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Clinical Trial
. 2001 Dec;139(6):785-9.
doi: 10.1067/mpd.2001.119593.

Impact of chronic transfusion on incidence of pain and acute chest syndrome during the Stroke Prevention Trial (STOP) in sickle-cell anemia

S T Miller  1 , E Wright, M Abboud, B Berman, B Files, C D Scher, L Styles, R J Adams; STOP Investigators
Affiliations
Clinical Trial

Impact of chronic transfusion on incidence of pain and acute chest syndrome during the Stroke Prevention Trial (STOP) in sickle-cell anemia

S T Miller et al. J Pediatr. 2001 Dec.

Abstract

Objective: The Stroke Prevention Trial (STOP) demonstrated that chronic transfusion is highly effective in reducing the risk of stroke in children with sickle-cell disease and an abnormal transcranial Doppler ultrasonography examination result. Our objective was to determine whether chronic transfusion therapy reduces the incidence of pain and acute chest syndrome.

Methods: During STOP, 130 children with sickle-cell anemia or sickle beta(0)-thalassemia and abnormal transcranial Doppler ultrasonography examination result were randomly assigned to chronic transfusion (n = 63) or observation (n = 67). In addition to monitoring for stroke, nonneurologic sickle-cell complications were identified and recorded.

Results: Mean age at STOP study entry was 8.3 +/- 3.3 years, and mean follow-up was 19.6 +/- 6.5 months. Hospitalization rates (based on intent-to-treat analysis) for acute chest syndrome were 4.8 and 15.3 per 100 patient-years (P =.0027) and for pain were 16.2 and 27.6 per 100 patient-years (P =.13) in the chronic transfusion and observed groups, respectively. If analyzed according to treatment actually received, the difference in pain rate becomes significant (9.7 vs 27.1 events per 100 patient-years, P =.014), and transfusion remains protective from acute chest syndrome (2.2 vs 15.7 events per 100 patient-years, P =.0001).

Conclusions: Compliance with aggressive chronic transfusion reduces the frequency of acute chest syndrome and pain episodes.

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