Protocol

TWiTCH: TCD with Transfusions Changing to Hydroxyurea (TWiTCH): A Phase III randomized clinical trial to compare standard therapy (erythrocyte transfusions) with alternative therapy (hydroxyurea) for the maintenance of lowered TCD velocities in pediatric subjects with sickle cell anemia and abnormal pre-treatment TCD velocities

Diseases

Hematologic Disorders : Sickle cell disease

Description

For children with sickle cell anemia, chronic transfusions are given to reduce the risk of stroke. Transfusions work well to lower the velocities and lower the risk of stroke, but have serious side effects including iron overload, developing antibodies or getting an infection. Pilot data suggests that hydroxyurea significantly lowers TCD velocities in children with sickle cell anemia. Also, hydroxyurea treatment can prevent other events like acute chest syndrome. However it is not known if hydroxyurea will be as good as blood transfusions to prevent stroke. 

The primary goal of the TWiTCH trial is to compare 24 months of alternative therapy (hydroxyurea) to standard therapy (transfusions) for children with sickle cell anemia and abnormal TCD velocities, who currently receive chronic transfusions for the prevention of primary stroke. For the alternative treatment schedule (hydroxyurea) to be declared at least as good as the standard treatment regimen (transfusions), after adjusting for baseline differences, the hydroxyurea-treated group must have similar outcomes to that observed with the transfusions. TWiTCH is a multi-center study.

Objectives

• To compare hydroxyurea treatment to transfusions for maintaining lowered Transcranial Doppler velocities in pediatric subjects with sickle cell anemia and abnormally high TCD velocities.
• To compare hydroxyurea treatment to transfusions in preventing primary stroke, management of iron overload, effects on quality of life, frequency of non-stroke neurological events and rate of other sickle cell related events.

Eligibility Criteria

• Patient must have severe form of sickle cell anemia (HbSS, HbSβ°thalassemia, HbSO_Arab).
• Patient must be between ≥ 4 and ≤ 15.99 years of age at the time of enrollment.
• Patient must have documented abnormally high Transcranial Doppler Velocity.
• Patient must have received at least of 12 months of chronic monthly transfusions.
• Patients who have had a clinical stroke or TIA will not be enrolled on this study.
• Patients who cannot tolerate chronic transfusion therapy will not be enrolled on this study.
• Patients who cannot take daily hydroxyurea will not be enrolled in this study.
• Patients who are using other agents for sickle cell disease will not be enrolled in this study.

For the current eligibility status of this clinical study, referring physicians must contact St. Jude Children's Research Hospital at 1-866-2ST-JUDE (1-866-278-5833).

Contact

Jane Hankins, MD
St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105 USA
Voice: 1-866-2ST-JUDE (1-866-278-5833)
FAX: 901-595-5068

Referring or consulting physicians only: protocolinfo@stjude.org

For all other inquiries about St. Jude Children's Research Hospital studies: info@stjude.org

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