In patients with hereditary transthyretin amyloidosis (hATTR) with cardiomyopathy (CM), health-related quality of life (HRQoL) is lower than that in patients with other cardiac disorders, according to findings from the Nordic PROACT study published in ESC Heart Failure.
Individuals with hATTR-CM experience a progressive accumulation of amyloid fibers of misfolded transthyretin protein in their heart, which is associated with developing restrictive cardiomyopathy, along with arrhythmia and clinical heart failure. Noncardiac manifestations of disease include spinal stenosis and carpal tunnel syndrome.
The severity of a patient’s disease can be assessed based on New York Heart Association (NYHA) classes, as well as National Amyloidosis Centre (NAC) disease stages—both of which offer prognostic data. It is well known that the physical symptoms of hATTR-CM can have a significant effect on a persons’ HRQoL and activities of daily living, with disease progression debilitating and potentially life threatening.
Nordic PROACT (patient-reported outcomes in transthyretin amyloid cardiomyopathy trial) was designed to evaluate the HRQoL of contemporary patients from 12 hospital cardiology outpatient clinics in Denmark, Finland, Norway, and Sweden who were living with symptomatic hATTR-CM. Further, associations between HRQoL, based on KCCQ, EuroQoL (EQ-5D-5L), and Major Depression Inventory, and disease severity, comorbidities, and diagnostic delays, were examined as well.
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The study comprised 2 electronic questionnaires: (1) the eCaseReportForm, which was designed for the investigator, and (2) the ePatientReportedOutcomes, which was intended for the patient. Study participants included those aged 18 years or older who had received a diagnosis of hATTR-CM 3 months or more prior to enrollment.
Nordic PROACT enrolled 169 participants—70 from Denmark, 61 from Sweden, and 19 each from Finland and Sweden. Overall, 92% of the participants were men and 8% were women. The mean patient age was 78 years. Regarding disease severity, 54% of the participants were categorized as being in NYHA class II, 35% were in NYHA class III, and 53% were in NAC stage 1. Patients’ mean time since disease diagnosis was 3.1 years.
Mean KCCQ total symptom score was 64.3±23.1, overall summary score was 64.8±20.9, and clinical summary score was 65.1±21.5. EQ-5D-5L total utility score was 0.8±0.2, whereas EQ-5D-5L visual analog score score was 62.9±20.6.
Participants who reported higher NYHA classes and NAC stages of disease experienced a poorer HRQoL, as evidenced by lower KCCQ and EQ-5D-5L scores, as well as higher Major Depression Inventory scores.
Following adjustments for multiple testing, associations between KCCQ, EQ-5D-5L, and Major Depression Inventory and the covariate NYHA class remained significant (P <.05). Strongly significant correlations between KCCQ overall summary score and KCCQ (physical limitation), KCCQ (social limitation), and KCCQ (QoL) were reported as well (P <.001 for all).
“The prevalence of depression [among the patients evaluated] was low,” the researchers noted. “Clear correlations were found between measures of HRQoL and disease severity, as assessed by NYHA and NAC, underlining the importance of early diagnosis and treatment,” they concluded.
Eldhagen P, Lehtonen J, Gude E, et al; Nordic PROACT study group. Health-related quality of life among transthyretin amyloid cardiomyopathy patients. ESC Heart Fail. Published online March 22, 2023. doi:10.1002/ehf2.14350