Sickle Cell Disease Treatment and Survival Rate

November 25, 2024

This page was reviewed under our medical and editorial policy by YunZu (Michele) Wang, M.D., assistant professor, Department of Pediatrics, City of Hope® Cancer Center Duarte

Several safe and effective treatment options are available that may ease the symptoms of sickle cell disease and treat the underlying causes of this genetic blood disorder.

Who Treats Sickle Cell Disease?

The patient’s care team may consist of the following experts:

  • Hematologists, who are experts in treating disorders of the blood
  • Geneticists, who help with genetic evaluations of sickle cell disease traits
  • Pathologists, who evaluate blood samples as part of the diagnosis and management process
  • Specialized pharmacies, which supply medications that are difficult to find elsewhere, especially for hard-to-treat conditions
  • Supportive medicine providers, who manage sickle cell- and treatment-related side effects and symptoms

How Is Sickle Cell Disease Treated?

Doctors may recommend one sickle cell disease treatment or a combination of several treatments, depending on the severity and type of sickle cell disease a patient has. Some of the more common treatment options include medications and blood transfusions. However, advanced gene therapy and bone marrow transplants may also be recommended for certain patients.

Sickle Cell Disease Medication

A range of medications are available to treat various aspects of sickle cell disease. These include:

Hydroxyurea, an oral medicine pill, can help alleviate more serious symptoms such as sickle cell crisis, chest pain and the need for hospitalization. It can be prescribed for children and adults, but cannot be taken during pregnancy.

L-glutamine, a type of pain medication, can reduce severe symptoms of sickle cell disease and manage sickle cell pain. It can be taken by patients aged 5 and over.

Crizanlizumab-tmca is an intravenous medication, which can be prescribed for people 16 and older. It’s given once a month under clinical supervision and is designed to reduce sickling in the red blood cells in order to prevent blockages.

A hematologist or other physician may also recommend over-the-counter or prescription pain medication, as well as antibiotics and vaccines to help manage the risk of infection.

Blood Transfusion

In some cases, patients with sickle cell disease may undergo a blood transfusion. This procedure, performed intravenously under a clinician’s supervision, helps boost the number of healthy red blood cells in a patient’s body, helping manage and reduce the effects of sickle cell disease.

Doctors may recommend blood transfusions for patients whose symptoms don’t get better with medication alone or for those who are at higher risk of complications of their disease.

Red Blood Cell Exchange

Some people with sickle cell disease may benefit from apheresis. During this procedure, the patient’s blood is exchanged for a donor’s healthy red blood cells to reduce the volume of sickled blood cells from the body.

Apheresis is typically performed on an outpatient basis. During the session, the patient will have an intravenous (IV) line placed in one arm to remove his or her blood. In the other arm, another IV will be placed to infuse the patient with donor blood cells.

Bone Marrow Transplant

Bone marrow transplants, also called hematopoietic stem cell transplants, are another treatment for sickle cell disease. This type of transplant may be able to cure the condition if the patient has a suitable bone marrow donor, most often a sibling or other close relative. However, the bone marrow transplant procedure has several possible side effects and, in rare cases, is not always curative.

How Does a Bone Marrow Transplant Treat Sickle Cell Disease?

A bone marrow transplant treats sickle cell disease by providing the patient with a transfusion of healthy blood stem cells from a matching donor. The procedure involves these steps:

  • Before the transplant, the patient is given radiation therapy or chemotherapy that “conditions” the bone marrow to make space for donor blood cells.
  • The donor is given a medicine that stimulates the production of more healthy stem cells.
  • Doctors then collect the healthy donor stem cells and transfuse them into the patient’s blood.
  • The donor stem cells travel to the “space” in the patient’s bone marrow, where they can begin to produce healthy red blood cells.

Sickle Cell Anemia Gene Therapy

A newer approach to treating sickle cell disease is gene therapy, which involves introducing changes to the DNA inside the cells that produce hemoglobin. By treating sickle cell disease on a genetic level, gene therapy can improve or, in most cases, cure the condition.

There are currently two approved gene therapies for sickle cell disease, both of which are given intravenously and are developed using cells from the patient’s own body. They are:

Exagamglogene autotemcel (Casgevy®), which introduces a modified gene that instructs the body’s cells on how to produce normal red blood cells

Lovotibeglogene autotemcel (Lyfgenia®), which works by changing an existing gene in the body so that it produces normal red blood cells

Both gene therapy options can be prescribed to patients aged 12 and over.

Gene therapy for sickle cell disease involves steps that are similar to that of the bone marrow transplant process, including using a drug (typically chemotherapy) for conditioning.

Sickle Cell Crisis Treatment

Sickle cell pain crisis refers to sudden, serious pain experienced by someone with sickle cell disease. It can affect any part of the body, but it is most common in the arms and legs, joints and chest. It’s caused by misshapen red blood cells blocking the bloodstream.

Because it can lead to serious complications, people experiencing a sickle cell crisis should alert their care team right away. The care team can help the patient determine whether symptoms should be managed at home or if he or she should seek expert medical care.

Survival and Prognosis

Studies have shown that Americans with sickle cell disease have a life expectancy that’s more than 20 years shorter than the average. This is because of the potential for serious and sometimes life-threatening effects of the condition, including blockages in the blood vessels leading to acute pain, as well as higher risk of heart health issues and problems with the kidneys and liver.

The most important aspect of improving survival rates for those with sickle cell disease is to ensure that people are screened for the condition and have access to care to manage the symptoms of sickle cell disease.

Steps if Interested in Gene Therapy at City of Hope

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Contact — Reach out to City of Hope by phone or email for more information.

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Consult — Meet with a provider at City of Hope to learn about gene therapy.

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Consent to treatment for Gene Therapy.

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Collect the cells and send them out to gene editing. This takes about 6 months.

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Complete a preconditioning regimen and then infuse the cells.

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Cells that have been edited will take some time to engraft. Patient will be monitored for side effects and followed by a provider.

References
References
  • National Heart, Lung, and Blood Institute (2024, September 30). Sickle cell disease treatment. 
    https://www.nhlbi.nih.gov/health/sickle-cell-disease/treatment

  • Han H., Hensch L., et al. Hematology ASH Education Program (2021, December 10). Indications for transfusion in the management of sickle cell disease. 
    https://ashpublications.org/hematology/article/2021/1/696/482990/Indications-for-transfusion-in-the-management-of

  • Borhade M, Patel P, et al. StatPearls (2024, February 25). Sickle cell crisis. 
    https://www.ncbi.nlm.nih.gov/books/NBK526064/

  • Jared Kaltwasser, AJMC (2023, March 24). Sickle cell disease cuts 20 years from life expectancy, study finds. 
    https://www.ajmc.com/view/sickle-cell-disease-cuts-20-years-from-life-expectancy-study-finds

  • Centers for Disease Control and Prevention (2024, May 15). Data and statistics on sickle cell disease. 
    https://www.cdc.gov/sickle-cell/data/index.html