Sickle Cell Disease

Hypothetical Patient Profile

Learn more about the disease and the patient burden

Sickle cell disease (SCD) is the most common inherited blood disorder. More than 20 million people worldwide suffer from SCD, including approximately 100,000 individuals in Europe.1,2 The worldwide incidence is estimated to be between 300,000 and 400,000 neonates globally each year. About 100,000 Americans live with SCD3, with African Americans being the group most often affected by the disease – one out of 365 African Americans are born with SCD.4 When sickled cells travel through small blood vessels, they can get stuck and obstruct the blood flow. Pain is the most common complication of SCD, and the number one reason that people with SCD go to the emergency room or hospital.5

Red blood cell transfusions are frequently used to treat anemia, acute chest syndrome, stroke, and pain, four complications associated with SCD.6 The number of RBC units transfused varies based on each patient’s need, ranging from occasional transfusions to treat a “pain crisis” to once or twice a month to prevent strokes.7 Blood Transfusions can have serious complications, such as iron overload, a condition by which iron builds up in the body, causing life-threatening damage to the organs.8

1. National Heart, Lung and Blood Institute, NIH. “Cure sickle. Cell Initiative.” Accessed October 2020. 2. Modeling based on epidemiology, demographics, mortality. 3. MedlinePlus. “Sickle cell disease.” Accessed October 2020. 4. Cincinnati Children’s Hospital. “Four Things to Know About Sickle Cell Disease.” Accessed October 2020. 5. Centers for Disease Control and Prevention (CDC). Accessed October 2020. 6. Froedtert & Medical College of Wisconsin. “Blood Transfusions to Treat Sickle Cell Disease.” Accessed October 2020. 7. Sickle Cell Disease News. “Blood Transfusion.” Accessed October 2020. 8.Centers for Disease Control and Prevention (CDC). “Complications and Treatments of Sickle Cell Disease.” Accessed October 2020.

Hypothetical Profile

Jenna has experienced several pain crises and is always fearful of having another one. She does not really understand why this is happening to her.

Since having her first stroke at the age 3, Jenna receives chronic blood transfusions to deal with her disease. She relies on her mother and doctors to provide the best care possible to prevent and alleviate the symptoms of her disease. Every 3-4 weeks she has to go to the outpatient clinical for a blood transfusion which takes her away from school and friends.

Kids wonder why she has to go to the hospital so much. She just wants to have fun and be like the other kids.


  • Experienced a stroke
  • Frequent pain crises
  • Gall bladder surgery


  • Hydroxyurea
  • Exchange blood transfusions
  • Daily vitamins (including folic acid)
  • Drink plenty of water


  • Prevent from having another stroke
  • Reduce the number of transfusions
  • Ability to keep playing soccer


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