Patient–reported Psychophysical Well–being in Transthyretin Amyloid Cardiomyopathy: A Comparison Between Five–year Follow–up Cohorts of Tafamidis–treated and Untreated Patients

Background Transthyretin Amyloid Cardiomyopathy (ATTR–CM) is a rare disease associated with a substantial impairment in patients’ psychophysical well–being and quality of life. Purpose to describe the difference in anxiety, depression, symptom percepition, and fatigue between five years follow–up patients with ATTR–CM treated with tafamidis and those not receiving ATTR–CM specific therapy. Methods We analyzed the clinical and psychological characteristics of two five years follow–up cohorts of patients with ATTR–CM, constituted by 36 patients treated with tafamidis and 5 patients not receiving tafamidis, followed at the Center for Amyloidosis of Florence and the University Hospital of Graz. Patients were evaluated using a sociodemographic questionnaire, the Hospital Anxiety and Depression Scale (HADS) to assess anxiety and depression, the Kansas City Cardiomyopathy Questionnaire (KCCQ) to assess heart failure–related symptom perception in daily life; and the PROMIS Fatigue and Dyspnea questionnaires. Results Patients treated with tafamidis were younger (78.5 [75.75–84] vs 88.5 [85.75–89.25]) and had a better NYHA functional class (p=0.01), while left ventricular ejection fraction was similar compared with patients not receiving tafamidis. The KCCQ showed a more favorable symptoms profile in patients treated with tafamidis for the total score (50.9 [22.0–71.3] vs 75.5 [57.9–82.7], p=0.04) and the clinical symptoms score (45.9 [29.8–76.3] vs 83.1 [65.4–92.7], p=0.006). Similarly, patients treated with tafamidis reported low fatigue burden assessed with PROMIS Fatigue score (55.75 [51.85–67.95] vs 48.4 [43.9–54.38], p=0.009). No significant differences were observed in HADs Anxiety or depression score, as for PROMIS Dyspnea score. Patients treated with tafamidis showed a lower impact of cardiac amyloidosis in daily life symptoms respect of the cohort not treated with tafamidis. Conclusion In our follow–up cohort, patients with ATTR–CM treated with tafamidis reported higher levels of psychophysical well–being in daily life compared with those not receiving tafamidis. Nevertheless, longitudinal analyses including baseline assessments in the same cohort will be essential to better evaluate the impact of tafamidis treatment on patient–reported psychophysical well–being over time.