Patients with sickle cell disease or other hemolytic anemias should be aware that numerous clinical trials are exploring non-transfusion therapies for these serious blood disorders. See www.clinicaltrials.gov. I am a pediatric hematologist and encourage people to look at these trials when relatively well and consider voluntary participation in them. For example, a study of non-transfusion therapy in sickle cell disease recently closed because of slow enrollment. A series of such slow-enrolling studies will discourage the pharmaceutical industry from even proposing to try new therapies for a certain condition, which means that options may never get to be evaluated properly or approved by the FDA. What are the best ways to get this message out?
- Dr. Lewis Hsu
Blood Conservation Program and Marian Anderson Sickle Cell Center
St. Christopher's Hospital for Children
Philadelphia, Pennsylvania