This website is intended for Healthcare Professionals practicing in Europe, Canada, Australia and New Zealand. Developed and funded by BioMarin.

Managing MPS

Coordinated care for your patients: driving optimal outcomes in MPS management

Coordinated, multidisciplinary care is critical in the management of complex multisystemic genetic diseases1

Decades of ongoing research and clinical experience have produced a new era in the optimal management of mucopolysaccharidosis (MPS) disorders. This rapidly evolving standard of care for MPS relies on the geneticist at the centre of a healthcare delivery model that embodies coordinated, multidisciplinary care and provides physicians unmatched opportunities to change patients’ lives.1–3

Optimising patient outcomes requires coordinated care.1–3

MPS care team diagram

Facilitating efficient coordination of clinicians across specialities3

The heterogeneous and variable nature of MPS disorders necessitates a personalised approach to coordinated patient care.4 The aim of coordinated care is to help patients achieve a greater quality of life, which includes:

  • More active lives with MPS disorders
  • Sustained ability to achieve activities of daily living
  • Empowerment through participation in optimising their own outcomes3

For paediatric patients with chronic, complex, multisystemic genetic diseases such as MPS, care through a coordinated approach is associated with reduction in healthcare utilisation and improved health outcomes.5–8

Prompt diagnosis and comprehensive multisystemic baseline evaluation at diagnosis and prior to enzyme replacement therapy (ERT) with a coordinated team are critical first steps toward establishing an individualised management plan.1

Coordination must be implemented across all elements of the broader healthcare system (e.g. speciality care, hospitals, home healthcare and community services) and within patients’ individualised management plans.3 Your role can be essential to the implementation of best practices in the management of MPS disorders and improvement of patient outcomes.

To address the underlying cause and multisystemic complications of MPS, management guidelines for certain MPS sub-types call for a coordinated approach consisting of a team of specialists anchored by a geneticist or metabolic physician.

benefits table FPO

Progress and promise through three pillars of care

In the new era of MPS, the many aspects of disease management may be grouped into three pillars of care designed to optimise patient outcomes.

Application of optimal MPS disease management, grouped into the following three pillars of care, can help to improve patient outcomes:

  • Treatment with ERT, if available9
  • Lifetime management
  • Best practices in procedural care
3 pillares FPO

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  1. Hendriksz CJ et al. International guidelines for the management and treatment of Morquio A syndrome. Am J Med Genet Part A 2014;9999A:1–15.
  2. Muenzer J. The mucopolysaccharidoses: a heterogeneous group of disorders with variable pediatric presentations. J Pediatr 2004;144(suppl 5):S27–S34.
  3. Agency for Healthcare Research and Quality. Defining the PCMH. Accessed December 15, 2015.
  4. Hendriksz CJ et al. Review of clinical presentation and diagnosis of mucopolysaccharidosis IVA. Mol Genet Metab 2013;110:54–64.
  5. Casey PH et al. Effect of hospital-based comprehensive care clinic on health costs for Medicaid-insured medically complex children. Arch Pediatr Adolesc Med 2011;165(5):392–398.
  6. Mosquera RA et al. Effect of an enhanced medical home on serious illness and cost of care among high-risk children with chronic illness: a randomized clinical trial. JAMA 2014;312(24):2640–2648.
  7. Klitzner TS et al. Benefits of care coordination for children with complex disease: a pilot medical home project in a resident teaching clinic. J Pediatr 2010;156(6):1006–1010.
  8. Gordon JB et al. A tertiary care-primary care partnership model for medically complex and fragile children and youth with special health care needs. Arch Pediatr Adolesc Med 2007;161(10):937–944.
  9. Hendriksz C. Improved diagnostic procedures in attenuated mucopolysaccharidosis. BrJ Hosp Med 2011;72(2):91-95.
  10. Muenzer J et al. International Consensus Panel on the Management and Treatment of Mucopolysaccharidosis I. Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics 2009;123(1):19-29.
  11. Muenzer J, Beck M, Eng CM, et al.Genet Med. 2011;13(2):95–101. doi:10.1097/GIM.0b013e3181fea459.
  12. Kakkis ED et al. The mucopolysaccharidoses. In: Berg BO, ed Principles of child neurology. New York, NY: McGraw-Hill; 1996:1141–1166.