Sickle Cell Disease
What is sickle cell disease?
Sickle cell disease (SCD) is an inherited blood disorder that affects over 100,000 people of all ages in the U.S. It’s passed on from parents to children through genes. Genes are the instructions that tell your body how to work. SCD causes your body to make abnormal hemoglobin. Hemoglobin is a protein in your red blood cells.
Healthy red blood cells are round and flexible. They move through your small blood vessels and bring oxygen to all parts of your body. With SCD, your red blood cells are misshapen, hard and sticky. They get stuck in your blood vessels and clog them. This can cause you to have pain, infection, organ damage, low blood count, strokes and other serious health problems.
SCD affects each person differently, so there is no “best treatment” for everyone with SCD. Hydroxyurea, L-glutamine, voxelotor and crizanlizumab-tmca are the only approved medicines for SCD. They can help lower the number of pain crises, but they do not make SCD go away.
Other options to reduce SCD symptoms include:
Medicines to prevent blood clots
These medicines will not cure the disease but may help you feel better.
Is there a cure?
A blood or marrow transplant (BMT) is the only known cure for SCD. BMT is not surgery. It’s a treatment that takes healthy blood-forming cells from a donor and gives them to you through an intravenous (IV) infusion to replace the unhealthy cells.
Who can get a BMT?
Kids and teens with severe SCD may be able to get a BMT. If they have a brother or sister who matches them and can be their donor, they can get a BMT at some hospitals. Otherwise, they may be able to get a BMT if they join a clinical trial.
Almost 1 in 5 people with SCD have a healthy, fully matched sibling donor. Siblings can be your donor even if they have sickle cell trait. If you don’t have a matched donor in your family, your doctor can search the Be The Match Registry®. Finding a donor can take time, so your doctor may want to search the registry even if you don’t need a transplant now.
Currently, adults with severe SCD are only able to get a BMT if they join a clinical trial. Find clinical trials for SCD at ctsearchsupport.org/.
Ask your doctor about early HLA typing. You may not need a transplant right now, but early typing will help prepare you and your doctors if you need one in the future.
Learn more about sickle cell disease and BMT at BeTheMatch.org/SCD.