PREVENTION OF CONVERSION TO ABNORMAL TCD WITH HYDROXYUREA IN SICKLE CELL ANEMIA: A PHASE III INTERNATIONAL RANDOMIZED CLINICAL TRIAL

Children with sickle cell anemia (SCA) and conditional transcranial Doppler (TCD) ultrasound velocities (170-199 cm/sec) may develop stroke. However, with limited available clinical data, the current standard of care for conditional TCD velocities is observation. The efficacy of hydroxyurea in preve...

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Veröffentlicht in:American journal of hematology 2015-11, Vol.90 (12), p.1099-1105
Hauptverfasser: Hankins, Jane S., McCarville, M. Beth, Rankine-Mullings, Angela, Reid, Marvin E., Lobo, Clarisse L.C., Moura, Patricia G., Ali, Susanna, Soares, Deanne, Aldred, Karen, Jay, Dennis W., Aygun, Banu, Bennett, John, Kang, Guolian, Goldsmith, Jonathan C., Smeltzer, Matthew P., Boyett, James M., Ware, Russell E.
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container_end_page 1105
container_issue 12
container_start_page 1099
container_title American journal of hematology
container_volume 90
creator Hankins, Jane S.
McCarville, M. Beth
Rankine-Mullings, Angela
Reid, Marvin E.
Lobo, Clarisse L.C.
Moura, Patricia G.
Ali, Susanna
Soares, Deanne
Aldred, Karen
Jay, Dennis W.
Aygun, Banu
Bennett, John
Kang, Guolian
Goldsmith, Jonathan C.
Smeltzer, Matthew P.
Boyett, James M.
Ware, Russell E.
description Children with sickle cell anemia (SCA) and conditional transcranial Doppler (TCD) ultrasound velocities (170-199 cm/sec) may develop stroke. However, with limited available clinical data, the current standard of care for conditional TCD velocities is observation. The efficacy of hydroxyurea in preventing conversion from conditional to abnormal TCD (≥200 cm/sec), which confers a higher stroke risk, has not been studied prospectively in a randomized trial. Sparing Conversion to Abnormal TCD Elevation (SCATE #NCT01531387) was an NHLBI-funded Phase III multicenter international clinical trial comparing alternative therapy (hydroxyurea) to standard care (observation) to prevent conversion from conditional to abnormal TCD velocity in children with SCA. SCATE enrolled 38 children from the United States, Jamaica, and Brazil [HbSS ( 36 ), HbSβ 0 -thalassemia ( 1 ), and HbSD ( 1 ), median age 5.4 years (range, 2.7-9.8)]. Due to slow patient accrual and administrative delays, SCATE was terminated early. In an intention-to-treat analysis, the cumulative incidence of abnormal conversion was 9% (95% CI 0 to 35%) in the hydroxyurea arm and 47% (95% CI 6 to 81%) in observation arm at 15 months ( p =0.16). In post-hoc analysis according to treatment received, significantly fewer children on hydroxyurea converted to abnormal TCD velocities, compared to observation (0% versus 50%, p =0.02). After a mean of 10.1 months, a significant change in mean TCD velocity was observed with hydroxyurea treatment (−15.5 versus +10.2 cm/sec, p =0.02). No stroke events occurred in either arm. Hydroxyurea reduces TCD velocities in children with SCA and conditional velocities.
doi_str_mv 10.1002/ajh.24198
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The efficacy of hydroxyurea in preventing conversion from conditional to abnormal TCD (≥200 cm/sec), which confers a higher stroke risk, has not been studied prospectively in a randomized trial. Sparing Conversion to Abnormal TCD Elevation (SCATE #NCT01531387) was an NHLBI-funded Phase III multicenter international clinical trial comparing alternative therapy (hydroxyurea) to standard care (observation) to prevent conversion from conditional to abnormal TCD velocity in children with SCA. SCATE enrolled 38 children from the United States, Jamaica, and Brazil [HbSS ( 36 ), HbSβ 0 -thalassemia ( 1 ), and HbSD ( 1 ), median age 5.4 years (range, 2.7-9.8)]. Due to slow patient accrual and administrative delays, SCATE was terminated early. In an intention-to-treat analysis, the cumulative incidence of abnormal conversion was 9% (95% CI 0 to 35%) in the hydroxyurea arm and 47% (95% CI 6 to 81%) in observation arm at 15 months ( p =0.16). In post-hoc analysis according to treatment received, significantly fewer children on hydroxyurea converted to abnormal TCD velocities, compared to observation (0% versus 50%, p =0.02). After a mean of 10.1 months, a significant change in mean TCD velocity was observed with hydroxyurea treatment (−15.5 versus +10.2 cm/sec, p =0.02). No stroke events occurred in either arm. 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title PREVENTION OF CONVERSION TO ABNORMAL TCD WITH HYDROXYUREA IN SICKLE CELL ANEMIA: A PHASE III INTERNATIONAL RANDOMIZED CLINICAL TRIAL
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