Neurohormonal Therapy in Patients With Transthyretin Cardiac Amyloidosis: Is it an Option?

BACKGROUND: Neurohormonal therapy is strongly recommended for heart failure with reduced left ventricular ejection fraction (LVEF). However, its role in transthyretin cardiac amyloidosis remains uncertain given concerns regarding tolerability and the perception that systolic dysfunction reflects an irreversible situation. CASES SUMMARY: We report 3 patients with wild-type transthyretin cardiac amyloidosis (wtATTR-CA) who initially presented with heart failure and reduced LVEF (≤35%). Guideline-directed medical therapy was initiated and was carefully titrated. During follow-up, these patients experienced clinical improvement and recovery of LVEF to ≥50%, allowing subsequent initiation of tafamidis, which is only approved for LVEF ≥50% in our country. DISCUSSION: These cases suggest that neurohormonal therapy may be feasible and beneficial in selected patients with wtATTR-CA and reduced LVEF, challenging the traditional paradigm of irreversible systolic dysfunction. TAKE-HOME MESSAGES: Guideline-directed medical therapy may be considered on an individualized basis in patients with wtATTR-CA and reduced LVEF. Some patients may tolerate treatment and experience improvement.