Management of MPS VII
Overall management of disease and symptoms
MPS VII is a multisystemic, progressive disorder that causes multi-organ dysfunction1-3
- Because MPS VII is heterogeneous, a multidisciplinary team is required to help manage and treat the underlying condition1,4
- Patients with MPS VII can have varying degrees of disease severity in different areas of the body. Specialists need to be aware of how the disease affects their particular area of expertise1
- As people with MPS VII age, their signs and symptoms may worsen. In some cases, people with MPS VII may develop new signs and symptoms, which may also progress1-3
Enzyme replacement therapy
There is a recombinant form of human beta-glucuronidase available as an enzyme replacement therapy indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII.
Supportive care and treating ongoing complications should always continue.
Patients with MPS VII can have respiratory and cardiovascular complications, skeletal manifestations, loss of hearing and vision, and gastrointestinal symptoms.3
Symptomatic management may include:1,3
- Oxygen for respiratory insufficiency
- Continuous positive airway pressure (CPAP) machines
- Tracheotomy for severe airway obstruction
- Physical therapy for joint stiffness
- Cardiac valve replacement therapy
Anesthesia, surgical interventions, and emergencies
Patients with MPS VII may have cervical cord compression, cervical instability, and compressed airways. Giving an anesthetic to such patients requires skill and should always be undertaken by an experienced anesthetist.1-3
If the patient must go to a different hospital in the event of an emergency, be sure to inform the anesthetist that there may be problems with intubation. Also work with the MPS VII patient and their family to help inform their school, work, and any other caregivers of these concerns.1-3
Joint stiffness and limitation of motion are common features of MPS VII and can cause significant loss of function.
Range-of-motion exercises (passive stretching and bending of the limbs) may offer some benefits in preserving joint function, and should be started early.
References: 1. Montaño AM, Lock-Hock N, Steiner RD, et al. Clinical course of sly syndrome (mucopolysaccharidosis type VII). J Med Genet. 2016;53(6):403-418. 2. Mucopolysaccharidosis type VII. National Institutes of Health Genetics Home Reference website. https://ghr.nlm.nih.gov/condition/mucopolysaccharidosis-type-vii. Accessed August 14, 2018. 3. National MPS Society. A Guide to Understanding MPS. Durham, NC: National MPS Society. https://mpssociety.org/cms/wp-content/uploads/2017/04/MPS_VII_2008.pdf. Accessed August 14, 2018. 4. Mitchell J, Berger KI, Borgo A, et al. Unique medical issues in adult patients with mucopolysaccharidoses. Eur J Intern Med. 2016;34:2-10.