STEM CELL TRANSPLANT

 
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What is a stem cell transplant?

A stem cell transplant (also known as a bone marrow transplant) replaces your own stem cells that produce abnormal blood cells (such as red blood cells that contain sickle hemoglobin) with healthy ones from another person (called a donor).

 
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Hematopoietic (or blood forming) stem cells are the mother cells that usually live in the bone marrow. They can be also be found in the umbilical cord (the cord that connects a baby to its mother in the womb).

Stems cells can become BLOOD CELLS. They can become:

  • Red blood cells: carry oxygen through the body

  • White blood cells: Fight infection

  • Platelets: help blood clot and prevent excess bleeding.

There are 3 types of stem cell donors:

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Matched related

A brother or sister who has the same bone marrow type and the same mother and father as you.

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Matched unrelated

Volunteers who are unrelated to you, but have the same bone marrow type as you.

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Haploidentical

Partially matched family member (usually a mother or father). This type is still considered experimental.

Is a stem Haploidenticalcell transplant the right choice for you?

Adult patients

A transplant may be recommended if you have had one or more complications below, suggesting that you have severe sickle cell disease:

  • You had a stroke.

  • You had 2 or more episodes of acute chest syndrome in the past year.

  • You have frequent pain despite taking hydroxyurea or getting blood transfusion on a regular basis

  • You have pulmonary hypertension (high pressure in your lungs).

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Children

Most children with sickle cell disease will live to young adulthood, so a transplant may be a reasonable option for a child who has had:

  • A stroke

  • Serious problems with the lung, kidney, or heart from sickle cell disease that is getting worse even with medical treatment.

Risks of a transplant

 

  • A transplant can lead to severe complications or even death, so it is not a good option for everyone. The risk of death from transplant in the first two years after treatment is higher for children than the risk of death from sickle cell disease.

  • At this time, stem cell transplant and gene therapy are the only known cures for sickle cell disease. Both are still experimental. We also do not know the short- and long-term complications of stem cell transplants and gene therapy.

 
 
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Stem cell transplant checklist

For you and your health care provider

 

Is it right for you?

🄯 Talk with your provider to see if a stem cell transplant is right for you.

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SECTION OVERVIEW

  • At this time, a stem cell transplant is the only known cure for sickle cell disease.

  • A stem cell transplant is not the best treatment for everyone who has sickle cell disease.

  • All of the long-term side effects of a stem cell transplant are not yet known.