A bone marrow or cord blood transplant is the only treatment that has a stabilizing effect on the problems caused by globoid-cell leukodystrophy (GLD), or Krabbe disease. For some patients it can be life-saving and prevent severe disability. The transplant replaces the patient’s blood-forming cells missing the important enzyme with healthy ones from the donor that contain the enzyme.
The type of transplant used for GLD 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, prior to transplant 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 can make the enzyme the body was missing.
The entire process, from the start of chemotherapy until hospital discharge, can last weeks to months followed by many months of recovery at home.
Understanding if transplant would help your child with GLD
Our patient services coordinators can answer your questions and provide support and education to help you navigate your transplant journey.
Because children with untreated GLD have mental and neurologic problems that get worse over time, it is best to have a transplant as soon as possible after diagnosis. Children who get a transplant early, before a lot of damage has happened, can have normal or near-normal mental development.
Transplants for late-onset GLD have had better results than transplants for early-onset GLD. This is because late-onset GLD gets worse more slowly than early-onset GLD. It takes months for the transplanted cells to make enough healthy cells for the body. The disorder can continue to cause damage during that time.
Transplants for children with early-onset GLD who have already developed severe damage have had poor results. So it’s important to move forward with transplant as quickly as possible after diagnosis. For parents who know that GLD runs in the family, children can be tested at birth. Transplant can be done soon after to get the best possible results. If the disease was not known in the family before, it may take some time to diagnose. Moving forward with transplant as soon as possible helps to minimize permanent damage.
For these reasons, doctors recommend that children with GLD be referred to a transplant doctor as soon as they are diagnosed.1 A transplant doctor who is an expert in GLD can explain the risks and benefits of transplant.
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