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Blood, 1 August 2006, Vol. 108, No. 3, pp. 847-852.
Prepublished online as a Blood First Edition Paper on April 6, 2006; DOI 10.1182/blood-2005-10-009506.
 Previous Article | Next Article 
Submitted October 20, 2005
Accepted March 17, 2006
Stroke Prevention Trial in Sickle Cell Anemia (STOP):
Extended Follow-up and Final Results
Margaret T. Lee*, Sergio Piomelli, Suzanne Granger, Scott T. Miller, Shannon Harkness, Donald J. Brambilla, and Robert J. Adams
Division of Pediatric Hematology and Blood and Marrow Transplantation, Columbia University
New England Resarch Institutes
SUNY-Downstate Medical Center, Kings County Hospital
New England Reseach Institutes
New England Research Institutes
Medical College of Georgia
* Corresponding author; email: ml653{at}columbia.edu.
The Stroke Prevention Trial in Sickle Cell Anemia (STOP)
was a randomized trial to evaluate whether chronic
transfusion could prevent initial stroke in sickle cell
children at high risk as determined by transcranial
Doppler (TCD). The trial demonstrated a large benefit of
transfusion and was halted early. After termination of
the trial, patients participated in a Post-Trial Follow-
up Study. More patients in the Transfusion group (70%)
elected transfusion for primary stroke prevention
compared to those on Standard Care (45%). Six patients
with persistently abnormal TCD developed stroke. A
minority with initially abnormal TCD remained stroke-
free without transfusion. Except for lower baseline and
follow-up TCD velocities compared to those with stroke,
no predictive features of this apparent lower risk
subgroup could be determined. TCD results at last
testing in 108 that did not have stroke were: normal
(44.4%), conditional (26.9%), abnormal (22.2%) and
inadequate (6.5%). Patients on transfusion were more
likely to have normal TCD. Transfusion resulted in iron
overload and alloimmunization but no infection. The
study provides new information on acceptance rates and
long-term effects of transfusion. Persistent TCD
elevation signals ongoing stroke risk. Reduction in TCD
over time without transfusion is observed in some cases
and requires further study.
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