Hematopoietic Stem Cell Transplant—Partially Effective for Some Individuals

One treatment that can address the underlying cause of MPS VI is a procedure called hematopoietic stem cell transplant (HSCT). This term actually refers to two closely related procedures, one called bone marrow transplant and the other called cord blood transplant. HSCT may be a realistic option for some MPS VI individuals.

Our bone marrow provides our bodies with a steady supply of new red blood cells and white blood cells to replace cells that have been worn out. The basic concept of HSCT is to replace an MPS VI individual's bone marrow with marrow from a healthy person without the disease. Once the new bone marrow begins functioning, cells that it produces (specifically, the white blood cells) manufacture arylsulfatase B (ASB)—the enzyme that is missing in people with MPS VI. So the person with MPS VI gains his or her own supply of ASB enzyme.

HSCT should ideally only be performed if a donor can be found who is tissue-matched to the MPS VI individual who will receive the stem cells. In many cases, a tissue-matched donor cannot be found. HSCT is also associated with significant risks to the patient. Careful consideration should be given before choosing HSCT.

You should discuss the risks and benefits of HSCT with your physician.