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Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia

Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia
Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia
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.).
0028-4793
699-710
Debaun, Michael R.
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Gordon, Mae
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Mckinstry, Robert C.
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Noetzel, Michael J.
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Meier, Emily R.
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Howard, Thomas H.
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Majumdar, Suvankar
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Inusa, Baba P.d.
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Telfer, Paul T.
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Kirby-allen, Melanie
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Mccavit, Timothy L.
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Kamdem, Annie
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Kwiatkowski, Janet L.
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King, Allison A.
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Rhodes, Melissa M.
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Thompson, Alexis A.
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Heiny, Mark E.
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Hirtz, Deborah
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Casella, James F.
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Debaun, Michael R.
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Gordon, Mae
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Mckinstry, Robert C.
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Noetzel, Michael J.
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White, Desiree A.
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Sarnaik, Sharada A.
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Howard, Thomas H.
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Majumdar, Suvankar
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Inusa, Baba P.d.
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Telfer, Paul T.
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Kirby-allen, Melanie
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Mccavit, Timothy L.
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Kamdem, Annie
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King, Allison A.
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Rhodes, Melissa M.
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Kirkham, Fenella J.
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Kalinyak, Karen A.
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Quinn, Charles T.
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Miller, J. Philip
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Lehmann, Harold
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Kraut, Michael A.
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Ball, William S.
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Hirtz, Deborah
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Casella, James F.
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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 and Casella, James F. (2014) Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia. New England Journal of Medicine, 371 (8), 699-710. (doi:10.1056/NEJMoa1401731).

Record type: Article

Abstract

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.).

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e-pub ahead of print date: 21 August 2014
Published date: 21 August 2014

Identifiers

Local EPrints ID: 418476
URI: https://eprints.soton.ac.uk/id/eprint/418476
ISSN: 0028-4793
PURE UUID: 485ca5b0-4613-4af8-a115-2918838f4835
ORCID for Fenella J. Kirkham: ORCID iD orcid.org/0000-0002-2443-7958

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Date deposited: 09 Mar 2018 17:30
Last modified: 19 Nov 2019 01:51

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Contributors

Author: Michael R. Debaun
Author: Mae Gordon
Author: Robert C. Mckinstry
Author: Michael J. Noetzel
Author: Desiree A. White
Author: Sharada A. Sarnaik
Author: Emily R. Meier
Author: Thomas H. Howard
Author: Suvankar Majumdar
Author: Baba P.d. Inusa
Author: Paul T. Telfer
Author: Melanie Kirby-allen
Author: Timothy L. Mccavit
Author: Annie Kamdem
Author: Gladstone Airewele
Author: Gerald M. Woods
Author: Brian Berman
Author: Julie A. Panepinto
Author: Beng R. Fuh
Author: Janet L. Kwiatkowski
Author: Allison A. King
Author: Jason M. Fixler
Author: Melissa M. Rhodes
Author: Alexis A. Thompson
Author: Mark E. Heiny
Author: Rupa C. Redding-lallinger
Author: Fenella J. Kirkham ORCID iD
Author: Natalia Dixon
Author: Corina E. Gonzalez
Author: Karen A. Kalinyak
Author: Charles T. Quinn
Author: John J. Strouse
Author: J. Philip Miller
Author: Harold Lehmann
Author: Michael A. Kraut
Author: William S. Ball
Author: Deborah Hirtz
Author: James F. Casella

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