Pediatric Sickle Cell Disease
The Sickle Cell Disease Program at Children’s is one of the nation’s largest serving pediatric patients. It focuses on the prevention of disease complications and management using the newest treatment strategies, including hydroxyurea, chronic transfusions, stem cell (bone marrow) transplantation and state-of-the-art approaches to infection prevention, pain management and treatment of specific organ-related complications (chest syndrome, priapism, avascular necrosis of the femoral head, etc.).
What We Provide
- Our widely acclaimed diagnostic and treatment program for children with sickle cell disease is one of the nation's largest, with more than 700 active patients.
- Children's Health offers standard treatment modalities, such as chronic blood transfusion programs, hydroxyurea therapy and stem cell transplantation. We are also studying molecular targeted treatment for pain control in sickle cell disease.
- Hematologists on the medical staff at Children's have spearheaded the development of a comprehensive set of diagnostic and management guidelines for children with sickle cell disease that have been endorsed by the Texas Department of Health.
- Sickle cell patient information, such as every hospitalization and major complication, has been collected for 20 years on a computerized database, which helps improve continuity of care and research.
Each year, our staff cares for more than 700 children with the various forms of sickle cell disease, often with the very treatments our researchers developed. These elements of our program stand out as exceptional:
- Neurology: Neurological complications are common in sickle cell patients, particularly stroke and headache. Michael Dowling, M.D., Ph.D., M.S.C.S., is one of a handful of pediatric neurologists in the world dedicated to both the care and research of neurologic disease in sickle cell patients. Because of his collaboration, we can offer Transcranial Doppler (TCD) testing in our clinic as a part of our comprehensive visits.
- Chronic transfusion: Proven to help prevent stroke in sickle cell patients, transfusions are a critical element of our overall program. Nurses and nurse practitioners are dedicated to this area, which manages transfusions for more than 90 children. Our transfusion program avoids Port-a-Cath placement in most patients. We are experienced with all available iron overload medication options and offer the erythrocytapheresis procedure, a specialized transfusion technique that prevents iron overload.
- Transition: A hematologist in the Department of Medicine at UT Southwestern, manages a transition program that prepares our patients for the complex adult medical system. This unique resource allows teenage patients to develop a relationship with the same physician who will care for them after transition.
- Bone marrow transplant: Bone marrow transplant is increasing as a treatment strategy for sickle cell patients in our program over the past few years. This specialized treatment is the only available cure for sickle cell disease.
From its earliest days, the program has aggressively pursued development of new treatment approaches. In the 1980s, it led the way on the use of prophylactic penicillin in infants and continuous intravenous opioid analgesics in children. The 1990s brought pioneering studies of the outpatient management of fever in low-risk patients, and studies of intravenous corticosteroid agents on children with vaco-occlusive crisis and acute chest syndrome.
Around the country, hematologists rely on data from the Dallas Newborn Cohort, a cohort of children with sickle cell disease cared for here since 1983. Providing comprehensive care for these children has allowed us to identify the medical problems that they face as they become young adults.
We collaborated with other leading pediatric sickle cell centers to study the use of hydroxyurea in infants. Nicknamed BABY HUG, landmark findings were published in Lancet. BABY HUG II, a follow-up study, is being led by Zora R. Rogers, M.D.