What is Krabbe disease?
Krabbe is a disease you’re born with that affects metabolism. Metabolism is how the body breaks down food into energy. In Krabbe disease, the body is missing an important protein to break down fat-based substances in the body. When the fat-based substances aren’t broken down, they build up and cause damage. In Krabbe disease, the myelin sheath, which protects nerve cells, is damaged. Then, nerves in the brain and throughout the body don’t work properly.
Children with Krabbe disease have difficulty with memory, learning, speaking, understanding, and other mental functions. They also have problems with movement, including muscle weakness and stiff joints.
How does blood or marrow transplant (BMT) work for Krabbe disease?
BMT, also known as a bone marrow transplant or blood stem cell transplant, can stop the Krabbe disease from causing more damage. It replaces the blood-forming cells (stem cells) that are missing the important protein with healthy ones. With healthy blood-forming cells, your child will be able to break down fat-based substances normally. This keeps your child from having more damage to nerve cells. But, transplant can’t fix any damage that has already happened.
Allogeneic transplant is used for Krabbe disease. This type of transplant uses healthy blood-forming 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. 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?
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 you and your child. Questions you may want to ask include:
- What are the chances transplant will stop the Krabbe disease from getting worse?
- 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.