Sickle cell program
Sickle cell disease is an inherited blood disorder characterized by chronic anemia, a lifetime risk of serious infection and debilitating pain episodes. In addition, there are short and long term effects on virtually every organ system, including the brain, heart, lungs, spleen and eyes. As a result of these complications, children affected by sickle cell disease are at risk for poor growth and development and frequent hospitalization, which may ultimately affect their quality of life.
Sickle cell disease is the most common genetic disorder in the United States and affects more than 75,000 individuals, primarily those of African or African American race. Approximately 1 in 500 babies are born with the disease annually. In almost every state, including Illinois, children with sickle cell disease are identified at birth through a newborn screening program. The primary goal of these programs is to promote early identification of sickle cell disease and its variants, provide referral for appropriate services and encourage practices that prevent early complications.
Recent breakthroughs in pediatric research, including early detection of complications and improvements in treatment, have given hope for a healthier future to many children with sickle cell disease. However, too many others are still suffering — their childhood dominated by pain, life-threatening infections and frequent hospitalizations. More research and new treatments are needed.
Comprehensive Sickle Cell Disease Program at Children’s Memorial
The Comprehensive Sickle Cell Disease Program at Children’s Memorial is a nationally recognized center of excellence that currently follows almost 400 individuals with sickle cell disease and its variants. Directed by Horace Smith, MD, the program provides sickle cell disease care in all aspects, including both inpatient and outpatient services, aimed at managing the disease and preventing its complications. The program offers transfusion services through a therapeutic day hospital as well as routine screening by transcranial Doppler ultrasound to assess for risk of stroke.
Our comprehensive sickle cell disease clinic is held twice monthly. An additional clinic is held monthly for patients taking hydroxyurea, a common medication used to treat severe sickle cell disease and its complications. Our staff consists of three other full-time pediatric hematologists, two nurse specialists/nurse practitioners, a genetic counselor and a social worker dedicated to the program. Activities sponsored by the program include: a parent to parent network, community education and outreach, annual social activities for patients and their families, scholastic achievement programs and a formal program for transitioning patients to adult centered care.
A strong research program in sickle cell disease complements the clinical care offered by the Comprehensive Sickle Cell Disease Program. Children’s Memorial Hospital is currently one of nine centers around the country participating in the National Institutes of Health (National Heart, Lung and Blood Institute) sponsored Sickle Cell Disease Clinical Research Network. There are currently 10 research studies in sickle cell disease open for enrollment, including:
- NIH sponsored Silent Infarct Transfusion Trial (SITT) and Stroke with Transfusions Changing to Hydroxyurea (SWITCH) trial;
- Phase II study examining the use of an sPLA2 inhibitor for prevention of acute chest syndrome in children with sickle cell disease presenting with pain and fever (IMPACTS);
- Early detection of pulmonary hypertension and heart disease using Doppler echocardiography in children and young adults with sickle cell disease;
- Genetics of opioid response in children with sickle cell disease undergoing treatment for painful episodes;
- Role of stem cell transplantation from a matched unrelated donor source in children with sickle cell disease.
For more information on these research studies and contact information, please see http://www.childrensmrc.org/hot/Hematology/.