Clinical Trial: Stroke Prevention in Sickle Cell Anemia (STOP 1)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title:

Brief Summary: To reduce episodes of first time stroke by 75 percent in children with sickle cell anemia by the administration of prophylactic transfusion therapy.

Detailed Summary:

BACKGROUND:

Stroke, occurring in about 10 percent of pediatric patients with sickle cell disease, is one of the most devastating complications, with a high recurrence rate after the first episode. Several non-randomized studies have shown reduction in stroke recurrence when periodic blood transfusions are administered to maintain hemoglobin S under 30 percent. Periodic blood transfusions are associated with significant risks of iron overload and other complications and must be accompanied by parenteral iron chelation therapy. However, this has become a standard of care for prevention of recurrent stroke in SS children. Thus, a randomized trial of blood transfusion for secondary prevention would not be feasible because it would be considered unethical. Based on various studies, the recurrence rate is reduced from 46 to 67 percent to approximately 7 percent on transfusion therapy. Because most stroke patients are left with some neurological deficit, and face a lifetime of disability, primary prevention would have a significant impact on the management of patients. However, because of complications of blood transfusions, the hypothesis should be proven by a randomized clinical trial.

A primary prevention trial had not been possible because an acceptable means of detecting those children at risk of stroke was not available. The advent of TCD to identify arterial abnormalities for the prediction of stroke has provided a means of detection. TCD abnormalities have a high specificity (100 percent) and high sensitivity (90 percent) for detecting angiographically proven narrowing of arterial diameter. Thus, TCD examination of the basal cerebral arteries is predictive of who will develop a stroke.

DESIGN NARRATIVE:

Randomized, Phase II
Sponsor: Augusta University

Current Primary Outcome:

Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Augusta University

Dates:
Date Received: October 27, 1999
Date Started: July 1994
Date Completion: August 2000
Last Updated: December 21, 2015
Last Verified: November 2005