TCD with Transfusions Changing to Hydroxyurea (TWiTCH): Hydroxyurea Therapy As an Alternative to Transfusions for Primary Stroke Prevention in Children with Sickle Cell Anemia

Transcranial Doppler (TCD) screening in children with sickle cell anemia (SCA) identifies abnormally elevated cerebral artery flow velocities that confer an elevated risk for primary stroke. Chronic transfusions offer effective stroke prophylaxis in this setting, but must be continued indefinitely a...

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Bibliographic Details
Published in:Blood 2015-12, Vol.126 (23), p.3-3
Main Authors: Ware, Russell E., Davis, Barry R, Schultz, William H, Brown, Clark, Aygun, Banu, Sarnaik, Sharada A., Odame, Isaac, Fuh, Beng, George, Alex, Owen, William, Luchtman-Jones, Lori, Rogers, Zora R., Hilliard, Lee, Gauger, Cynthia, Piccone, Connie M., Lee, Margaret T., Kwiatkowski, Janet, Jackson, Sherron, Miller, Scott T., Roberts, Carla W, Heeney, Matthew M., Kalfa, Theodosia A., Nelson, Stephen C, Imran, Hamayun, Nottage, Kerri A, Alvarez, Ofelia A., Rhodes, Melissa, Thompson, Alexis A., Rothman, Jennifer, Helton, Kathleen J., Roberts, Donna, Coleman, Jamie, Bonner, Melanie J, Kutlar, Abdullah, Patel, Niren, Wood, John C, Piller, Linda, Wei, Peng, Luden, Judy, Mortier, Nicole A., Stuber, Susan, Luban, Naomi L.C., Cohen, Alan R., Pressel, Sara L, Adams, Robert J.
Format: Article
Language:English
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Summary:Transcranial Doppler (TCD) screening in children with sickle cell anemia (SCA) identifies abnormally elevated cerebral artery flow velocities that confer an elevated risk for primary stroke. Chronic transfusions offer effective stroke prophylaxis in this setting, but must be continued indefinitely and lead to transfusional iron overload. An alternative treatment strategy that offers similar effective protection against primary stroke, and provides control of iron overload, is needed. TCD With Transfusions Changing to Hydroxyurea (TWiTCH, NCT01425307) was an NHLBI-funded Phase III multicenter randomized clinical trial comparing 24-months of standard treatment (transfusions) to alternative treatment (hydroxyurea) in children with SCA and abnormal TCD velocities. All eligible children had received at least 12 months of transfusions. TWiTCH had a non-inferiority trial design; the primary study endpoint was the 24-month TCD velocity obtained from a linear mixed model, controlling for baseline (enrollment) values, with a non-inferiority margin of 15 cm/sec. The transfusion arm maintained children at HbS
ISSN:0006-4971
1528-0020
DOI:10.1182/blood.V126.23.3.3