Chemotherapy for AL Amyloidosis in Heart Failure
What is the effect of bortezomib, dexamethasone, and an alkylating agent (BDex+AA) as a first-line treatment strategy on mortality in patients with symptomatic heart failure from light-chain amyloidosis (AL) cardiac amyloidosis?
Patients newly diagnosed with symptomatic New York Heart Association (NYHA) ≥ class II heart failure due to AL amyloidosis were retrospectively studied. Initial treatment strategy was adjudicated and propensity score analysis was used to adjust for the nonrandomized allocation of treatments. Survival was assessed using a Cox proportional hazards model after adjusting for the propensity score for receiving treatment, age, NYHA class, and ejection fraction.
Among 106 treated patients (ages 64.6 ± 11.3 years, 63% male, 76% lambda subtype), 40 received the three-drug regimen and 66 received other regimens. Mortality was 65% overall, 48% in the BDex+AA cohort (median survival time, 821 days), and 76% in patients who received other regimens (median survival time, 223 days). Initial treatment with BDex+AA was associated with decreased mortality after multivariable adjustment (hazard ratio, 0.209; 95% confidence interval, 0.069-0.636; p = 0.006). This association remained after further adjustment for components of the Mayo Stage.
The authors concluded that use of BDex+AA in the treatment of AL amyloidosis in patients presenting with symptomatic heart failure is associated with improved survival after adjusting for clinical variables.
This study reports that the use of bortezomib, dexamethasone, and an alkylating agent (BDex+AA) in the treatment of AL amyloidosis in patients presenting with symptomatic heart failure has increased over time. This regimen appears to be associated with improved survival after adjusting for clinical variables. There is a significant need for clinical trials in AL amyloidosis with novel agents given the persistently poor prognosis in the setting of cardiac involvement. Although there are several randomized controlled trials enrolling AL patients with cardiac involvement, these studies are excluding patients above a certain level of N-terminal pro–B-type natriuretic peptide. Given the universally poor prognosis of AL with heart failure, future studies of newer agents in AL amyloidosis need to include patients with symptomatic heart failure.
Keywords: Alkylating Agents, Amyloidosis, Cardiomyopathies, Dexamethasone, Heart Failure, Mortality, Natriuretic Peptide, Brain, Peptide Fragments, Stroke Volume, Survival
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