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.
RCT Entities:
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.
Authors: Amanda M Brandow; C Patrick Carroll; Susan Creary; Ronisha Edwards-Elliott; Jeffrey Glassberg; Robert W Hurley; Abdullah Kutlar; Mohamed Seisa; Jennifer Stinson; John J Strouse; Fouza Yusuf; William Zempsky; Eddy Lang Journal: Blood Adv Date: 2020-06-23
Authors: Elizabeth S Klings; Roberto F Machado; Robyn J Barst; Claudia R Morris; Kamal K Mubarak; Victor R Gordeuk; Gregory J Kato; Kenneth I Ataga; J Simon Gibbs; Oswaldo Castro; Erika B Rosenzweig; Namita Sood; Lewis Hsu; Kevin C Wilson; Marilyn J Telen; Laura M Decastro; Lakshmanan Krishnamurti; Martin H Steinberg; David B Badesch; Mark T Gladwin Journal: Am J Respir Crit Care Med Date: 2014-03-15 Impact factor: 21.405
Authors: Mehdi Nouraie; Jane A Little; Mariana Hildesheim; J Simon R Gibbs; Claudia R Morris; Roberto F Machado; Gregory J Kato; Victor R Gordeuk; Mark T Gladwin Journal: Am J Hematol Date: 2019-09-17 Impact factor: 10.047