Children's of Minnesota treats more than 1,000 children with blood disorders each year – including over 80 percent of all Minnesota children with sickle cell disease or hemophilia. Kids with chronic blood disorders are at risk for other health issues. Fortunately, in recent years, major advances have been made in improving the quality and length of life for these children and teens.
Stroke prevention in Sickle Cell Disease
The hematology program at Children's is one of 25 sites in the U.S. selected for a National Institutes of Health funded study on stroke prevention in children with sickle cell disease with Children's hematologist, Stephen Nelson, MD serving as primary investigator. These patients are at a risk of experiencing strokes, which can be severely disabling and life threatening.
The risk of having a stroke is assessed using a trans-cranial Doppler (TCD) test. Patients who are at a higher risk of having a stroke as identified by TCD, will be randomly assigned to one of two stroke prevention treatments. Patients will be treated either with preventative transfusions or a daily oral medication, hydroxyurea. Results of this study will help establish new standards of care in stroke prevention.
Inhibitors in Hemophilia
Children's provides access to important therapy and research opportunities for patients with inherited bleeding disorders, both common and rare. One of the most common complications of therapy in patients with severe hemophilia is the development of antibodies, also known as inhibitors. When an inhibitor is present, it reduces the effectiveness of clotting factors given to a patient to help blood clot.
Children's is one of 42 centers that recently completed an international hemophilia inhibitor study, and is now participating in a new international study, focused on improving care for patients with hemophilia who have inhibitors that are difficult to treat. Children's hematologist, Margaret Heisel Kurth, MD, co-director of the HTC and National Hemophilia Foundation Clinician of the Year for 2010, recently published a study looking at the U.S. experience in immune tolerance induction in hemophilia A patients. She is also involved in a new international study, focused on improving care for patients with hemophilia who have inhibitors that are difficult to treat.
New therapies in hemophilia and rare bleeding disorders
Children's hematologist, Susan Kearney, MD, co-director of the HTC, is involved in research studies related to the use of plasma derived and recombinant factor XIII concentrate for patients who have an inherited deficiency. She is also principal investigator on studies that will allow access to long acting factor concentrates for patients with hemophilia A and B.