Key result
Targeted amyloidosis therapies improve heart failure symptoms and peripheral neuropathy in small case series.
Why the study?
ATTR-CA is an under-reported cause of congestive heart failure and cardiac arrhythmias requiring heightened clinical suspicion and multimodal diagnostic approaches, with recent advances in treatment.
Case Report (n=5)
No
Highlights the diagnostic challenges of cardiac amyloidosis and illustrates the potential for symptomatic improvement using novel targeted therapies like tafamidis and inotersen in clinical practice.
Suggests possible symptom relief with targeted therapies in cardiac amyloidosis; hypothesis-generating and should not yet change practice.
ATTR-CA is an under-reported cause of congestive heart failure (CHF) and cardiac arrhythmias. Heightened clinical suspicion along with a multimodal investigative approach is often required in diagnosing this potentially fatal condition. Tafamidis and inotersen have shown promising results in terms of progression-free survival by ameliorating CHF symptoms and peripheral neuropathies in clinical trials. In this case series of five patients, we present three wild-type cardiac amyloidosis (ATTRwt-CA), one familial cardiac amyloidosis (ATTRm-CA) and one primary cardiac (AL-CA). The diagnostic modality was different for each patient. ATTRwt-CA, ATTRm-CA and AL-CA patients received tafamidis, inotersen and chemotherapy with bone marrow stem-cell transplantation, respectively.
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Rahman et al. (2021) conducted a case report in Cardiac amyloidosis (ATTR-CA and AL-CA) (n=5). Amyloidosis targeted therapies (tafamidis, inotersen, chemotherapy with stem cell transplant) was evaluated on Symptom improvement and follow-up clinical status.
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