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What is Wiskott-Aldrich syndrome (WAS)?

WAS is an inherited immune system disorder. The immune system helps the body fight infections. In WAS, the immune system doesn’t work very well. Babies born with WAS may have many infections and bruise or bleed easily. 

How does blood or marrow transplant (BMT) work for WAS?

BMT, also known as a bone marrow transplant or blood stem cell transplant, is the only known cure for WAS. It replaces the unhealthy immune system with a healthy one. Blood-forming cells (stem cells) create the immune system.

Allogeneic transplant is used for WAS. This type of transplant uses healthy blood-forming cells (stem cells) donated by someone else to replace the unhealthy blood-forming cells. These healthy cells can come from a family member, unrelated donor or umbilical cord blood. The cells create the immune system. First, the child gets chemotherapy (chemo), with or without radiation, to kill the unhealthy cells. Then, the healthy donated cells are given to your child through an intravenous (IV) catheter. The new cells travel to the inside of the bones and begin to make healthy cells.

The entire transplant process, from the start of chemo or radiation, until hospital discharge, can last weeks to months. This is followed by many months of recovery near the transplant center and at home. The transplant team will closely care for your child to prevent and treat any complications.

When should my child see a transplant doctor?

Most children have a better chance of a cure if they have a transplant soon after diagnosis. Your child should see a transplant doctor as soon as your child is diagnosed.

Your child’s first appointment with a transplant doctor

At the first appointment, the transplant doctor will:

  • Review your child’s medical history
  • Talk with you about your child’s treatment options
  • Discuss the risks and benefits of transplant
  • Recommend the best time for your child to get a transplant and prepare for treatment
  • Start a donor search

Questions to ask your doctor

Ask questions so you understand your treatment options and can make decisions that are best for your child. Questions you may want to ask include:

  • What are my child’s chances of a cure with transplant? Without transplant?
  • What are the possible side effects of transplant? How can they be reduced?
  • How might my child’s quality of life change over time, with or without transplant?

Most recent medical review completed March 2017.