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The treatment you receive for sickle cell disease will depend on your specific symptoms. Possible treatments include blood transfusions, medications such as hydroxyurea, and emerging therapies.
Sickle cell disease (SCD) is a genetic blood disorder that affects hemoglobin, the oxygen-carrying protein in red blood cells (RBCs). SCD causes RBCs to become rigid, sticky, and misshapen (sickle-shaped). These cells tend to build up in smaller blood vessels and can slow or block blood flow. This can lead to pain, infections, and other serious health problems.
A cure for SCD is not an option for most people. But treatments are available to reduce the symptoms, lower the risk of complications, and extend your life. Let’s explore the main treatments for SCD, how they work, their benefits, and their potential risks.
Types of sickle cell disease
Many people use the terms “sickle cell disease (SCD)” and “sickle cell anemia (SCA)” interchangeably. However, SCA is one type of SCD. It’s the most common and most severe type. It’s also known as hemoglobin SS disease (Hb SS).
Other types of SCD include:
hemoglobin SC disease (Hb SC)
hemoglobin SD disease (Hb SD)
hemoglobin SE disease (Hb SE)
sickle cell thalassemia
hereditary persistence of fetal hemoglobin-sickle cell disease syndrome (HPFH)
Medications for sickle cell disease
Medications are usually the primary treatment for SCD. They help reduce the frequency and severity of painful episodes known as sickle cell crises.
The Food and Drug Administration (FDA) has approved four SCD medicationsTrusted Source to date:
hydroxyurea
voxelotor
L-glutamine
crizanlizumab
Hydroxyurea is one of the most commonly prescribed medications. It works by encouraging the production of fetal hemoglobin, which can improve the shape and function of your RBCs. As a result, it can help reduce the frequency of pain crises and the need for blood transfusions.
However, hydroxyurea comes withTrusted Source many possible risks and side effects, such as:
stomach issues
mouth ulcers
decreased white blood cell count, which may increase infection risk
In rare cases, long-term use of hydroxyurea can cause cancer.
Blood transfusions for sickle cell disease
Blood transfusions are another common treatment for SCD. They help increase the number of healthy RBCs, which can prevent serious complications such as stroke.
Simple transfusions involve receiving RBCs from a donor. This type of transfusion can quickly increase oxygen delivery in your body and relieve severe anemia (low RBC levels).
Exchange transfusions are a bit more complicated. During an exchange infusion, some of your blood is removed and replaced with donor blood to improve the ratio of sickle RBCs to healthy RBCs.
While blood transfusions can be lifesaving, they come with many serious risks, including a risk of iron overload, which can damage your organs over time, and the risk of an immune reaction to donor blood.
Because of the potential risks, doctors recommend blood transfusions only in specific situations, such as when you have severe anemia or before major surgeries.
Stem cell transplants for sickle cell disease
A stem cell or bone marrow transplant offers a potential cure for SCD. This procedure involves replacingTrusted Source the ineffective blood-forming stem cells in your bone marrow with healthy ones from a donor.
Stem cell transplants have roughly an 85% success rateTrusted Source in curing SCD, but they carry serious risks.
Most importantly, there is a risk of potentially life threatening graft-versus-host disease (GVHD), a condition in which the new stem cells attack your body. Additionally, the procedure requires high doses of chemotherapy to destroy your existing bone marrow, which can have severe side effects.
Because of the risk of GVHD, stem cell transplants are generally used only in young children with severe SCD. This is because children are less likely to have extensive organ damage after the procedure.
Gene therapy for sickle cell disease
Gene therapy is an emerging treatment that holds promise for curing SCD by directly targeting the genetic cause of the disease. In gene therapy, doctors harvest and modify your stem cells to correct (“edit”) the genetic mutation that causes SCD. They then use the stem cell transplant procedure to deliver the corrected cells back into your body.
In 2023, the FDA approvedTrusted Source the first two gene therapies for SCD. The procedure is much safer than a conventional stem cell transplant because it doesn’t come with the risk of GVHD. However, it involves chemotherapy, which can cause severe side effects.
Complementary and alternative treatments for sickle cell disease
Some people with SCD explore complementary and alternative therapies to help manage their symptoms and improve their quality of life. These treatments include:
Hydration and nutrition: Staying well hydrated and eating a nutritious diet can help manage SCD symptoms and prevent complications.
Supplements: Healthcare professionals often recommend supplementing with specific vitamins and minerals, such as folic acid, to help your body produce RBCs. But more researchTrusted Source is needed to confirm their effectiveness.
Cannabis:
Some studiesTrusted Source have reported an improvement in pain symptoms in people with SCD who use cannabis. However, medical cannabis is not FDA-approved for SCD and is still not available or legal in many areas.
Other alternative treatments: Some people find relief from pain and stress through herbal remedies, yoga, or acupuncture, although more research is needed in this area.
Always consult a healthcare professional before trying any alternative treatments, as these treatments may interact with your current medications or affect your health.