Xechem International Inc. (fka XKEM)

[lsd673]

Study of Children with Sickle Cell Anemia Stroke Prevention Stopped


Posted by Bea Lang on Friday, June 25, 2010, 11:47


The National Heart, Lung, and Blood Institute (NHLBI) has stopped a clinical trial evaluating a new approach to reduce the risk of recurrent stroke in children with sickle cell anemia and iron overload because of evidence that the new treatment was unlikely to prove better than the existing treatment.

Sickle cells are red blood cells with sickle hemoglobin become C-shaped, stiff, and sticky when they release the oxygen they carry. The deformed cells impede blood flow, causing severe pain and organ damage. About 10 percent of children with sickle cell disease suffer a stroke. Having experienced one, they are at high risk of having another unless they receive preventive treatment. Sickle cell disease affects more than 70,000 Americans.

The study tested whether the drug hydroxyurea, the only FDA-approved drug for treating sickle cell anemia, known to prevent complications of sickle cell disease in adults, was as effective as transfusions, the standard therapy, in reducing the risk of recurrent strokes. The study had 133 participants between the ages of 5 and 18 who had already experienced a stroke. Without further preventive measures, these children were at high risk of another stroke as well as life-threatening conditions due to iron overload. All had been receiving the standard treatment of blood transfusions for at least 18 months and had high levels of iron before entering the study.

The study also compared approaches to remove excess iron, a consequence of regular blood transfusions. Participants who were switched to hydroxyurea underwent regular phlebotomy (blood removal) to eliminate excess iron that had accumulated from their earlier transfusions and the others who continued to receive transfusion therapy were given standard oral iron-removal drug deferasirox, and phlebotomy did not reduce liver iron better than deferasirox therapy. Analysis of the available data indicated that continuing the trial was unlikely to show that phlebotomy would provide a greater benefit than deferasirox to control iron accumulation.

“Protecting our participants is an important factor in determining whether to stop a trial,” said Susan B. Shurin, MD, acting director of the NHLBI, who is a board-certified hematologist and pediatrician. The NHLBI is part of the National Institutes of Health. ” When an experimental treatment fails to meet its predetermined goals, it is best to return participants to standard treatment as soon as possible.” By the time the trial was halted, approximately one-third of participants had completed the study, during which they were treated and monitored for 30 months. At enrollment, participants were randomly assigned to either the alternative or the standard treatment group. The study’s independent Data and Safety Monitoring Board (DSMB) reviewed interim results from the trial and recommended stopping the study. The NHLBI accepted the recommendation and stopped the study on May 6.

N strokes occurred in the 66 participants who received the standard therapy of blood transfusions and deferasirox, but seven strokes occurred in the group of 67 participants who received hydroxyurea with phlebotomy. Study participants and their families have been contacted, and they will discuss future care options with their health care providers.

http://techcombo.com/2010/06/25/study-of-children-with-sickle-cell-anemia-stroke-prevention-stopped-123/

.