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Home Science Areas of Focus Rare Disease Sickle Cell Anemia

Managing Symptoms and Developing a Treatment Plan

Sickle cell disease, a rare hematological disease, results in misshapen red blood cells that can block the flow of blood through the body1 (called vaso-occlusive crises2), causing intense pain, as well as problems such as stroke, acute chest syndrome, joint damage, kidney issues, occasionally even heart problems.1 Learn more about Vaso-Occlusive Crisis (VOC) and Management in the report by the Sickle Cell Disease Council for Change

Learn more about the results of our national sickle cell disease poll with Howard University and the NNPA

  • Sickle cell disease is a group of inherited red blood cell diseases that causes the cells to be crescent (“sickle”) rather than disk shaped. It is traced to abnormalities in hemoglobin, the protein that carries oxygen throughout the body.1

    Patients with the disease are now living longer than they did in the past, thanks to a better understanding of how the disease works and earlier diagnosis and treatment.3 Creating a plan for managing symptoms of the disease is critical in improving patient outcome and requires close collaboration with healthcare providers. This collaboration is especially important when patients make the transition from seeing a pediatrician to seeing a doctor who treats adults.

    Sickle cell disease is usually accompanied by anemia, a condition in which you lack enough healthy red blood cells to carry oxygen to your body’s tissues.1 The defect that causes thalassemia (or other conditions such as hemoglobin C or E) can also be inherited along with the defect that causes sickle cell disease.4 However, the signs and symptoms of the disease are similar to having only anemia.1

  • Sickle cell disease is passed on genetically by carriers or people with the “trait” (those who carry only one sickle cell gene along with one normal gene and typically do not have any manifestations of the disease other than mild anemia). If both parents carry the gene, there is a 25% chance per pregnancy of having a child with sickle cell disease and a 50% chance of passing the gene on to their children.1

    In the US, an estimated 100,000 people4 have the disease and it occurs in one in 365 African Americans births.5 In other parts of the world (Sub-Saharan Africa, the Middle East, South America, Caribbean Islands, and India), the prevalence is much more, with millions of people being affected.5

  • Symptoms can vary in form and severity, depending on patient, but typically include1:

    • Serious pain
    • Fatigue
    • Shortness of breath
    • Headaches
    • Dizziness
  • Sickle cell disease is diagnosed by a blood test. In the US, all newborns are now screened for the condition before they are discharged from the hospital.6 Parents are usually given the results of this test at the child’s one-month checkup. If a child is diagnosed with sickle cell trait, his or her parents may wish to see a genetic counselor to understand what it means to be a carrier of the sickle cell gene and what the chances of having a child with the disease might be.

    If the child has sickle cell disease, they should see a hematologist (specialist in blood diseases) to better understand the type of sickle cell disease the child has and to provide optimal care.

  • A number of therapies are being developed, including gene therapy which holds potential for the future. Stem cell treatment can cure the disease for some patients, however, there is currently no widely-available cure. In cases where a patient's red blood cell count falls to a life-threatening level, blood transfusions may be needed. Bone marrow transplants have been used to treat some forms of the disease and have cured symptoms in some patients, but the transplant procedure can be risky and complex.6

    Managing symptoms is the key to helping patients live a normal life. Patients should understand the disease and their individual symptoms and track them closely, with the help of a hematologist, to understand what triggers them. Patients should see a hematologist at least twice a year and a primary care physician annually.

    Because treatment relies on clear communication, patients should visit prospective doctors and choose a physician they are comfortable with. For patients on the brink of adulthood, it is best to get referrals while they are still being treated by a pediatrician.

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1. National Organization for Rare Disorders. Sickle Cell Disease. Accessed February 10, 2020.

2. Uwaezuoke S, Ayuk A, Mbanefo N, Ezenwosu O, et al. Vaso-Occlusive Crisis in Sickle Cell Disease: Current Paradigm on Pain Management. J Pain Res. 2018;11:3141-3150.

3. Jenerette C, Lauderdale G. Successful Aging with Sickle Cell Disease: Using Qualitative Methods to Inform Theory. J Theory Constr Test. 2008;12(1):16-24.

4. Centers for Disease Control and Prevention. What is Sickle Cell Disease? Accessed February 10, 2020.

5. Centers for Disease Control and Prevention. Data & Statistics on Sickle Cell Disease. Accessed February 10, 2020.

6. NIH National Heart, Lung, and Blood Institute. Sickle Cell Disease. Accessed February 10, 2020.