Table 1 Strategies for management of patients with MPS through critical clinical situations
From: Critical clinical situations in adult patients with Mucopolysaccharidoses (MPS)
| Strategy | Details |
|---|---|
| Involve all relevant specialities in surgical preparations and incorporate into the MDT (Fig. 1) |
Coordinate through a metabolic specialist Incorporate paediatric HCPs if additional expertise is required Ensure the MDT is provided with expert information on patient needs and possible complications |
| Gain advice when developing standard operating procedures |
Collate input across the MDT Incorporate information from guidelines, recommendations, publications and congresses |
| Monitor patient for progression of symptoms and development of adult-specific conditions (Multidisciplinary review and MPS Passport in Supplementary information) |
Monitor complex symptoms throughout the patient’s life and new symptoms as they emerge Screen for common adult diseases, such as diabetes, cancer and hypertension |
| Discuss surgical procedures with patient and family well in advance |
Allow patient and family to ask questions about surgery and choose where surgery is carried out Explain risks and benefits of proceeding with or not proceeding with surgery Present other therapeutic options and likely outcomes |
| Assess surgical risk and always carry out preoperative assessments (The preoperative assessment in Supplementary information) |
Collate information from the MDT Balance risk of poor surgical outcomes with risk of no surgery Include input of patient and family preferences, and likely impact on quality of life Carry out all assessments required for general anaesthesia, even if a local anaesthetic is planned |
| Hold MDT meetings prior to surgery (The preoperative assessment in Supplementary information) |
Ensure appropriate surgical expertise is available • Confirm surgical plans, and review current assessment results |
| Stabilise symptoms prior to surgery | Manage cardiac and respiratory dysfunction that may increase surgical risk |
| Individualise procedures and equipment for each patient (Surgical preparations in Supplementary information) |
Prepare paediatric equipment and replacement devices (e.g. cardiac valves) for patients of small stature Adapt post-surgical management (e.g. post-surgical fluid volumes) for patients of small stature |
| Allow time for inclusion of additional procedures during surgery |
Make surgeons aware of the potential need to manage complications associated with scarring from previous surgeries and/or MPS pathology Be prepared for: • Unsuccessful surgery • Problems with intubation • Haemorrhage • Post-surgical tracheostomy • Pain and/or discomfort • Infection |
| Anticipate need for emergency expert assistance during surgery | Ensure that paediatric anaesthetists, interventional radiologists and ENT specialists are aware of the procedure |
| Prepare a post-surgical care plan (Post-surgical care in Supplementary information) | Ensure that different options are available based on surgical outcomes and emergency procedures |
| Support patient and family after the critical clinical situation |
Follow up patients with specialist members of the MDT Provide guidance on recovery and follow-up Ensure families support patients in following medical recommendations Provide prophylactic management and therapies to reduce negative impacts of ERT cessation |
| Keep up to date with expert opinions |
Attend regional and national meetings Contact other experts for advice |