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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.

Sickle Cell Illustration of red blood cells

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.​

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Other options to reduce SCD symptoms include:

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  • Pain Medicine

  • Blood Transfusions

  • Medicines to prevent blood clots

  • Antibiotics

 

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

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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.

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Transplant is a journey. The goal is to help you live free from sickle cell pain, prevent new complications, and improve your quality of life.

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How does a BMT work?

 

There are several steps in the blood or marrow transplant process. Visit the Resources page to learn vital information about the transplant process and download helpful handouts.

Steps in the blood or marrow transplant process infographic

Who can get a transplant?

 

It’s important to talk with your doctor about whether or not transplant is the best treatment option for you right now. Doctors might recommend transplant if you had:​ 

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  • More than 3 severe pain crises in the last 2 years 

  • A stroke or silent stroke 

  • Acute chest syndrome 2 or more times in the last 2 years 

  • 8 or more red blood cell transfusions in a year 

  • Pulmonary hypertension 

  • Chronic pain lasting longer than 6 months 

  • Ultrasounds that show you may be at a high risk for having a stroke 

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What are the risks and side effects?

 

Transplant does come with risks. Every person will have a different experience. Your experience may depend on your age, your health, and how well your donor matches you. Some side effects can happen early after transplant, while others can happen years after transplant.

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Some risks of transplant include:

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  • The donated cells don’t make the new white blood cells, red blood cells and platelets you need (graft failure) • Graft-versus-host disease (GVHD), when the donated cells fight your body

  • Infections

  • Bleeding

  • Seizures

  • Not being able to have kids (infertility)

 

Sometimes there are serious complications that can result in death. It is important to talk to a transplant doctor about your risk, potential side effects and complications. You, your family and care team can weigh the risks and benefits together to decide if BMT is right for you.

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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. Questions you may want to ask:

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  • 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?

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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.

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