Sickle Cell Disease Treatment Options

Many factors influence treatment for your child’s sickle cell disease, including your child’s age and overall health, as well as the severity of the disease. Our care team uses a variety of treatments to support your child’s health. These include the following:

Blood transfusions for sickle cell disease

If your child is in sickle cell crisis, he or she may need a blood transfusion to remove sickle cells and replace them with healthy red blood cells. Repeated transfusions can help prevent severe complications, including stroke. The procedure is completed while your child is awake, via an IV line or through your child’s port, taking one to four hours.

Gene therapy for sickle cell disease

We are proud to be a Qualified Treatment Center for LYFGENIA, an FDA-approved gene therapy for some people with sickle cell disease ages 12 and older.

This gene therapy takes stem cells, or cells that make blood, from the patient’s body and adds new copies of a gene called HBB (beta-globin) that isn’t working correctly in patients with sickle cell disease. These modified cells are then put back into the patient’s body through an infusion, where they will start to make healthy red blood cells.

We also offer two other gene therapies for sickle cell disease through clinical trials. We are the only hospital in the Western United States to offer these clinical trials. 

Medicines for sickle cell disease

Medicines, such as hydroxyurea, are given to reduce the number of sickle cells in your child’s blood. Other medicines can improve anemia and reduce pain. Antibiotics may be given to prevent life-threatening infections.

Stroke assessments

If your child is at risk for stroke, we perform tests to view the blood flow in the brain and guide treatment accordingly.

Stem cell (bone marrow) transplants for sickle cell disease

Stem cell transplants are performed to treat, and cure, sickle cell disease. When healthy red blood stem cells are placed into your child’s bone marrow, your child’s body may then begin to grow its own healthy red blood cells.

At Stanford Medicine Children’s Health, we are known for providing innovative, leading-edge stem cell transplants, especially for blood diseases and disorders. These include haploidentical stem cell transplants, which allow for a partially matched donor such as a parent. To make graft-versus-host disease (GvHD) less likely, our world-renowned stem cell transplant doctors may perform an alpha/beta T-cell depleted haploidentical stem cell transplantation—selectively eliminating the immune system’s cells (alpha/beta T cells) from the donor stem cells and lowering the risk for GvHD.