A bone marrow or cord blood transplant is the only treatment that can stop the effects of Hurler syndrome at this time. A bone marrow or cord blood transplant begins with chemotherapy, with or without radiation, to destroy the diseased cells and marrow. The transplant replaces diseased blood-forming cells with healthy ones.
The type of transplant used for Hurler syndrome 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 red blood cells, white blood cells and platelets. These new cells have the enzyme needed to break down GAG 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 Hurler syndrome
Our patient services coordinators can answer your questions and provide support and education to help you navigate your transplant journey.
Because children with untreated Hurler syndrome 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 development, and damage to the organs is stopped. Some physical problems, except for those affecting the skeleton and eyes, may also be improved.
However, transplants for children who have already developed severe damage have had poor results. If the disorder has caused a lot of organ damage, a child has a higher risk of developing life-threatening complications from transplant. In addition, a transplant may not undo damage the disease has already done to the body.
For these reasons, doctors recommend that children with Hurler syndrome be referred to a transplant doctor as soon as they are diagnosed.1 A transplant doctor who is an expert in Hurler syndrome 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