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Treatments and drugs
By Mayo Clinic staffBecause there's no cure for Hunter syndrome, treatment focuses on managing signs, symptoms and complications to provide some relief for your child as the disease progresses. Treatment may involve the following:
- Relief for respiratory complications. Removal of tonsils and adenoids can open up your child's airway and relieve sleep apnea. But as the disease progresses, tissues continue to thicken and these problems can come back. Some types of breathing devices can help with upper airway obstructions and sleep apnea. Keeping your child's airway open can also avoid low blood oxygen levels (hypoxemia).
- Addressing heart complications. Your child's doctor will want to watch closely for cardiovascular complications, such as high blood pressure, heart murmur and leaky heart valves. If your child has severe cardiovascular problems, your doctor may recommend surgery to replace heart valves.
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Treatment for skeletal and connective tissue problems. Most children with Hunter syndrome don't heal well and often have complications from surgery. That limits options for addressing skeletal and connective tissue complications. For example, surgery to stabilize the spine using internal hardware is difficult when bones are fragile.
Your child's joint flexibility can be improved with physical therapy, which helps address stiffness and maintain function. However, physical therapy can't prevent the progressive decline of mobility. Your child may eventually need to use a wheelchair because of pain and limited stamina.
Surgery can repair hernias, but because of weakness in connective tissues, results usually aren't ideal. The procedure often needs to be repeated. One option is to manage your child's hernias with supportive trusses rather than surgery because of the risks of anesthesia and surgery.
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Managing neurological complications. Problems associated with the buildup of fluid and tissue around the brain and spinal cord are difficult to address because of the inherent risks in treating these parts of the body. Your child's doctor may recommend surgery to drain excess fluids or remove built-up tissue.
If your child has seizures, your doctor may prescribe anticonvulsant medications.
- Managing behavioral problems. If your child develops abnormal behavior as a result of Hunter syndrome, providing a safe home environment is one of the most important ways you can manage this challenge. Treating behavior problems with medications has had limited success because most medications have negative effects on other manifestations of the disease.
- Addressing sleep issues. The sleep patterns of a child with Hunter syndrome become more and more disorganized, causing some children to be active around the clock. Medications including sedatives and especially melatonin can improve sleep. Keeping a strict bedtime schedule and making sure your child sleeps in a well-darkened room also can help. In addition, creating a safe environment in your child's bedroom — putting the mattress on the floor, padding the walls, removing all hard furniture, placing only soft, safe toys in the room — may help you rest easier if you know your child has less opportunity for injury.
Treatments in development
Although there's no cure for Hunter or other MPS syndromes, some treatments that are in their early stages have had some success in altering the natural course of the disease to slow its progress and lessen its severity.
These emerging treatments include:
- Bone marrow transplantation. If a healthy donor is found that matches your child's blood and tissue type, bone marrow transplantation can be used to treat some symptoms in milder forms of Hunter syndrome. Bone marrow is taken from the hip of the donor and transplanted to your child by injecting it into his or her veins (intravenously). This treatment can help ease the problems of breathing; mobility; and heart, liver and spleen function. It can also help prevent your child's mental regression. This treatment won't help with bone or vision problems.
- Cord blood transplantation. This treatment uses umbilical cord blood from an unrelated donor that's collected at the time of the donor's birth. The cord blood is transplanted intravenously. This treatment is designed to help your child's bone marrow and enzyme activity recover. It can also reduce the effects of the disease on facial and skin features, joint mobility, hearing, and enlargement of the liver and spleen.
- Enzyme therapy. This treatment uses man-made or genetically engineered enzymes, which are injected directly into your child's bloodstream to replace your child's missing or defective enzymes and ease the disease symptoms. The Food and Drug Administration gave its approval in July 2006 for the first human enzyme replacement therapy for Hunter syndrome. People involved in tests of the drug idursulfase (Elaprase) showed improvement in their ability to walk after a year of weekly treatments. The infusion has produced severe allergic reactions in some people, so your doctor will monitor your child closely if this treatment is chosen.
These therapies show promise, but they don't provide a cure. More research is needed.
