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Table 1 Strategies for management of patients with MPS through critical clinical situations

From: Critical clinical situations in adult patients with Mucopolysaccharidoses (MPS)

StrategyDetails
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 proceduresCollate 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 advanceAllow 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 surgeryManage 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 surgeryMake 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 surgeryEnsure 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 situationFollow 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 opinionsAttend regional and national meetings
Contact other experts for advice
  1. ENT Ear, Nose and Throat, ERT Enzyme replacement therapy, HCP Healthcare professional, MDT multidisciplinary team, MPS mucopolysaccharidosis