Treatments for Sickle Cell Disease
The most common treatments for complications of sickle cell disease (SCD) are hydroxyurea and blood transfusions. However, the type and severity of complications of SCD are different for everyone. So your doctor may recommend medicines, procedures, and lifestyle changes that are specific to you.
The only cure for sickle cell disease is a bone marrow transplant. This procedure is very rarely done because it is risky and requires a matched donor in the family. The goal of treatment for SCD is avoiding pain episodes, relieving symptoms, and preventing complications.
What medicines treat sickle cell disease?
Until recently, hydroxyurea was the only drug that treats SCD approved by the U.S. Food and Drug Administration (FDA). It reduces the number of pain crises and episodes of acute chest syndrome by increasing fetal hemoglobin levels and decreasing white blood cell count. It also reduces the need for blood transfusions and hospital visits.1-3
The FDA recently approved other drugs that treat SCD in children and adults. These drugs work in different ways to lower the risk for anemia and reduce the frequency of pain crises. These drugs are:4
- L-glutamine (Endari)
- Crizanlizumab (Adakveo)
- Voxelotor (Oxbryta)
What other medicines are used to prevent or treat complications?
Preventing infections in children with sickle cell disease has greatly improved childhood survival rates. Children may take penicillin or other antibiotics once diagnosed or from 2 months old to 5 years old. Children older than 5 years old and adults may also take penicillin to prevent infections, especially if they have had their spleen removed.4,5
Vaccinations are also important to prevent infections in children with SCD. They should receive all routine childhood immunizations, including the annual flu shot. They should also get the pneumococcal and meningococcal vaccines.4,6
Pain is often managed at home with acetaminophen or non-steroidal anti-inflammatory drugs (NSAIDs), such as ibuprofen. Your doctor may recommend over-the-counter versions or prescribe stronger oral pain drugs, such as opioids. Severe pain may be treated with stronger intravenous (IV) drugs in a hospital.7
Other drugs are also used, depending on your specific complications. This may include:8
- Asthma drugs
- Iron chelation therapy to prevent iron overload during blood transfusions
- Skin drugs and ointments to treat leg ulcers
- Drugs that widen your blood vessels, like ACE inhibitors, to treat kidney disease
The most common procedure done to treat sickle cell disease is blood transfusion. Doctors may use single transfusions to treat complications, such as severe anemia and acute chest syndrome. Regular blood transfusions may be used to reduce the risk of stroke or if hydroxyurea is not working.3,7,9
Blood transfusions can either be simple or exchange. A simple blood transfusion adds blood from a donor without removing any of your blood. An exchange transfusion replaces some of your blood with the donor blood. Blood transfusion risks include immune reactions to the donor blood and excessive iron buildup.4,10
Other procedures may also be used to treat specific complications of SCD. Some examples include:8,11
- Surgical removal of the spleen (splenectomy) to treat or prevent splenic sequestration
- Injections, laser treatment, or surgery to address vision loss
- Devices or surgical procedures to open the airway and treat obstructive sleep apnea
- Injections or surgery to remove blood from the penis to treat priapism (prolonged erection)
- Dialysis or kidney transplants
- Surgery to treat bone complications
- Surgical removal of the gallbladder
- Bronchodilators or oxygen supplementation to treat acute chest syndrome
Lifestyle changes to manage SCD
Many daily habits can help you stay healthy and lower your risk of complications. These include:4
- Taking folic acid supplements
- Maintaining a healthy diet
- Drinking plenty of water
- Avoiding extreme temperatures and high altitudes
- Exercising moderately but regularly
- Avoiding smoking and minimizing alcohol
- Physical therapy
- Joining support groups or counseling
Talk to your doctor about specific changes you can make, such as what to eat and how much to exercise.
Are there any new therapies being researched?
The FDA prioritizes the development of new SCD treatments. Drug companies have also invested a lot of money into researching new drugs. Many drugs are currently in clinical trials for reducing the severity and duration of acute pain crises. Talk to your doctor if you are interested in participating in a clinical trial.7,12
More ambitious approaches to treating SCD are also closer to becoming possible. An example is gene therapy, where doctors modify the mutated hemoglobin gene in your DNA. This can be done by inserting normal hemoglobin genes into your genome. Newer technology, like CRISPR, can also be used to fix the mutation directly. These techniques are only experimental right now and not available to the general public. Large trials need to establish the safety, ethics, and long-term success of these methods.13,14