Table 1.
Proposed composition of the multidisciplinary team at centres treating PKU patients with pegvaliase.
| Specialty | Responsibilities |
|---|---|
| Consensus group recommendations about the composition of the multidisciplinary teams at centres treating PKU patients with pegvaliase | |
| Physician |
- Diagnosis and decisions on appropriate therapeutic approach, considering potential adverse events |
| - Pegvaliase prescription | |
| - Patient assessment prior to receiving pegvaliase | |
| - Titration of pegvaliase dose and assessment of patient progress during follow-up clinic visits | |
| - Management of adverse reactions | |
| Dietitian/nutritionist |
- Key role in monitoring and assuring adequate nutritional status of patients |
| - Detailed food pattern analysis pre- and post-pegvaliase | |
| - Nutrition re-education as a strategy to reduce the risk of lifestyle-related comorbidities | |
| - Assessment of nutritional intake | |
| - Interpretation of blood Phe results in conjunction with dietary evaluation | |
| - Assessment of patient progress during follow-up clinic visits | |
| - Contribute to decisions on appropriate therapeutic approach, based on individual (dietetic) patient status/needs | |
| - Point of contact for the patient in terms of blood Phe reporting and diet education | |
| Nurse |
- Education and practical support of patients on pegvaliase injections |
| - Point of contact for the patient in terms of managing drug titration and side effects | |
| - In some countries, nurse practitioners can also prescribe medication and assess patient's progress during follow-up clinic visits | |
| - Underline the global advice given by the physician and dietitian regarding treatment adherence, administration and dietary management | |
| If resources are available,the following specialties should be included in the multidisciplinary team: | |
| Psychologist/counsellor (highly recommended) |
- Surveillance and follow-up on any neurocognitive/psychological deficits |
| - Perform neurocognitive testing and quality of life assessments | |
| - Patient counselling and support | |
| - Monitor patient's fears and help manage their expectations | |
| Social worker |
- Assessing individual patients' socioeconomical needs and support system, and providing assistance in finding solutions in these areas |
| - Advising and supporting patients in contacting the necessary service providers/agencies | |
| Home support worker |
- Home support with blood sampling, treatment, social challenges and pregnancy management |
| - Can be any profession depending on availability, although a nurse would be able to provide education and support regarding injections and potential adverse events | |
| Coordinator | - Administrative assistant as central point of contact for e.g., organising appointments |
| - Especially valuable at treatment initiation to coordinate appointments with the multidisciplinary team | |
Phe: phenylalanine, PKU: phenylketonuria.