A stem cell or bone marrow transplant is the only cure for sickle cell anemia. But they’re risky procedures, so they’re reserved for people with severe disease. Newer treatments like gene therapy may become an option in the future.
Sickle cell anemia (SCA) is an inherited disorder that affects your red blood cells. It’s most common in people who live in or have ancestors from sub-Saharan Africa, India, Central or South America, or parts of the Mediterranean and Middle East.
Also known as subtype HbSS, SCA is the most common type of sickle cell disease. It occurs when you inherit two hemoglobin S genes from each parent. These genes cause your red blood cells to have a rigid, “sickle” shape.
When you have SCA, sickle cells can die prematurely, leaving your body with a red blood cell deficit. Due to their shape, they may also get stuck in your blood vessels. Combined, these two issues can lead to various health problems, some of which may be life threatening.
The only cure for SCA is a stem cell or bone marrow transplant. But there are limitations to these procedures, which makes their use in SCA relatively uncommon.
As scientists discover more about possible treatments for SCA, newer options may emerge.
Your bone marrow is where red blood cells form. With SCA, your bone marrow produces sickle-shaped red blood cells.
Your bone marrow is also the site of stem cell production. These are early cells that later become red and white blood cells and platelets.
As of January 2023, stem cell or bone marrow transplants are the only Food and Drug Administration (FDA) approved cures for SCA.
In theory, a bone marrow or stem cell transplant could provide your body a fresh start with new red blood cells that are not sickle-shaped.
Doctors primarily use stem cell or bone marrow transplants in young children with severe sickle cell disease, including SCA. This is because children are less likely to have extensive organ damage that could lead to life threatening complications after the procedure.
While people often discuss stem cell and bone marrow transplants interchangeably, these procedures involve key differences.
During a stem cell transplant, healthcare professionals take blood cells from a healthy donor and place them into the recipient’s bone marrow.
A bone marrow transplant, on the other hand, involves taking cells directly from bone marrow.
Stem cell transplants are more common than bone marrow transplants because it’s easier to collect them via a blood sample.
Doctors rarely recommend stem cell or bone marrow transplants for SCA. This is due to a high risk of life threatening complications. It’s also difficult to find a close enough match to ensure a successful procedure.
One of the main reasons stem cell or bone marrow transplants are not common for SCA is the risk of serious side effects. Complications may be fatal.
Finding a match
For a transplant to be successful, bone marrow or stem cells must come from a close match. This most often involves bone marrow or stem cells from a sibling. The donor must also be a human leukocyte antigen (HLA) match.
Who’s a good match for a stem cell transplant to cure sickle cell anemia?
A close relative is the best match for a stem cell transplant for SCA. Siblings tend to be the best donors due to the close match of their bone marrow with the recipient.
In cases when there isn’t a brother or sister available, a doctor may consider another relative.
Because SCA is an inherited disorder, scientists are investigating the possibility of gene therapies. While still in development, gene therapy could potentially cure sickle cell disease by either changing DNA in faulty genes or replacing them altogether.
If successful, gene therapy could work by taking a person’s own stem cells, modifying them in a lab, and then injecting them back into the bloodstream. In theory, these stem cells could then help generate healthy red blood cells in your blood marrow.
Gene therapy isn’t currently in use for SCA just yet. But it may be a more viable option in the future due to the lack of need for a donor.
Other treatment options for SCA primarily involve strategies to minimize pain and help prevent complications, such as infections or joint problems.
Other treatments may include:
- blood transfusions to treat anemia
- over-the-counter or prescription pain medications
- crizanlizumab (Adakveo) to prevent blood flow blockage and reduce pain crises
- hydroxyurea, a type of chemotherapy drug used to improve anemia
- voxelotor (Oxbryta), which helps prevent red blood cells from becoming sickle-shaped
- L-glutamine to treat pain
- penicillin to decrease infection risk
The following lifestyle strategies may also help:
- avoiding high altitudes
- minimizing activities that cause low blood oxygen levels, such as extreme exercise or mountain climbing
- adopting practices to keep yourself free of illnesses, such as washing your hands often and following a recommended vaccine schedule
What is the life expectancy for people with sickle cell anemia?
The average life expectancy for people with sickle cell disease is
Currently, the only approved cure for SCA involves either a bone marrow or stem cell transplant. But doctors seldom recommend these procedures due to the risk of possible life threatening complications.