There is currently no national consensus on the prescribing, monitoring or therapeutic drug monitoring (TDM) of sirolimus for patients with inborn errors of immunity (IEIs). Practice varies widely between centres, with different dosing strategies, monitoring frequencies, target trough levels and supporting evidence bases. Because IEI is a rare and heterogeneous group of conditions, individual clinicians and centres often rely on local practice, extrapolated evidence, or experience‑based decision‑making.
This survey aims to understand the current landscape of clinical practice across the UK and will help identify:
- Areas of variation in dosing and monitoring
- Common clinical indications
- Current target trough levels
- Existing local, national or international guidance/practices being used for all indications (for references)
- Gaps, challenges and unmet needs
- Additional therapies where clinicians feel national guidance is required
This information will ensure that the forthcoming guideline is grounded in real‑world practice, reflects the needs of the clinical community, and addresses areas of inconsistency or uncertainty.
The data collected will be analysed to:
- Map current national practice across immunology centres
- Identify areas of agreement that may inform suggested standards of care
- Highlight significant variation where evidence review and consensus‑building are needed
- Inform the structure, scope and recommendations of the national sirolimus guideline
Individual Trust‑level responses will not be identifiable in any published outputs. Findings will be summarised to support the guideline recommendations and inform the guideline indirectly.
