood transfusions versus standard care in this pediatric population.
Main Methods:
- A randomized, single-blind clinical trial was conducted.
- Children aged 5-15 with sickle cell anemia and existing silent cerebral infarcts were assigned to either regular blood transfusions or standard care.
- The primary endpoint was the recurrence of an infarct (stroke or new/enlarged silent infarct).
Main Results:
- The study followed 196 children for a median of 3 years.
- Children receiving regular blood transfusions had a significantly lower incidence of recurrent infarcts (6% vs. 14%).
- The incidence rate ratio for recurrent infarcts was 0.41 in favor of the transfusion group (P=0.04).
Conclusions:
- Regular blood-transfusion therapy is effective in reducing the recurrence of cerebral infarcts in children with sickle cell anemia.
- This intervention offers a significant benefit over standard care in preventing further neurological injury.
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.).
