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

M. R. DeBaun*, M. Gordon, R. C. McKinstry, M. J. Noetzel, D. A. White, S. A. Sarnaik, E. R. Meier, T. H. Howard, S. Majumdar, B. P.D. Inusa, P. T. Telfer, M. Kirby-Allen, T. L. McCavit, A. Kamdem, G. Airewele, G. M. Woods, B. Berman, J. A. Panepinto, B. R. Fuh, J. L. Kwiatkowski & 18 others A. A. King, J. M. Fixler, M. M. Rhodes, Alexis A Thompson, M. E. Heiny, R. C. Redding-Lallinger, F. J. Kirkham, N. Dixon, C. E. Gonzalez, K. A. Kalinyak, C. T. Quinn, J. J. Strouse, J. P. Miller, H. Lehmann, M. A. Kraut, W. S. Ball, D. Hirtz, J. F. Casella

*Corresponding author for this work

Research output: Contribution to journalArticle

214 Citations (Scopus)

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 bloodtransfusion 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.

Original languageEnglish (US)
Pages (from-to)699-710
Number of pages12
JournalNew England Journal of Medicine
Volume371
Issue number8
DOIs
StatePublished - Jan 1 2014

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Sickle Cell Anemia
Stroke
Observation
Recurrence
Incidence
Blood Transfusion
Nervous System Trauma
Neurologic Examination
Magnetic Resonance Imaging
Clinical Trials
Confidence Intervals
Therapeutics

ASJC Scopus subject areas

  • Medicine(all)

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DeBaun, M. R., Gordon, M., McKinstry, R. C., Noetzel, M. J., White, D. A., Sarnaik, S. A., ... Casella, J. F. (2014). Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia. New England Journal of Medicine, 371(8), 699-710. https://doi.org/10.1056/NEJMoa1401731
DeBaun, M. R. ; Gordon, M. ; McKinstry, R. C. ; Noetzel, M. J. ; White, D. A. ; Sarnaik, S. A. ; Meier, E. R. ; Howard, T. H. ; Majumdar, S. ; Inusa, B. P.D. ; Telfer, P. T. ; Kirby-Allen, M. ; McCavit, T. L. ; Kamdem, A. ; Airewele, G. ; Woods, G. M. ; Berman, B. ; Panepinto, J. A. ; Fuh, B. R. ; Kwiatkowski, J. L. ; King, A. A. ; Fixler, J. M. ; Rhodes, M. M. ; Thompson, Alexis A ; Heiny, M. E. ; Redding-Lallinger, R. C. ; Kirkham, F. J. ; Dixon, N. ; Gonzalez, C. E. ; Kalinyak, K. A. ; Quinn, C. T. ; Strouse, J. J. ; Miller, J. P. ; Lehmann, H. ; Kraut, M. A. ; Ball, W. S. ; Hirtz, D. ; Casella, J. F. / Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia. In: New England Journal of Medicine. 2014 ; Vol. 371, No. 8. pp. 699-710.
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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 bloodtransfusion 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.",
author = "DeBaun, {M. R.} and M. Gordon and McKinstry, {R. C.} and Noetzel, {M. J.} and White, {D. A.} and Sarnaik, {S. A.} and Meier, {E. R.} and Howard, {T. H.} and S. Majumdar and Inusa, {B. P.D.} and Telfer, {P. T.} and M. Kirby-Allen and McCavit, {T. L.} and A. Kamdem and G. Airewele and Woods, {G. M.} and B. Berman and Panepinto, {J. A.} and Fuh, {B. R.} and Kwiatkowski, {J. L.} and King, {A. A.} and Fixler, {J. M.} and Rhodes, {M. M.} and Thompson, {Alexis A} and Heiny, {M. E.} and Redding-Lallinger, {R. C.} and Kirkham, {F. J.} and N. Dixon and Gonzalez, {C. E.} and Kalinyak, {K. A.} and Quinn, {C. T.} and Strouse, {J. J.} and Miller, {J. P.} and H. Lehmann and Kraut, {M. A.} and Ball, {W. S.} and D. Hirtz and Casella, {J. F.}",
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DeBaun, MR, Gordon, M, McKinstry, RC, Noetzel, MJ, White, DA, Sarnaik, SA, Meier, ER, Howard, TH, Majumdar, S, Inusa, BPD, Telfer, PT, Kirby-Allen, M, McCavit, TL, Kamdem, A, Airewele, G, Woods, GM, Berman, B, Panepinto, JA, Fuh, BR, Kwiatkowski, JL, King, AA, Fixler, JM, Rhodes, MM, Thompson, AA, Heiny, ME, Redding-Lallinger, RC, Kirkham, FJ, Dixon, N, Gonzalez, CE, Kalinyak, KA, Quinn, CT, Strouse, JJ, Miller, JP, Lehmann, H, Kraut, MA, Ball, WS, Hirtz, D & Casella, JF 2014, 'Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia', New England Journal of Medicine, vol. 371, no. 8, pp. 699-710. https://doi.org/10.1056/NEJMoa1401731

Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia. / DeBaun, M. R.; Gordon, M.; McKinstry, R. C.; Noetzel, M. J.; White, D. A.; Sarnaik, S. A.; Meier, E. R.; Howard, T. H.; Majumdar, S.; Inusa, B. P.D.; Telfer, P. T.; Kirby-Allen, M.; McCavit, T. L.; Kamdem, A.; Airewele, G.; Woods, G. M.; Berman, B.; Panepinto, J. A.; Fuh, B. R.; Kwiatkowski, J. L.; King, A. A.; Fixler, J. M.; Rhodes, M. M.; Thompson, Alexis A; Heiny, M. E.; Redding-Lallinger, R. C.; Kirkham, F. J.; Dixon, N.; Gonzalez, C. E.; Kalinyak, K. A.; Quinn, C. T.; Strouse, J. J.; Miller, J. P.; Lehmann, H.; Kraut, M. A.; Ball, W. S.; Hirtz, D.; Casella, J. F.

In: New England Journal of Medicine, Vol. 371, No. 8, 01.01.2014, p. 699-710.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia

AU - DeBaun, M. R.

AU - Gordon, M.

AU - McKinstry, R. C.

AU - Noetzel, M. J.

AU - White, D. A.

AU - Sarnaik, S. A.

AU - Meier, E. R.

AU - Howard, T. H.

AU - Majumdar, S.

AU - Inusa, B. P.D.

AU - Telfer, P. T.

AU - Kirby-Allen, M.

AU - McCavit, T. L.

AU - Kamdem, A.

AU - Airewele, G.

AU - Woods, G. M.

AU - Berman, B.

AU - Panepinto, J. A.

AU - Fuh, B. R.

AU - Kwiatkowski, J. L.

AU - King, A. A.

AU - Fixler, J. M.

AU - Rhodes, M. M.

AU - Thompson, Alexis A

AU - Heiny, M. E.

AU - Redding-Lallinger, R. C.

AU - Kirkham, F. J.

AU - Dixon, N.

AU - Gonzalez, C. E.

AU - Kalinyak, K. A.

AU - Quinn, C. T.

AU - Strouse, J. J.

AU - Miller, J. P.

AU - Lehmann, H.

AU - Kraut, M. A.

AU - Ball, W. S.

AU - Hirtz, D.

AU - Casella, J. F.

PY - 2014/1/1

Y1 - 2014/1/1

N2 - 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 bloodtransfusion 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.

AB - 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 bloodtransfusion 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.

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U2 - 10.1056/NEJMoa1401731

DO - 10.1056/NEJMoa1401731

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JO - New England Journal of Medicine

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DeBaun MR, Gordon M, McKinstry RC, Noetzel MJ, White DA, Sarnaik SA et al. Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia. New England Journal of Medicine. 2014 Jan 1;371(8):699-710. https://doi.org/10.1056/NEJMoa1401731