Thalassemia Program

We work to improve the health and well-being of patients affected by thalassemia in a kid-friendly, family-focused environment. We provide comprehensive treatment, offering expert clinical care, a range of support services to assist families, community outreach, education and research into the nature, causes and treatments of hemoglobin disorders.

The thalassemia program is an active participant in the NHLBI-sponsored Thalassemia Clinical Research Network (TCRN). It has been designated a “Thalassemia Treatment Center of Excellence” by the Cooley’s Anemia Foundation. We’ve also received funding from the Centers for Disease Control and Prevention (CDC) and other federal agencies to perform research studies on the prevention of blood borne infections and other complications of thalassemia, and to enhance comprehensive care.

Our program actively collaborated with the Illinois Department of Public Health to begin universal newborn screening for thalassemia starting in 2008. As a result, families are able to confirm the diagnosis and receive specialized care for their infants as early as possible to prevent complications.

Most children with thalassemia require a life-long treatment of regular blood transfusions and iron chelation therapy. Complications from the disease and its treatment can include heart problems, liver damage, diabetes, infertility, growth failure and thin or deformed bones. Our specialists are trained to treat children with thalassemia, meet their specific needs and counsel all family members on preventive treatment measures and serious complications.

• Our team of pediatric hematologists are trained in treating all types of thalassemia diagnosis. Their goal is to provide the highest quality of comprehensive care, identifying and treating complications, improving the quality of life, and offering cutting-edge treatments and curative options. You can expect to see our hematologists during your clinic visit.    
• The pediatric nurse practitioner evaluates every thalassemia patient during visits for their routine transfusions under the supervision of a hematologist. You can expect to see our nurse practitioner at your transfusion visits in the Ambulatory Infusion Center.
• The nurse specialists dedicated to our thalassemia program provide continuing education and monitor compliance with chelation therapy. You will connect with our nurse specialists at clinical appointments, on the phone, and when utilizing MyChart.
• A social worker provides support regarding the diagnosis and therapy, while identifying all necessary resources available to the family. The social worker is available to all thalassemia patients at clinic visits, transfusion appointments, and inpatient admissions as needed. 
• Our genetic counselor is available to answer questions, provide education, and assist with family planning from diagnosis through all life stages. Our certified genetic counselor is likely the first person you will talk to from our team. They are available at clinic visits and by phone. 
• Our education liaison helps support patients with thalassemia succeed in a school setting. Patients and families can speak with the Education Liaison at clinical appointments, transfusion appointments, and during inpatient admissions. 
• The program manager connects patients and families to one another, facilitates patient and family advocacy events, and communicates important information to our patients through an email list, in-person appointments, and is always available by phone. You will also spend more time with the program manager when you/your child enters healthcare transition. 
• Research coordinators ensure that our thalassemia patients have access to cutting-edge treatments and novel therapies through clinical trials. You may meet one of our research coordinators at a clinic visit or over the phone. If you are enrolled in a study, you may have frequent communication with our research team.

The main symptom of thalassemia is moderate to severe anemia. Your treatment plan might include:

• Blood transfusions: Some patients with thalassemia may need regular blood transfusions to reduce the symptoms of anemia. The frequency of transfusions needed varies with each patient.
• Monitoring iron overload: Patients who receive regular transfusions can experience iron overload. Iron overload can cause complications like organ damage. It is important to monitor iron overload in thalassemia patients. We use imaging (specifically, magnetic resonance imaging (MRI)) to better understand a patient’s degree of iron overload.
• Managing iron overload: In thalassemia patients who are experiencing a high level of iron, our team will prescribe iron chelation therapy. Iron chelation helps the body to remove the extra iron that has built up in the body. There are different kinds of iron chelation treatments. Our providers will work with you to determine the best time to start iron chelation. 
• Medication: Our providers may talk to you about starting a medication called luspatercept. Luspatercept is a novel therapy for Transfusion Dependent Thalassemia and may help alleviate some symptoms of anemia and reduce the volume and/or frequency of blood transfusions.
• Curative options: Lurie Children’s offers both stem cell transplantation and gene therapy as curative options. The Comprehensive Thalassemia Program works closely with the Gene and Cellular Medicine Program to offer highly specialized treatment options for thalassemia patients. To learn more about gene therapy and thalassemia, explore our gene therapy resource. 

• ‘Thal Time’ Social Outing – Annual Event in Spring
  This event is purely social. It is an opportunity for patient and parents to connect with each other. Members of the Thalassemia Care Team will also be in attendance to connect one-on-one with patients and families.
• Annual Thalassemia Patient & Family Day – Annual Event in Fall
  The Annual Thalassemia Patient & Family Day is an inspiring and educational event dedicated to thalassemia patients, their families, advocates, and healthcare providers. The event aims to foster a deeper understanding of thalassemia, promote collaboration and education, and empower anyone involved in the journey of managing thalassemia. Participants will hear from both patients and providers on topics related to thalassemia care and healthy living. There will be activities for children of all ages, a light breakfast and lunch, and opportunities to connect with others in the community

For questions or comments about Patient and Family Events, contact Sarah Null – Program Manager at senull@luriechildrens.org.

Find additional information about thalassemia at: