BACKGROUND: A substantial minority of neurologically normal children with sickle cell disease have lesions consistent with cerebral infarction as seen on magnetic resonance imaging (MRI). OBJECTIVES: To determine if transfusion therapy affects the rate at which silent infarcts develop and to evaluate the contribution of MRI of the brain to stroke prediction by transcranial Doppler (TCD) ultrasonography. STUDY DESIGN:Children with elevated TCD ultrasonographic velocity were randomized to receive long-term transfusion therapy or standard care. Magnetic resonance imaging of the brain was obtained at randomization, annually, and with clinical neurologic events. The risk for new silent lesions and/or stroke was compared for each treatment arm. RESULTS: Among the 37% of subjects with silent infarcts, those receiving standard care were significantly more likely to develop new silent lesions or stroke than were those who received transfusion therapy. For subjects receiving standard care, those with lesions at baseline were significantly more likely to develop stroke or new silent lesions than those whose MRI studies showed no abnormality. CONCLUSIONS:Transfusion therapy lowers the risk for new silent infarct or stroke for children having both abnormal TCD ultrasonographic velocity and silent infarct. However, those with both abnormalities who are not provided transfusion therapy are at higher risk for developing a new silent infarct or stroke than are those whose initial MRI showed no abnormality. The finding of a silent infarct reinforces the need for TCD ultrasonographic screening and consideration of transfusion therapy if the abnormalities are seen. Similarly, elevated TCD ultrasonographic velocity warrants MRI of the brain because children with both abnormalities seem to be at increased risk for developing new silent infarct or stroke.
RCT Entities:
BACKGROUND: A substantial minority of neurologically normal children with sickle cell disease have lesions consistent with cerebral infarction as seen on magnetic resonance imaging (MRI). OBJECTIVES: To determine if transfusion therapy affects the rate at which silent infarcts develop and to evaluate the contribution of MRI of the brain to stroke prediction by transcranial Doppler (TCD) ultrasonography. STUDY DESIGN:Children with elevated TCD ultrasonographic velocity were randomized to receive long-term transfusion therapy or standard care. Magnetic resonance imaging of the brain was obtained at randomization, annually, and with clinical neurologic events. The risk for new silent lesions and/or stroke was compared for each treatment arm. RESULTS: Among the 37% of subjects with silent infarcts, those receiving standard care were significantly more likely to develop new silent lesions or stroke than were those who received transfusion therapy. For subjects receiving standard care, those with lesions at baseline were significantly more likely to develop stroke or new silent lesions than those whose MRI studies showed no abnormality. CONCLUSIONS: Transfusion therapy lowers the risk for new silent infarct or stroke for children having both abnormal TCD ultrasonographic velocity and silent infarct. However, those with both abnormalities who are not provided transfusion therapy are at higher risk for developing a new silent infarct or stroke than are those whose initial MRI showed no abnormality. The finding of a silent infarct reinforces the need for TCD ultrasonographic screening and consideration of transfusion therapy if the abnormalities are seen. Similarly, elevated TCD ultrasonographic velocity warrants MRI of the brain because children with both abnormalities seem to be at increased risk for developing new silent infarct or stroke.
Authors: James F Casella; Allison A King; Bruce Barton; Desiree A White; Michael J Noetzel; Rebecca N Ichord; Cindy Terrill; Deborah Hirtz; Robert C McKinstry; John J Strouse; Thomas H Howard; Thomas D Coates; Caterina P Minniti; Andrew D Campbell; Bruce A Vendt; Harold Lehmann; Michael R Debaun Journal: Pediatr Hematol Oncol Date: 2010-03 Impact factor: 1.969
Authors: Katherine R Cejda; Matthew P Smeltzer; Eileen N Hansbury; Mary Elizabeth McCarville; Kathleen J Helton; Jane S Hankins Journal: Pediatr Radiol Date: 2012-06-19
Authors: Margaret T Lee; Sergio Piomelli; Suzanne Granger; Scott T Miller; Shannon Harkness; Donald J Brambilla; Robert J Adams Journal: Blood Date: 2006-08-01 Impact factor: 22.113
Authors: Michael R DeBaun; F Daniel Armstrong; Robert C McKinstry; Russell E Ware; Elliot Vichinsky; Fenella J Kirkham Journal: Blood Date: 2012-02-21 Impact factor: 22.113
Authors: Janet L Kwiatkowski; Robert A Zimmerman; Avrum N Pollock; Wendy Seto; Kim Smith-Whitley; Justine Shults; Anne Blackwood-Chirchir; Kwaku Ohene-Frempong Journal: Br J Haematol Date: 2009-06-04 Impact factor: 6.998