A snapshot of the clinical characteristics of patients with transthyretin amyloid cardiomyopathy (ATTR-CM) in Australia and New Zealand was recently published in Heart Lung, and Circulation.
“Cardiac ATTR presents a significant number of patients managed in specialist amyloidosis centres across Australia and NZ, and is likely to continue to rise with increasing awareness.” the authors said.
The authors performed a survey of seven centers specializing in ATTR-CM throughout Australia and New Zealand. The aim was to recollect data about patients treated in those centers during 2022. All of the included patients had a confirmed ATTR-CM diagnosis either through cardiac scintigraphy or cardiac biopsy.
The survey identified more than 500 patients with ATTR-CM, of which approximately 65% had been newly diagnosed, and the rest were undergoing follow-ups. The great majority (more than 300) had wild-type ATTR-CM, while the rest had hereditary ATTR-CM or had declined genetic testing. About 88% of the patients were men.
Researchers calculated the Gillmore score of all patients with available data; the Gillmore score is a prognostic tool that classifies patients with ATTR-CM into three stages according to their expected overall survival, with Stage I having the longest and Stage III the shortest.
More than 67% of patients were Gillmore Stage I; 24% were Gillmore Stage II, and 9% were Gillmore Stage III. The authors observed that patients with wild-type ATTR-CM were more likely to be Gillmore Stage I. Similarly, patients with wild-type ATTR-CM tended to have less severe echocardiographic changes.
The authors found a correlation between younger age and the Gillmore score, with young patients having better scores. There was no correlation between sex and disease severity.
More than 75% of patients were taking targeted ATTR-CM therapy such as diflunisal; only 78 were taking disease-modifying therapy (tafamidis) in the context of clinical trials.
“Our data highlight the key role of dedicated amyloidosis clinics in addition to facilitating diagnosis, by providing access to disease-modifying therapies through clinical trials and compassionate access programs, both at the investigational stage, as well as for approved but yet unfunded therapies (e.g., tafamidis and patirisan),” the authors said.