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Sickle Cell & Transplant

What is sickle cell disease (SCD)?

Sickle cell disease (SCD) is a group of blood disorders passed from parents to children through genes. It is the most common inherited blood disorder affecting over 100,000 people of all ages in the U.S. It is primarily found in individuals of African, Mediterranean, South American, Southeast Asian and Middle Eastern descent.

 

SCD causes your body to make unhealthy red blood cells. Healthy red blood cells are round and flexible. They move through your small blood vessels and bring oxygen to all parts of your body. With SCD, your red blood cells are misshapen, hard and sticky. They get stuck in your blood vessels and clog them. This can cause you to have pain, infection, organ damage, low blood count, strokes and other serious health problems. This leads to life-long morbidity and reduced life expectancy through end-organ damage.

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What treatment is available?

SCD affects each person differently, so there is no “best treatment” for everyone with SCD. Hydroxyurea, L-glutamine, voxelotor and crizanlizumab-tmca are the only approved medicines for SCD. They can help lower the number of pain crises, but they do not cure SCD.

 

Other options to reduce SCD symptoms include:

  • Pain Medicine

  • Blood Transfusions

  • Medicines to prevent blood clots

  • Antibiotics

These treatments will not cure the disease but may help you feel better.

Is there a cure?

A blood or marrow transplant (BMT) is the only established cure for SCD. BMT is not surgery. It’s a treatment that takes healthy blood-forming cells from a donor and gives them to you through an intravenous (IV) infusion to replace the unhealthy cells. It can’t reverse organ damage from SCD, but it can stop more damage from happening.

How does a BMT work?

There are several steps in the blood or marrow transplant process. Watch these videos to learn how transplant can cure SCD and other diseases.

Who can get a transplant?

The decision to go to transplant is one that is made between you, your family and your doctors. Transplant may or may not be the best option for you. For people with an available donor, doctors might recommend transplant if you have any of the following:

  • More than 3 severe vaso-occlusive pain crises in a 2-year period

  • Stroke or silent stroke or problems with your mental ability for more than 24 hours

  • More than 2 episodes of acute chest syndrome in a 2-year period or recurrent acute chest syndrome

  • 8 or more red blood cell transfusions in 1 year

  • Chronic pain lasting longer than 6 months

  • Silent cerebral infarct

  • Tricuspid value regurgitant jet (TRJ) velocity exceeding 2.6 m/sec

  • Abnormal transcranial Doppler (TCD) velocity of more than 200 cm/sec or more than 185 cm/sec with intracranial vasculopathy

What are the risks and side effects?

Although transplant can cure SCD, it also has risks. Some people have minimal complications from transplant, while others have more serious complications that require treatment or hospitalization. Possible complications include:

  • Your body rejecting the new cells

  • Infections

  • Infertility

  • Temporary hair loss

  • Graft-versus-host disease (GVHD)

  • Death

The process of transplant can also be a difficult mental and emotional process.

 

Talk to a transplant doctor to learn more about your risks. Your family and care team should weigh the risks and benefits together to decide if a bone marrow transplant might be right for you.

Questions to ask your doctor

Ask questions so you understand your treatment options and can make decisions that are best for you. You are an active participant in the decision-making process with your care team. Question you may want to ask:

  • Is transplant an option for me?

  • What are the chances that transplant will cure my SCD?

  • What are the risks of waiting or trying other treatments before transplant?

  • Does my age affect the risks of transplant?

  • What are the possible side effects of transplant? How can they be reduced?

  • How might my quality of life change over time, with or without transplant?

What other treatments are available?

Doctors and researchers are studying new treatments for SCD. One possible cure is called gene therapy. Right now, gene therapy is only available through a clinical trial. Learn more about gene therapy, other treatments and find clinical trials.