Targeted Therapies May Reshape Care for Patients With Transthyretin Amyloid Cardiomyopathy

Recent years have brought improvements in diagnostic modalities as well as targeted therapies that could reshape the standard of care for patients with transthyretin amyloid cardiomyopathy, according to study findings published in the Annals of Pharmacotherapy.

“Transthyretin amyloid cardiomyopathy is a complex disease in which amyloidosis causes cardiomyopathy,” wrote lead and corresponding author Jankhna D. Yadav, MD, PharmD, RPh, of Pfizer, and coauthors. “Underdiagnosis is attributed to the clinical presentation being heterogeneous, indistinguishable from heart failure caused by other etiologies, and the need for invasive testing modalities, including endomyocardial biopsy.”

Until 2019, no treatments were approved to treat transthyretin amyloid cardiomyopathy, noted researchers. Instead, treatment focused on the management of heart failure symptoms associated with transthyretin amyloid cardiomyopathy and organ transplant.

However, therapies are now emerging that address the underlying pathophysiology of transthyretin amyloid cardiomyopathy, according to researchers. Treatments recently approved or in development span multiple treatment classes, including transthyretin stabilizers, human monoclonal antibodies, gene silencers, and CRISPR/Cas9 gene editing.

“Diagnostic modalities along with biomarker and genetic testing could detect disease earlier and target therapy more accurately,” concluded study authors. “Novel therapies demonstrate potential treatment benefits and can help shape the standard of care for these patients.”

Reference:
Yadav JD, Othee H, Chan KA, Man DC, Belliveau PP, Towle J. Transthyretin amyloid cardiomyopathy-current and future therapies. Ann Pharmacother. 2021;55(12):1502-1514. doi:10.1177/10600280211000351