A blood or marrow transplant is the only treatment that can stop the worsening effects of adrenoleukodystrophy (ALD). Transplants are only used in patients with the most severe form of ALD.
The type of transplant used for ALD is an allogeneic transplant. This type of transplant uses healthy blood-forming cells from a family member, unrelated donor, or umbilical cord blood unit.
For an allogeneic transplant, a patient gets chemotherapy, with or without radiation, to prepare his or her body for the treatment. Then, the replacement cells are infused into the patient’s blood stream. From there, the cells find their way into the bone marrow, where they start making healthy white blood cells, red blood cells and platelets. These new cells help the body break down the fatty acids and stop further damage to the body.
The entire process, from start of chemotherapy or radiation until hospital discharge, can last weeks to months followed by many months of recovery at home.
Understanding if transplant would help your child with ALD
Our patient services coordinators can answer your questions and provide support and education to help you navigate your transplant journey.
Because children with the severe form of ALD have physical and mental problems that get worse over time, it is best to have a transplant as soon as possible. Children who get a transplant early enough can have normal or near-normal mental and physical growth.
However, transplants for children who have already developed severe damage have had disappointing results. If ALD has caused a lot of damage, a child has a higher risk of having serious complications from transplant. Also, a transplant may not undo damage the disease has already done.
For these reasons, doctors recommend that children with ALD be referred to a transplant doctor as soon as they are diagnosed. A transplant doctor can explain the risks and benefits of transplant.
Learn more about bone marrow transplants and access resources to help you navigate your transplant journey.
1Recommended Timing for Transplant Consultation. Guidelines developed jointly by National Marrow Donor Program/Be The Match and the American Society for Blood and Marrow Transplantation (ASBMT). Available at: marrow.org/md-guidelines