CHKD Patients Participated in Clinical Trial

Norfolk, VA - Patients and physicians at Children’s Hospital of The King’s Daughters recently participated in a national clinical trial that helped prove the safety of an important new treatment to reduce the risk of strokes in patients with sickle cell disease.

The NIH-sponsored study of hydroxyurea therapy showed such promising results that officials terminated the study a year earlier than expected to enable more patients to receive the therapy. The study, which included 121 patients between age 4 and 16 in the United States and Canada, began in September of 2011. Of the 121 patients in the study, eight were patients at CHKD.

Known as TWiTCH (Transcranial Doppler With Transfusions Changing to Hydroxyurea), the therapy can benefit patients with cerebrovascular disease and increased stroke risk.

The standard therapy for children with sickle cell disease who are known to have an increased risk of stroke has been monthly blood transfusions. While effective, this traditional therapy can have significant side effects including severe iron overload and transfusion reactions, which have been increasingly recognized as sources of morbidity in patients with sickle cell disease. The new treatment avoids these side effects and works in multiple ways to decrease the clogging of blood vessels from sickle-shaped blood cells.

“The results of this study will have great impact on patients living with sickle cell disease across the world,” said pediatric hematologist/oncologist William Owen, MD, who directed CHKD’s participation in the program. “We are indebted to our dedicated patients and families for their participation in this most important trial. Additionally, this study would not have been possible without the joint effort by multiple divisions of our hospital including the Children’s Cancer and Blood Disorders Center, pharmacy, laboratory medicine, and radiology.”

Children’s Hospital of The King’s Daughters is the only freestanding children’s hospital in Virginia, serving the medical and surgical needs of the nearly 500,000 children throughout greater Hampton Roads, the Eastern Shore of Virginia and northeastern North Carolina. The CHKD Health System operates primary care pediatric practices, surgical practices, multi-service health centers and satellite offices throughout its service region.