Amyloid light chain (AL) cardiomyopathy (AL-CM) is a highly aggressive disease with high early mortality without prompt and effective treatment; however, its low prevalence and rapid clinical progression pose significant challenges to randomized controlled trials. As a result, plasma cell–directed management for AL amyloidosis is typically extrapolated from studies of multiple myeloma, and guidance has historically relied on case reports, case series, retrospective cohorts, and expert consensus statements.
Heart transplantation has emerged as a viable salvage therapy for carefully selected patients with advanced AL-CM without significant extracardiac involvement and adequate hematologic control. Prior expert consensus guidelines emphasize the importance of achieving a hematologic response before transplantation to optimize short- and long-term post–heart transplant outcomes.3,4 However, despite significant advances in AL therapy over the last decade,5 some patients remain refractory to all plasma cell–directed therapies.