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Blood transfusion therapy is feasible in a clinical trial setting in children with sickle cell disease and silent cerebral infarcts
Author(s) -
King Allison A.,
Noetzel Michael,
White Desirée A.,
McKinstry Robert C.,
DeBaun Michael R.
Publication year - 2008
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.21338
Subject(s) - medicine , disease , transfusion therapy , clinical trial , pediatrics , intensive care medicine , blood transfusion
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. Procedure 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. Pediatr Blood Cancer 2008;50:599–602. © 2007 Wiley‐Liss, Inc.