Patient Reported Outcomes In Amyloidosis Cardiomyopathy

Background Amyloidosis cardiomyopathy is a systemic disease with high risk of disability and death. Patient-reported outcomes (PROs) are validated tools that measure health-related quality of life (HRQoL) and provide prognostic information in patients with heart failure. However, data on HRQoL profile and prognostic value of PROs in patients with cardiac amyloidosis are limited. Methodology This is an observational study of patients with transthyretin amyloidosis (ATTR) and light chain amyloidosis (AL) who completed Kansas City Cardiomyopathy Questionnaire (KCCQ-12) at the time of clinic visit. We examined KCCQ-12 scores and their relationship to HF hospitalization or death in both amyloidosis groups. Results A total of 81 patients were included in the study, 48 with ATTR and 33 with AL with a mean age of 78±7 years and 65±10 years, respectively. KCCQ-12 results were similar in both groups and showed significant HRQoL limitation - median summary score of 38 (IQR 24-60) for ATTR and 51 (IQR 31-73) for AL, p = 0.128. Compared to AL, patients with ATTR had more impairment in the physical limitation domain (Table). Over a median follow-up of 343 days, HF hospitalization or death occurred in 23 (48%) patients with ATTR and 17 (51%) patients with AL. KCCQ score was associated with the risk of HF hospitalization or death with a correlation ratio of 0.9 for ATTR (p=0.025) and 1.0 for AL (p=0.03). ROC AUC for KCCQ-12 ability to predict HF hospitalization or death was 0.7 (95% CI, 0.54-0.84, p = 0.026) for ATTR and 0.8 (95% CI, 0.62-0.95, p = 0.006) for AL. In ATTR, the rate of HF hospitalization or death increased proportionally with decreasing KCCQ-12 scores while in patients with AL high events rates were seen for KCCQ-12 scores 60 or lower. (Figure). Conclusions Patients with ATTR and AL amyloidosis cardiomyopathy suffer from poor HRQoL and high burden of events. Our study shows that KCCQ provides important prognostic information in these patients. Routine KCCQ assessments could be used to identify high-risk patients in need of close monitoring.