Thalassemia Center

Our collaborative approach to thalassemia care

Thalassemia is a genetic blood condition that reduces your body's ability to produce healthy red blood cells and hemoglobin. Hemoglobin is a protein that carries oxygen throughout your body. As a result, thalassemia can affect many aspects of a person's health.

Our team of specialists works together to manage thalassemia and support healthy development so patients can stay active and feel their best.

At UCSF, patients and their families have access to:

• Hematologists

• Cardiologists

• Endocrinologists

• Geneticists

• Hepatologists

• Nephrologists

• Orthopedists

• Radiologists

• Reproductive endocrinologists

• Transfusion medicine specialists

Our treatments for thalassemia

Thalassemia care often includes medications, called chelation therapy, and blood transfusions. Stem cell transplants and gene therapy are important options for patients receiving regular transfusions, which we discuss with families.

Blood transfusions

Blood transfusions are the most common treatment for thalassemia. Some people need a blood transfusion every few weeks. A transfusion delivers healthy red blood cells from a donor to prevent anemia and ensure their body gets enough oxygen.

At UCSF, we have an advanced blood bank and experts in transfusion medicine to make these procedures as safe, effective and comfortable as possible. Our specialized lab provides donor blood for patients with thalassemia that is closely matched to their own, including rare blood types. They also supply blood for people with red blood cell antibodies, which are produced by immune cells and attack donor red blood cells.

Chelation therapy

People who receive regular blood transfusions have a high risk of iron overload. This is a serious complication where iron builds up in vital organs, such as the heart and liver. To prevent this, chelation therapy uses medications to remove excess iron from the body. These medicines are usually taken by mouth but can also be injected under the skin or infused into a vein.

Advanced monitoring for iron overload

During chelation therapy, we monitor iron levels and organ function using blood tests and specialized T2 star (T2*) MRI scans, which measure iron buildup in the heart and liver. This advanced, noninvasive technology is not widely available and helps us better protect each patient's long-term health.

Stem cell transplant and gene therapy for thalassemia

Gene therapy is an option for people with transfusion-dependent beta thalassemia. Two FDA-approved gene therapies, Zynteglo and Casgevy, are available at UCSF. These one-time treatments can eliminate the need for blood transfusions.

Gene therapy involves modifying a patient's stem cells, which are cells that make other blood cells. Treatment requires chemotherapy, hospitalization and intensive follow-up monitoring. We discuss the risks and benefits with families to help them make informed decisions.

A stem cell transplant is another potential cure for thalassemia. This procedure uses stem cells collected from a matched donor, either a sibling or unrelated person. UCSF is home to one of the top pediatric stem cell transplant programs in the nation.

Learn more about our expertise in blood and marrow transplant.