Table 2.
General principles for the management of patients with MPS VI
| Statement | Percentage consensus |
|---|---|
| All guidance statements are evidence Grade D (level 5 expert clinical opinion) | |
| Diagnosis of MPS VI during infancy is critical to optimise patient outcomes | 98% |
| The first consultation should be conducted by a physician with experience of treating MPS as soon as possible after diagnosis. This should include a full discussion of disease pathology, progression, treatment options and management. Ongoing information should be provided to optimise patient outcomes | 97% |
| Patients and caregivers should receive ongoing psychosocial support from a social worker and/or psychologist, and should be directed towards a MPS society or relevant patient organisation in their country | 94% |
| A comprehensive medical history and multi-system evaluation should be conducted within days of diagnosis to set a baseline for ongoing assessments and evaluate the physical and neurological manifestations of disease, functional ability and disease burden | 88% |
| Ongoing and regular multi-system monitoring, and assessments are recommended to track the natural history of MPS VI, monitor the impact of treatment and assess the need for treatment interventions to manage the symptoms of MPS VI. These should be conducted at every clinic visit, annually or in some cases as clinically indicated (for example pre- and post-operatively) | 100% |
| Timely interventions are recommended where clinically indicated by monitoring, to help avoid irreversible damage caused by the natural history of MPS VI, and to manage the disease manifestations and maintain long-term quality of life (QoL) | 99% |
| A multidisciplinary team (MDT) of metabolic specialists, surgeons and allied healthcare professionals (including, but not limited to: nurses, physiotherapists, occupational therapists, psychologists and audiologists) is required to manage the diverse range of disease manifestations of MPS VI | 99% |
| Co-ordination of the entire MDT care team is required prior to any procedure to determine the need for surgery, to discuss the benefits and risks of combining surgeries to minimise the need for multiple anaesthetics and to decide the optimal order of procedures. The decision to combine surgeries should take into consideration the surgical and intubation time, and complexity of procedures | 93% |
| The risks and benefits of any intervention and the competing risks of other medical problems should be assessed and discussed with patients, families and caregivers such that they can make an informed decision on the appropriateness of the therapy/surgery | 100% |
| Surgical procedures should be performed by (or under the guidance of) specialist surgeons and anaesthetists with experience of MPS, in medical centres with intensive care units | 99% |
| Management of pain should be a fundamental part of the care of patients with MPS VI, with the aim of improving QoL and maintaining mobility. Refer to general guidelines for pain management | 100% |