Comprehensive Sickle Cell Center

Our approach to sickle cell disease treatment

Sickle cell disease is an inherited blood disorder that causes red blood cells to become sickled, or crescent shaped. These misshapen cells can block blood flow and reduce oxygen delivery throughout the body. This can lead to serious health complications, such as anemia and acute episodes of severe pain (pain crises).

At UCSF, families find age-appropriate care at every stage of life. Our team partners with patients and families to manage symptoms, prevent complications and support healthy growth and development.

Pain management

We work closely with patients and families to find the best pain management solutions for acute and chronic pain. Many care team members are involved in helping our patients, including:

• Emergency room physicians

• Orthopedic surgeons

• Pain specialists

• Psychologists

Antibiotics

Sickle cell disease is identified through routine newborn screening, allowing our team to begin care early. Infants with sickle cell disease typically take penicillin to prevent infections. Children continue taking antibiotics until age 5, after completing all routine immunizations. Some children may need to take antibiotics longer.

Hydroxyurea treatment

Hydroxyurea is the most widely used treatment for sickle cell disease. Most children start taking it daily during their first year of life to reduce red blood cell sickling and improve blood flow.

Hydroxyurea can reduce:

• Pain crises

• Hospitalizations

• Need for blood transfusions

• Episodes of acute chest syndrome (difficulty breathing, chest pain and coughing)

• Stroke risk

Hydroxyurea is a safe and effective, FDA-approved treatment that's supported by decades of research. Our team helps families establish a daily medication routine so children receive the greatest possible benefit from treatment.

Blood transfusions

Most people with sickle cell disease need blood transfusions at some point in their lives. Transfusions reduce the amount of sickled red blood cells and improve blood flow, lowering the risk of complications, such as stroke.

Whether your child needs transfusions depends on their symptoms and how they respond to hydroxyurea. Children with a high risk of stroke may need transfusions to prevent one. Children who have had a stroke need transfusions every two to four weeks.

Our hematologists and transfusion medicine specialists work together to ensure safe, carefully matched transfusions and reduce the risk of complications.

Bone marrow transplant and gene therapy

A bone marrow transplant (BMT) may be an option for children with a closely matched donor, which could be a sibling, parent or unrelated donor. The procedure replaces a patient's abnormal stem cells with healthy donor stem cells, which can produce healthy, new red blood cells.

Gene therapy is a newer treatment for people with sickle cell disease. The procedure collects a patient's own stem cells, modifies them in a lab to reduce sickling and infuses them back into the patient's bloodstream. UCSF offers both FDA-approved gene therapies for children – Casgevy (age 2 and older) and Lyfgenia (age 12 and older).

UCSF researchers are also leading a groundbreaking clinical trial that's taking a different approach to gene therapy. It uses gene editing to directly correct the genetic cause of sickle cell disease, with the aim of curing the condition.

Both BMT and gene therapy may cure sickle cell disease, but they are intensive treatments with long-term risks, including infertility. Our team helps families understand the risks and benefits so they can make informed treatment decisions.

Learn more about our expertise in blood and marrow transplant.

Specialized care for brain and reproductive health

Our multispecialty teams address the specific needs of people with sickle cell disease. For brain health, experts in hematology, neuropsychology, neuroradiology and neurovascular medicine work together to monitor stroke risk and evaluate cognitive function.

We also offer reproductive health care for adult and adolescent patients with sickle cell disease. Experts in adolescent medicine, gynecology, maternal-fetal medicine and urology work together to provide comprehensive care. Our team addresses concerns such as pain management during menstruation, pregnancy planning, genetic counseling and managing priapism (prolonged, painful erection).

For patients with infertility and those planning to have gene therapy or bone marrow transplant, we offer fertility preservation counseling. Our hematologists and obstetricians work closely together to help patients have the healthiest, safest pregnancies possible.