Sickle cell disease (SCD) is a group of inherited red blood cell disorders. In SCD, the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle.” People with SCD can live full lives and enjoy most of the activities that other people do. If you have SCD, it’s important to learn how to stay as healthy as possible. Click on any of the links below to learn more.

Causes

SCD is a genetic condition that is present at birth. It is inherited when a child receives two genes - one from each parent - that code for abnormal hemoglobin.

Diagnosis

SCD is diagnosed with a simple blood test. In children born in the United States, it most often is found at birth during routine newborn screening tests at the hospital. In addition, SCD can be diagnosed while the baby is in the womb. Diagnostic tests before the baby is born can check for chromosomal or genetic abnormalities in the baby. Because children with SCD are at an increased risk of infection and other health problems, early diagnosis and treatment are important.

Talk to your doctor to find out how to get tested and to explain the results after testing.

Treatment

Specific Treatments to Prevent SCD Complications

SCD is a disease that worsens over time. Treatments are available that can prevent complications and lengthen the lives of those who have this condition. These treatment options and their effects can be different for each person, depending on the symptoms and severity of their disease. It is important to understand the benefits and risks of each treatment option. Currently, the FDA has approved four treatments for SCD[1].

1. Hydroxyurea (pronounced “hi-DROK-see-yoo-REE-uh”) may help people with SCD ages 2 years and older. More information about hydroxyurea can be found here.
2. L-glutamine (pronounced “L-gloo-ta-meen,”), or ENDARI® may help people with SCD ages 5 years and older. More information about L-glutamine can be found on page 2 here.
3. Voxelotor (pronounced “vox-EL-o-tor”), or OXBRYTA® may help people with SCD ages 4 years and older. More information about Voxelotor can be found on the FDA website here.
4. Crizanlizumab (pronounced “criz-an-liz-u-mab”), or ADAKVEO® may help people with SCD ages 16 years and older. More information about Crizanlizumab can be found on page 1 here.

Several other treatments and therapies for SCD have recently been developed that are still undergoing clinical trials and thus have not yet been approved by the FDA.

Specific treatments to cure SCD

Curative treatments are available for SCD, but they are not widely available yet.

1. Bone Marrow Transplant:

   Bone marrow is a soft, fatty tissue inside the center of the bones, where blood cells are made. A bone marrow or stem cell transplant is a procedure that takes healthy cells that form blood from one person—the donor—and puts them into someone whose bone marrow is not working properly (the person with SCD).

   Bone marrow or stem cell transplants are very risky and can have serious side effects, including death. For the transplant to work, the bone marrow must be a close match. Usually, the best donor is a brother or sister.
   Bone marrow or stem cell transplants are most common in cases of severe SCD for children who have minimal organ damage from the disease.

   Bone marrow transplants require chemotherapy prior to the procedure, so the treatment is difficult. It includes a lengthy hospital stay. 

   Click here to learn more about bone marrow transplants for SCD: Be the Match 
   And, if you’re healthy, consider becoming a bone marrow donor.
   (Include video of Akshay Sharma giving a crash course.)

2. Gene Therapy
   
   Scientists are developing several types of gene therapy. One type has recently been approved by the FDA. Other types are still in clinical trials. Gene therapy also requires chemotherapy. Click to learn more about gene therapy.
   (Include video of Akshay Sharma giving a crash course.)

SCD in newborns

Specialty clinics

It is important for SCD patients to be seen in specialty clinics, where doctors (hematologists) and other staff are trained specifically in SCD. Unfortunately, not everyone with SCD has access to a specialty clinic. These are the SCD treatment centers in TN. All of them take TennCare and all treat patients from a wide geographical area. Click on these center to learn more.

Memphis

• St. Jude Children’s Research Hospital
• Regional One, Diggs Kraus
• Methodist Comprehensive Sickle Cell Center

Nashville

• Meharry Medical College
• Vanderbilt

Knoxville

• Coming soon