Background: Tafamidis is the first disease-modifying therapy approved for transthyretin amyloid cardiomyopathy (ATTR-CM) (NICE, 2024). Randomised trials (ATTR-ACT) showed reductions in mortality and heart-failure hospitalisations (Maurer et al., 2018), but contemporary real-world cohorts show mixed outcomes, and treatment success strongly depends on early, accurate diagnosis and appropriate patient selection (Shah et al., 2024; Müller et al., 2024). The discrepancy in real world outcomes can be linked to disease stage at initiation and diagnostic pathway adherence. In Ireland Tafamidis is available via an HSE Managed Access Protocol requiring clinician application (HSE, 2023), but there is no published national assessment of real-world outcomes of the treatment.
Aims and hypothesis: This study will (1) measure clinical outcomes after Tafamidis initiation: functional testing (6-minute walk distance where recorded or OPD functional status), cardiovascular hospitalisations, strokes, and mortality; and (2) identify predictors of favourable or poor response. We hypothesise that a less favourable response to treatment is likely to be associated with age > 80 years, advanced disease with reduced left ventricular function, restrictive filling patterns, and a left ventricular wall thickness > 20 mm.
Methods: Retrospective cohort study of patients approved for Tafamidis (HSE MAP) between 2020–2025. Baseline diagnostic data, imaging, ATTR subtype, NYHA, biomarkers, 6MWT if available, dates of initiation, and outcomes will be extracted. Based on these factors adherence rates to HSE initiation criteria, Kaplan-Meier survival estimates, Cox regression adjusted for age, NYHA, ATTR subtype and comorbidity, and comparison of outcomes by adherence to diagnostic criteria will be judged.
Expected outputs: Characterisation of Tafamidis prescribing habits in Ireland, survival and hospitalisation outcomes after Tafamidis, and recommendations for optimised prescribing. These results may lead to optimisation of clinical practice for selecting patients who will benefit significantly by treatment with Tafamidis.