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Credit: St Jude Children’s
Research Hospital
Treatment with hydroxyurea is a comparable alternative to blood transfusions for reducing the risk of stroke in children with sickle cell disease (SCD), according to the National Heart Lung and Blood Institute (NHLBI).
Early results of the phase 3 TWiTCH trial showed that daily doses of hydroxyurea lower the transcranial Doppler (TCD) blood velocity in children with SCD to a similar degree as blood transfusions, thereby decreasing the risk of stroke.
Based on these findings, the NHLBI has terminated the trial early, about a year before it was originally scheduled to end.
“Early results indicate that TWiTCH is a success,” said Russell E. Ware, MD, PhD, principal investigator of the study and director of hematology at Cincinnati Children’s Hospital Medical Center.
“A group of outside experts has been reviewing the TWiTCH data every few months to ensure the safety of children in the clinical trial and to monitor the data. This group met recently and, after careful consideration of the interim data results, recommended that the study be stopped since hydroxyurea worked as well as transfusions to lower TCD velocities.”
The NHLBI said it cannot release results from the TWiTCH trial, but some details are available. Researchers enrolled 121 children between the ages of 4 and 16 who had SCD and abnormally elevated TCD velocities.
Half of patients received transfusions, and the other half received daily hydroxyurea, which has not yet been approved for children with SCD.
The clinical data-collection portion of the study was originally scheduled for the 24-month mark, but collection was stopped after only half of the children completed the treatment phase.
At the first interim analysis, a data and safety monitoring board found that hydroxyurea was not inferior to regular blood transfusions in lowering TCD velocities. And the board said the strength of the statistical finding was unlikely to change with the collection of additional data.
After reviewing the board’s recommendation, the NHLBI decided to end the study early, as the primary endpoint had been met.
Study participants have been notified about the trial’s ending, and they will have the opportunity to receive the care they feel is best suited for them.
“No child should ever suffer a stroke, which is why it was so important for the NHLBI to support the TWiTCH trial,” said Gary Gibbons, MD, director of the NHLBI. “This critical research finding opens the door to more treatment options for clinicians trying to prevent strokes in children living with the sickle cell disease.” 
Credit: St Jude Children’s
Research Hospital
Treatment with hydroxyurea is a comparable alternative to blood transfusions for reducing the risk of stroke in children with sickle cell disease (SCD), according to the National Heart Lung and Blood Institute (NHLBI).
Early results of the phase 3 TWiTCH trial showed that daily doses of hydroxyurea lower the transcranial Doppler (TCD) blood velocity in children with SCD to a similar degree as blood transfusions, thereby decreasing the risk of stroke.
Based on these findings, the NHLBI has terminated the trial early, about a year before it was originally scheduled to end.
“Early results indicate that TWiTCH is a success,” said Russell E. Ware, MD, PhD, principal investigator of the study and director of hematology at Cincinnati Children’s Hospital Medical Center.
“A group of outside experts has been reviewing the TWiTCH data every few months to ensure the safety of children in the clinical trial and to monitor the data. This group met recently and, after careful consideration of the interim data results, recommended that the study be stopped since hydroxyurea worked as well as transfusions to lower TCD velocities.”
The NHLBI said it cannot release results from the TWiTCH trial, but some details are available. Researchers enrolled 121 children between the ages of 4 and 16 who had SCD and abnormally elevated TCD velocities.
Half of patients received transfusions, and the other half received daily hydroxyurea, which has not yet been approved for children with SCD.
The clinical data-collection portion of the study was originally scheduled for the 24-month mark, but collection was stopped after only half of the children completed the treatment phase.
At the first interim analysis, a data and safety monitoring board found that hydroxyurea was not inferior to regular blood transfusions in lowering TCD velocities. And the board said the strength of the statistical finding was unlikely to change with the collection of additional data.
After reviewing the board’s recommendation, the NHLBI decided to end the study early, as the primary endpoint had been met.
Study participants have been notified about the trial’s ending, and they will have the opportunity to receive the care they feel is best suited for them.
“No child should ever suffer a stroke, which is why it was so important for the NHLBI to support the TWiTCH trial,” said Gary Gibbons, MD, director of the NHLBI. “This critical research finding opens the door to more treatment options for clinicians trying to prevent strokes in children living with the sickle cell disease.”
Credit: St Jude Children’s
Research Hospital
Treatment with hydroxyurea is a comparable alternative to blood transfusions for reducing the risk of stroke in children with sickle cell disease (SCD), according to the National Heart Lung and Blood Institute (NHLBI).
Early results of the phase 3 TWiTCH trial showed that daily doses of hydroxyurea lower the transcranial Doppler (TCD) blood velocity in children with SCD to a similar degree as blood transfusions, thereby decreasing the risk of stroke.
Based on these findings, the NHLBI has terminated the trial early, about a year before it was originally scheduled to end.
“Early results indicate that TWiTCH is a success,” said Russell E. Ware, MD, PhD, principal investigator of the study and director of hematology at Cincinnati Children’s Hospital Medical Center.
“A group of outside experts has been reviewing the TWiTCH data every few months to ensure the safety of children in the clinical trial and to monitor the data. This group met recently and, after careful consideration of the interim data results, recommended that the study be stopped since hydroxyurea worked as well as transfusions to lower TCD velocities.”
The NHLBI said it cannot release results from the TWiTCH trial, but some details are available. Researchers enrolled 121 children between the ages of 4 and 16 who had SCD and abnormally elevated TCD velocities.
Half of patients received transfusions, and the other half received daily hydroxyurea, which has not yet been approved for children with SCD.
The clinical data-collection portion of the study was originally scheduled for the 24-month mark, but collection was stopped after only half of the children completed the treatment phase.
At the first interim analysis, a data and safety monitoring board found that hydroxyurea was not inferior to regular blood transfusions in lowering TCD velocities. And the board said the strength of the statistical finding was unlikely to change with the collection of additional data.
After reviewing the board’s recommendation, the NHLBI decided to end the study early, as the primary endpoint had been met.
Study participants have been notified about the trial’s ending, and they will have the opportunity to receive the care they feel is best suited for them.
“No child should ever suffer a stroke, which is why it was so important for the NHLBI to support the TWiTCH trial,” said Gary Gibbons, MD, director of the NHLBI. “This critical research finding opens the door to more treatment options for clinicians trying to prevent strokes in children living with the sickle cell disease.”