A recent study published in the European Heart Journal – Quality of Care & Clinical Outcomes demonstrated that the recently developed Transthyretin Amyloidosis – Quality of Life Questionnaire (ATTR-QOL) was able to capture the disease burden of patients with transthyretin amyloid cardiomyopathy (ATTR-CM) and other forms of ATTR.
Scores on the ATTR-QOL were positively associated with various patient-reported measures and markers of disease. This study is the first to confirm the survey’s applicability in clinical settings.
While organ-specific and general health questionnaires have been used in both ATTR care and studies, the authors noted these often fail to capture the specific disease burden of ATTR.
The Amyloidosis Research Consortium developed the ATTR-QOL to better capture patient outcomes. It consists of two scores: frequency and severity of symptoms and impact of disease on daily life. Higher scores indicate a more negative impact of the disease.
The study included 137 patients with ATTR-CM, nine with transthyretin amyloid neuropathy and 48 with a mixed phenotype. All participants were patients at Columbia University who completed the ATTR-QOL.
Read more about ATTR-CM signs and symptoms
Participants were 86% male and had a median age of 80 years. Most individuals had wild-type ATTR and early-stage disease.
Patients with a mixed phenotype had worse symptom and impact scores than participants with only cardiac or neurological involvement. In addition, patients with a greater New York Heart Association class, National Amyloidosis Center score and Columbia stage had poorer ATTR-QOL scores.
In terms of functionality, those with greater ATTR-QOL scores tended to have shorter 6-minute walk test distances and poorer Short Physical Performance Battery values.
The researchers also found that ATTR-QOL had good agreement with other established measure of quality of life. Moreover, the questionnaire displayed strong discriminatory ability, meaning it could differentiate between different groups of patients.
“Our results confirm that ATTR-QOL reflects disease severity and effectively distinguishes between different severity levels, supporting its potential clinical applicability,” the study’s authors concluded. “The ability to collect this comprehensive information through a single, unified questionnaire could provide more insight into the patient experience.”
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