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 now offering a clinical trial testing gene therapy, specifically a technique known as CRISPR, for patients with severe sickle cell disease who are 12-40 years old. This gene editing technique takes some cells that create blood (stem cells) from the patient’s body, removes the mutated DNA that causes sickle cell from the patient’s genes, then puts the correct DNA sequence into the patient’s genes and infuses the cells back in the patient with hopes that they will flourish, replacing damaged cells with healthy ones. We are one of three centers in the United States to offer this trial, and the only center in the Western United States.
We are participating in other clinical trials for sickle cell disease, and families travel to us from across the nation to take advantage of these opportunities. While most studies are currently for adults only, you can look for clinical trials that may involve children at clinicaltrials.stanford.edu.
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.
