Key result
Recent ATTR-CM trials enroll milder disease, with 12-month placebo mortality dropping to ~5-7%.
Why the study?
Recent advancements in awareness, diagnosis, and treatment options for ATTR-CM have likely influenced trial populations, prompting an assessment of temporal trends in baseline characteristics of enrolled patients.
How have patient characteristics and outcomes evolved over time in ATTR-CM clinical trials?
Systematic Review
How have patient characteristics and outcomes evolved over time in ATTR-CM clinical trials?
Patients enrolled in recent ATTR-CM trials have less advanced disease and lower placebo-group mortality compared to earlier trials, reflecting earlier identification and evolving therapeutic landscapes.
Answers are grounded in this paper and cite their sources.
Placebo mortality decline warrants caution in cross-trial comparisons; confirms evolving ATTR-CM populations with less advanced disease.
BACKGROUND: Over the past decade, advancements in awareness, diagnosis, and new treatment options for transthyretin amyloid cardiomyopathy (ATTR-CM) have likely influenced the characteristics of patient populations enrolled in clinical trials. This systematic review aims to assess temporal trends in baseline characteristics of patients enrolled in ATTR-CM clinical trials. METHODS: Systematic literature searches were conducted on September 25, 2024, using Embase, MEDLINE, and Cochrane databases via Ovid, and were supplemented by gray literature searches. Overall, 58 publications on 13 clinical trials enrolling patients with ATTR-CM during 2008-2022 were included. Baseline characteristics of participants enrolled in these trials and key clinical outcomes, including all-cause mortality (ACM), were analyzed. RESULTS: Key baseline characteristics - including the proportion of participants with variant ATTR-CM; New York Heart Association class; N-terminal pro-B-type natriuretic peptide and troponin levels; and estimated glomerular filtration rate - varied according to enrollment year. ACM in placebo groups at 12 months decreased from 9.0% in ATTR-ACT (2013-2015) to 6.9% in ATTRibute-CM (2019-2020), 5.6% in APOLLO-B (2019-2021), and 4.3% and 5.5% in HELIOS-B (overall and monotherapy populations, respectively; 2019-2021). CONCLUSIONS: Patients enrolled in more recent clinical trials exhibited baseline characteristics consistent with less advanced ATTR-CM than earlier clinical trials. These descriptive findings are consistent with a temporal shift toward earlier identification and enrollment of patients with ATTR-CM, occurring alongside advances in diagnostic techniques and availability of disease-modifying treatments. These observations highlight the importance of evaluating the relevance of clinical outcomes and trial design in evolving patient populations and therapeutic landscapes.
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Masri et al. (2026) conducted a systematic review in Transthyretin amyloid cardiomyopathy (ATTR-CM). More recent trial enrollment year (2019-2021) vs. Earlier trial enrollment year (2013-2015) was evaluated on Baseline characteristics and all-cause mortality at 12 months. Patients enrolled in recent ATTR-CM trials had less advanced disease, with 12-month placebo group all-cause mortality decreasing from 9.0% in 2013-2015 to 4.3-6.9% in 2019-2021.
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