Symptom Management

Symptomatic treatments play an important role both in managing disease complications and improving quality of life, even in patients receiving disease-specific therapy.

Some of the symptomatic treatments used in the management of MPS I and MPS II are shown in the table below.1,2

Symptom management in MPS I/MPS II

Condition Treatment
Cardiovascular manifestations
Hypertension Angiotensin-converting enzyme inhibitors, angiotensin receptor blockers, diuretics, calcium-channel blockers
Arrhythmias Ablation, antiarrhythmic drugs,  anticoagulants, placement of an implantable cardioverter defibrillator
Neurological manifestations
Behavioural problems Behavioural therapy and/or behaviour-modifying medication
Seizures Anticonvulsants
Ocular manifestations
Glaucoma Intraocular pressure-lowering eye drops
Refractive errors Prescription of corrective lenses
Musculoskeletal manifestations Physiotherapy, use of orthopaedic devices (e.g. orthotic footwear, braces, corsets and walking aids)
Hearing loss secondary to persistent middle ear effusion Hearing aids or insertion of ventilation tubes
Airway abnormalities
Obstructive sleep apnoea
Upper airway obstruction
Nocturnal supplemental oxygen. CPAP, BiPAP
Pain caused by gingival cysts Analgesics, antibiotics, gum massage

 

References

  1. 1.Scarpa M, Almassy Z, Beck M, et al. (2011) Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease. Orphanet J Rare Dis 6: 72.
  1. 2.Muenzer J, Wraith JE, Clarke LA, International Consensus Panel on M, Treatment of Mucopolysaccharidosis I. (2009) Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics 123(1): 19-29.