Health‐related quality of life among transthyretin amyloid cardiomyopathy patients

Abstract

Aims Transthyretin amyloid cardiomyopathy (ATTR CM) is a progressive and severe heart disease with physical and psychological implications. The Nordic PROACT study was conducted to investigate the health‐related quality of life (HRQoL) in ATTR CM patients. Methods and results The Nordic PROACT study was a cross‐sectional non‐interventional study conducted in 12 cardiology hospital clinics across Norway, Sweden, Finland and Denmark. Men and women aged ≥18 years diagnosed with symptomatic ATTR CM were included. The investigator provided information on medical history, biomarkers, current treatment, co‐morbidities and disease severity according to the New York Heart Association (NYHA) class and the National Amyloidosis Centre (NAC) staging. Patients completed the HRQoL questionnaires in the form of the Kansas City Cardiomyopathy Questionnaire (KCCQ), the EQ‐5D‐5L index with Visual Analog Scale (VAS), and the Major Depression Inventory (MDI). A total of 169 patients (mean ± SD age 77.7 ± 6.2 years) were included. Ninety‐two per cent were men. Seventy‐six per cent had wildtype ATTR CM (ATTRwt CM) and 15% had a hereditary form of ATTR CM (ATTRv CM) while 9% were genetically unclassified. Most patients were in NYHA class II (54%) and NAC stage 1 (53%). Participation in randomized clinical trials (RCT) was noted in 58% of the patients. The 169 ATTR CM patients had a mean ± SD KCCQ score of 64.3 ± 23.1 for total symptom score, 64.8 ± 20.9 for overall summary score (OSS) and 65.1 ± 21.5 for clinical summary score. The EQ‐5D‐5L total utility score was 0.8 ± 0.2 and the EQ‐5D‐5L VAS score was 62.9 ± 20.6. The vast majority (89%) did not report any signs of depression. Patients with ATTRv CM had a higher KCCQ OSS as compared with ATTRwt CM, while EQ‐5D‐5L utility score, EQ‐5D‐5L VAS and MDI were similar. Non‐RCT participants had a poorer HRQoL as compared with RCT participants as reflected in lower KCCQ OSS and EQ‐5D‐5L VAS scores and a higher MDI score. Patients with higher NYHA classes and NAC disease stages had a poorer HRQoL as demonstrated by lower KCCQ and EQ‐5D‐5L scores and higher MDI scores. Correlation between KCCQ, EQ‐5D‐5L and MDI and the covariate NYHA class remained significant (P < 0.05) after adjusting for multiple testing. Conclusions KCCQ scores were lower than previously reported for patients with other heart diseases of non‐ATTR CM origin. The HRQoL measures correlated well to NYHA class and NAC disease stage. The prevalence of depression appeared to be low.

Determinants of health status in older patients with transthyretin cardiac amyloidosis: a prospective cohort study

“…In our cohort, median values for KCCQ were 66 (50–75) points, in line with other recent observational studies [ 22 , 23 ]. Until recently, ATTR-CA was considered a progressive disease characterized by poor and rapidly worsening quality of life [ 22 ]: advanced age, diagnostic delay, systemic symptoms, and multiple comorbidities are all factors which may potentially account for this trend.…”

“…By September 2022, only two patients had received Tafamidis for a considerable amount of time (>12 months). Given the limited number of patients and restricted exposure to the drug to produce tangible effects on KCCQ based on data from the ATTRACT trial, we did not include this in the multivariable model [ 23 ].…”

Health care resource use, diagnostic delay and disease burden in transthyretin amyloid cardiomyopathy in Sweden

“…Diagnostic delays of several years between the first cardiac symptom and ATTR-CM diagnosis have also been demonstrated elsewhere [ 26 ]. Furthermore, this diagnostic delay is signified by a patient’s disease burden in terms of frequent hospital visits and poor quality of life [ 6 , 27 ].…”

Burden of untreated transthyretin amyloid cardiomyopathy on patients and their caregivers by disease severity: results from a multicenter, non-interventional, real-world study

“…Previous studies evaluating the humanistic burden of transthyretin amyloidosis suggest a significant physical, emotional, and social impact on patients and their caregivers ( 9 – 20 ). However, most of these studies focused on patients with variant transthyretin amyloidosis, which can have a predominantly neurological phenotype ( 10 – 12 , 14 , 16 , 19 ).…”

“…However, most of these studies focused on patients with variant transthyretin amyloidosis, which can have a predominantly neurological phenotype ( 10 – 12 , 14 , 16 , 19 ). Only a few studies have specifically evaluated the burden of ATTR-CM ( 9 , 13 , 15 , 17 , 20 ).…”