Published online September 3, 2007
PEDIATRICS Vol. 120 No. 4 October 2007, pp. e1107-e1116 (doi:10.1542/peds.2006-2002)
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SPECIAL ARTICLE

Preventing Stroke Among Children With Sickle Cell Anemia: An Analysis of Strategies That Involve Transcranial Doppler Testing and Chronic Transfusion

Maitreyi Mazumdar, MD, MPHa,b, Matthew M. Heeney, MDc,d, Colin M. Sox, MD, MSe and Tracy A. Lieu, MD, MPHe,f

a Department of Neurology
c Divisions of Hematology/Oncology
f General Pediatrics, Children's Hospital Boston, Boston, Massachusetts
b Harvard Pediatric Health Services Research Fellowship Program, Boston, Massachusetts
d Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts
e Department of Ambulatory Care and Prevention, Center for Child Health Care Studies, Harvard Medical School and Harvard Pilgrim Health Care, Boston, Massachusetts

BACKGROUND. Transcranial Doppler ultrasonography can identify children with sickle cell anemia who are at elevated risk of stroke and may benefit from chronic transfusions. Uncertainty about the risk/benefit trade-offs of chronic transfusion has led some clinicians to decide not to offer transcranial Doppler ultrasonography screening.

OBJECTIVES. Our goals were to (1) compare the projected benefits and risks of 6 primary stroke-prevention strategies, (2) estimate the optimal frequency of screening, and (3) identify key assumptions that influence the risk/benefit relationship.

METHODS. We designed a decision model to compare 6 primary stroke-prevention strategies: (1) annual transcranial Doppler ultrasonography screening until age 16 with children at high risk of stroke receiving monthly transfusion for life; (2) annual transcranial Doppler ultrasonography until age 16 with transfusions until age 18; (3) biannual transcranial Doppler ultrasonography until age 16 with transfusions until age 18; (4) annual transcranial Doppler ultrasonography until age 10 with transfusion until age 18; (5) 1-time screening at age 2 with transfusion until age 18; and (6) no intervention. Assumptions were derived from the published literature.

RESULTS. For a hypothetical cohort of 2-year-old children, the optimal strategy was transcranial Doppler ultrasonography screening annually until age 10 with children at high risk receiving monthly transfusions until age 18. The optimal strategy would prevent 32% of strokes predicted to occur without intervention. The optimal strategy led to benefits similar to more intensive screening and transfusion strategies but resulted in fewer adverse events. All the intervention strategies resulted in net losses in life expectancy, because the projected mortality averted by stroke prevention was outweighed by the projected increase in mortality from transfusion. Results were sensitive to adherence rates to iron-chelation therapy.

CONCLUSIONS. The optimal stroke-prevention strategy was projected to be annual transcranial Doppler ultrasonography screening until age 10 with transfusion for children at high risk until age 18. Better adherence to chelation therapy would improve life expectancy in all intervention strategies.

Key Words: sickle cell anemia • stroke • transcranial Doppler • transfusions • health services research

Abbreviations: TCD—transcranial Doppler ultrasonography • NHLBI—National Heart, Lung, and Blood Institute • STOP—Stroke Prevention Trial in Sickle Cell Anemia