Blood transfusion therapy is feasible in a clinical trial setting in children with sickle cell disease and silent cerebral infarcts.
BACKGROUND
Silent cerebral infarcts are both morbid and progressive in children with sickle cell disease (SCD). While blood transfusion therapy is effective primary and secondary stroke prevention, the efficacy and acceptance of blood transfusion therapy for children with silent cerebral infarcts is unknown. The overall goals of this study were to determine the feasibility and short-term efficacy of blood transfusion therapy for silent cerebral infarcts.


METHODS
We conducted a single-arm feasibility intervention study. Children with SCD and silent cerebral infarcts received blood transfusion therapy. Short-term efficacy was defined as no progression of MRI findings on repeat MRI.


RESULTS
Ten children enrolled in the study, and nine were evaluable. Five were male. The average age was 10.0 years (range 3.1-16.8). Seven of nine completed the transfusion therapy for over 2 years, with an average duration of therapy of 2.7 years (range 1-6). Twenty infarcts and four cases of Moya Moya were present on the initial MRI. Seven of nine subjects had repeat MRIs. Of these seven, there were a total of 18 infarcts. One infarct increased in size and one case of Moya Moya progressed on repeat MRI. All other lesions were stable or became smaller in diameter while on transfusion therapy during the time of follow up (mean 3.9 years, range 2.1-5.7).


CONCLUSIONS
Blood transfusion therapy is a feasible therapy for the prevention of progression of silent cerebral infarcts.
King
Allison A
AA
Department of Pediatrics, Washington University School of Medicine, St. Louis Children's Hospital, St. Louis, Missouri, USA. king_a@kids.wustl.edu
Noetzel
Michael
M
White
Desirée A
DA
McKinstry
Robert C
RC
Debaun
Michael R
MR
D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000150:complications / Q000628:therapy*; D001925:Brain Damage, Chronic / Q000517:prevention & control; D002544:Cerebral Infarction / Q000175:diagnosis / Q000209:etiology / Q000473:pathology / Q000517:prevention & control / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D017707:Erythrocyte Transfusion; D005240:Feasibility Studies; D005260:Female; D006451:Hemoglobin, Sickle / Q000032:analysis; D006801:Humans; D008279:Magnetic Resonance Imaging; D008297:Male; D009072:Moyamoya Disease / Q000209:etiology / Q000473:pathology / Q000628:therapy; D009483:Neuropsychological Tests
D006451:Hemoglobin, Sickle