St. Joseph�s Children�s Hospital Participates in Clinical Trial for Pediatric Patients with Sickle Cell Disease
06/27/2006
St. Joseph�s Children�s Hospital is participating in a nationwide clinical trial that focuses on an investigational medication for iron overload, a serious complication in patients with sickle cell disease (SCD). Each year, one-third of the SCD patients in the United States undergo blood transfusions to help prevent serious complications from the disease, including iron overload. If left untreated, excess iron in the body can be fatal.
The compound being studied, ICL670, is an iron chelator that binds to the extra iron in the blood and then removes it out of the body through the feces. Since May 2004, St. Joseph�s Children�s Hospital has treated more than a dozen SCD patients with ICL670.
Despite the benefits of blood transfusions, the new red blood cells that are introduced directly into the bloodstream cause an increase in total body iron. Free iron, in excess, is highly toxic, and its over-accumulation can damage several organs, including the liver, heart and pancreas, resulting in such serious consequences as congestive heart failure, cirrhosis and diabetes.
More than 70,000 Americans have SCD and more than two million Americans have the sickle cell trait, meaning they carry one copy of the gene. SCD causes red blood cells to lose their ability to change shape, thus clogging the blood vessels. Among the potential complications of SCD are pain, stroke, infections, leg ulcers, kidney damage, anemia, and delayed growth. There is no cure for SCD. Usually diagnosed in infancy or early childhood, SCD is passed from one generation to the next. This means that if each parent has one copy of the gene, each child has a one in four chance of inheriting the disease.
