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Medications and Treatments

Routine Medical Care

Routine medical care is essential for people living with sickle cell disease (SCD) to stay healthy and prevent complications. A dedicated sickle cell service often includes preventive antibiotics (usually up to age 5), routine immunizations, regular testing to look at organ health, advice on nutrition, and regular visits with a primary care physician or SCD provider. Maintaining overall wellness through methods like adequate rest, a healthy diet, and moderate exercise is also an important part of that routine care. 

For specialized support, comprehensive sickle cell centers or clinics offer coordinated care from a team of experts who understand the needs of people living with SCD. These clinics provide comprehensive care that includes diagnosis, treatment, complication management, education, and support for individuals with sickle cell disease and their families. Teams made up of a variety of specialists, including hematologists, nurses, social workers, psychologists, and others, work together to address the medical, emotional, and social needs of each patient.1 

To find a center near you or learn more about these services, visit the National Alliance of Sickle Cell Centers (NASCC) website. 

Check out the following pages for more information: 

Disease Modifying Therapies

A disease modifying therapy (DMT) is a treatment that can slow or reduce the effects of a disease. There are several common DMTs used to treat sickle cell disease. Disease modifying therapies do not cure a disease. You should talk with your doctor about any of these options to see if they might work for you. 

Transfusion2: Blood transfusions are often used to treat sickle cell disease. Normal red blood cells (RBCs) carry oxygen to the entire body, and sickle RBCs do not carry oxygen very well. Blood transfusions introduce healthy, normal RBCs, and help carry more oxygen to the body and unblock blood vessels. Receiving monthly transfusions for more than 1 year leads to iron overload. Individuals receiving regular transfusion may develop antibodies that make transfusion difficult to near impossible. 

 

Hydroxyurea3: Hydroxyurea helps the body make a special kind of hemoglobin called fetal hemoglobin (or hemoglobin F). Hemoglobin F helps red blood cells stay round and flexible, which makes them better at carrying oxygen through the body. So, people who take hydroxyurea usually experience fewer pain events, decreased hospitalization, less need for transfusion, and fewer episodes of acute chest.  

 

L-Glutamine4: L-glutamine is a medication that can help reduce the frequency of painful events in adults and children 5 years of age and older. It works by helping to prevent damage to red blood cells, so that they are less likely to burst. 

Transformative Therapies

Transformative therapies are one-time treatments with a goal of long-term effects of reducing the impact of a disease permanently. In some cases, transformative therapies can cure sickle cell disease, but data is still being collected about how the cure takes place and for how long. 

Currently, there are two accepted transformative therapies for sickle cell disease: 

 

Bone Marrow Transplant

Bone marrow transplant5 is a treatment that can cure sickle cell disease. When a person gets a bone marrow transplant, their unhealthy blood-forming cells are replaced with healthy cells from a donor. This allows the body to produce normal red blood cells instead of sickled cells. It cannot fix organ or nerve damage from sickle cell disease, but it can stop more damage from happening.  

To get a bone marrow transplant, a person needs a donor that matches their human leukocyte antigen (HLA) type, which is a marker on the cells in the body that helps the immune system recognize normal cells vs. invasive things like infections. A person can receive donated bone marrow from family members (related donors) or from someone outside of their immediate family (unrelated donors). For more information, check out the National Marrow Donor Program.  

 

Gene Therapy

Gene therapy6 is a procedure that changes specific genes to help treat diseases. Gene therapy for sickle cell disease works in a similar way to bone marrow transplant, where unhealthy blood-forming cells are replaced with healthy cells. In gene therapy, however, the healthy cells come from the person getting treated instead of a donor.  

 

Gene therapy can treat sickle cell disease by: 

  1. Removing the patient’s cells 
  2. Changing them to add or turn on non-sickle hemoglobin or to help faulty genes work properly. 
  3. Re-infusing them back into the patient. 

 

Currently, there are two types of gene therapy that are available commercially: 

 

Children’s Healthcare of Atlanta hosts a series of virtual webinars featuring information on transformative therapies for sickle cell disease. Topics include gene therapy, matched sibling and unrelated donor transplant, fertility preservation and more. For more information, click here.  

Clinical Trials

Clinical trials are used to test the safety and effectiveness of drugs and devices. There is typically a pharmaceutical company that sponsors the trial, and a team of researchers, doctors, and medical professionals conducts the trial.  Clinical trials are also overseen by several different regulatory bodies, like the Food and Drug Administration (FDA), and have strict rules about who can be involved.  

Find clinical trials near you:  

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  1. Platt AF, Eckman J, Hsu LL. General Medical Care. In: Hope and Destiny: The Adult Patient and Parent’s Guide to Sickle Cell Disease and Sickle Cell Trait. 6th ed. Hilton Publishing Company; 2023:104-129. 
  2. Children’s Healthcare of Atlanta. Sickle cell disease: Packed Red Blood Cell Transfusion. https://www.choa.org/-/media/Files/Childrens/teaching-sheets/sickle-cell-disease—packed-rbc-transfusion.pdf?la=en&hash=2F772D05D674D4397A59E7EA1D2FC4D41B117F83. 
  3. American Society of Hematology. Hydroxyurea for sickle cell disease. Hematology.org.https://www.hematology.org/-/media/hematology/files/education/hydroxyurea-booklet.pdf. 
  4. U.S. National Library of Medicine. L-glutamine: Medlineplus Drug Information. MedlinePlus. September 15, 2017. https://medlineplus.gov/druginfo/meds/a617035.html. 
  5. Children’s Healthcare of Atlanta. Sickle cell disease: blood and marrow transplant (BMT). https://www.choa.org/-/media/Files/Childrens/teaching-sheets/sickle-cell-disease–blood-and-marrow-transplant-bmt.ashx  
  6. Children’s Healthcare of Atlanta. Sickle cell disease: Gene therapy. https://www.choa.org/-/media/Files/Childrens/teaching-sheets/sickle-cell—gene-therapy.ashx 
  7. U.S. National Library of Medicine. Exagamglogene autotemcel: Medlineplus Drug Information. MedlinePlus. April 20, 2024. https://medlineplus.gov/druginfo/meds/a624018.html. 
  8. U.S. National Library of Medicine. Lovotibeglogene Autotemcel injection: Medlineplus Drug Information. MedlinePlus. April 20, 2024. https://medlineplus.gov/druginfo/meds/a624020.html. 

Indicators of an Emergency Situation

Patients and families should watch for the following conditions that need an urgent medical evaluation:
• Fever of 101° F or higher
• Chest pain
• Shortness of breath
• Increasing tiredness
• Abdominal swelling
• Unusual headache
• Any sudden weakness or loss of feeling
• Pain that will not go away with home treatment
• Priapism (painful erection that will not go down)
• Sudden vision change

Learn more about sickle cell disease

Resources from:

Arise Initiative

ASH

CDC

NIH

SCDAA