The goals of managing MPS II are to improve quality of life, to slow down the progression of the disease, and to prevent permanent tissue and organ damage. Currently there is no cure for MPS II. However, early intervention may help prevent irreversible damage. Treatment options for MPS II include those aimed at disease management and supportive or palliative care (care that makes a person with a disease that cannot be cured more comfortable), as well as those aimed at treating the underlying enzyme deficiency.
Hematopoietic Stem Cell Transplant (HSCT) and Bone Marrow Transplant
For some years HSCT has been used to treat children with MPS. Some children with MPS I have benefited from HSCT, but this procedure currently is not recommended for individuals with MPS II. HSCT in MPS II has not been shown to have any effect in preventing the damage to the brain that occurs with severe MPS II.The National MPS Society may be able to put you in touch with parents whose children with MPS II have had this treatment so you may be better informed.
Enzyme replacement therapy (ERT)
ERT for MPS II was approved by the FDA in 2006. Elaprase® is a manufactured version of the body’s natural iduronate sulfatase enzyme. Elaprase improves lung function, endurance, reduces the size of the liver and decreases the levels of GAG in the urine. It does not cross the blood-brain barrier at normal doses and thus is not anticipated to have an impact on any neurocognitive decline occurring in individuals with MPS II. Treatments of Elaprase are given weekly through intravenous infusions. For parents to fully understand the risks, benefits and limitations of ERT, it is important to talk with physicians familiar with MPS II ERT and families undergoing this treatment. The National MPS Society can put you in touch with physicians and families so you can become better informed before reaching a decision.