What causes sickle cell disease?
A gene mutationmutationAn abnormal change in a gene that causes it to malfunction that produces abnormal hemoglobinhemoglobinA protein in your red blood cells that carries oxygen throughout your body, which can lead to a
lifelong genetic disease marked by unpredictable, progressive symptoms.
lifelong genetic disease marked by unpredictable, progressive symptoms.
For a deeper understanding, let’s take a look inside the body.
Understanding how a normal red blood cell works and what causes it to sickle can help when making decisions with your doctor on how to treat it.
Normal red blood cell
Sickled red blood cell
- When a genegeneA sequence of DNA responsible for controlling inherited traits called the HBB gene changes inside your body, it can cause red blood cellsred blood cellsA hemoglobin-containing cell that carries oxygen throughout your body to make an abnormal form of hemoglobin called sickle hemoglobinsickle hemoglobinAn abnormal form of the hemoglobin protein including red blood cells that are sickled (or half-moon shaped) (HbS) instead of normal adult hemoglobin (HbA)
- Repeated blockages and damage to the blood vessels over time can prevent your organs and tissues from getting the oxygen they need. This can cause vaso-occlusive events (VOEs)vaso-occlusive events (VOEs)In a LYFGENIA clinical study, VOEs were defined as any of the following events requiring evaluation at a medical facility: an episode of acute pain with no medically determined cause other than vaso-occlusion, lasting more than 2 hours, acute chest syndrome, acute hepatic sequestration, or acute splenic sequestration
Current disease-modifying therapies do not address the disease at the genetic level.
Common sickle cell disease
treatment options
(eg, disease modifying therapies (DMTs), oral and IV pain
medication, and blood transfusions)
Common sickle cell disease treatment options
(eg, disease modifying therapies (DMTs), oral and IV pain medication, and blood transfusions)
- These treatment options are designed to help manage sudden and severe symptoms
- These treatment options aim to decrease the frequency and severity of symptoms, and they usually require lifelong use
Allogeneic stem cell transplants
(also called bone marrow transplants or stem cell transplants)
Allogeneic stem cell transplants
(also called bone marrow transplants or stem cell transplants)
- These are one-time treatments for people with sickle cell disease. They’re usually recommended for people under 16 with a sibling donor who is a match and is available
- Allogeneic stem cell transplants may also carry risks of donor-related complications such as graft-versus-host disease
More topics to explore
More topics to explore