Estimating the health benefits of timely diagnosis and treatment of transthyretin amyloid cardiomyopathy

Rozenbaum, Mark H; Large, Samuel; Bhambri, Rahul; Stewart, Michelle; Young, Robert; Doornewaard, Alexander van; Dasgupta, Noel R.; Masri, Ahmad; Nativi-Nicolau, José

doi:10.2217/cer-2021-0071

Cited by 10 publications

(13 citation statements)

References 32 publications

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“…7,8 Timely diagnosis and treatment is expected to significantly improve life expectancy for patients with ATTR-CM up to 8 years depending on type of ATTR-CM. 9 Therefore, early diagnosis of ATTR-CM is essential to ensure that patients can benefit from disease-modifying treatment in order to maximize quality of life and reduce the need for healthcare interactions. 9,10 Although the epidemiology of ATTR-CM is beginning to emerge in published research using register data, 8,[11][12][13] treatment patterns for ATTR-CM patients and their healthcare resource use are not well documented.…”

Section: Introductionmentioning

confidence: 99%

“…9 Therefore, early diagnosis of ATTR-CM is essential to ensure that patients can benefit from disease-modifying treatment in order to maximize quality of life and reduce the need for healthcare interactions. 9,10 Although the epidemiology of ATTR-CM is beginning to emerge in published research using register data, 8,[11][12][13] treatment patterns for ATTR-CM patients and their healthcare resource use are not well documented. There is a large body of work on the humanistic and economic burden of heart failure from around the world, [14][15][16] but only few studies on the healthcare resource use of ATTR-CM.…”

Section: Introductionmentioning

confidence: 99%

“…Diagnostic delay and misdiagnosis is common 6 and contributes to low median survival, which has been estimated to be as short as 2.5–3.6 years depending on disease stage at diagnosis and to be half of that in other HF patients 7,8 . Timely diagnosis and treatment is expected to significantly improve life expectancy for patients with ATTR‐CM up to 8 years depending on type of ATTR‐CM 9 . Therefore, early diagnosis of ATTR‐CM is essential to ensure that patients can benefit from disease‐modifying treatment in order to maximize quality of life and reduce the need for healthcare interactions 9,10 …”

Section: Introductionmentioning

confidence: 99%

“…Timely diagnosis and treatment is expected to significantly improve life expectancy for patients with ATTR‐CM up to 8 years depending on type of ATTR‐CM 9 . Therefore, early diagnosis of ATTR‐CM is essential to ensure that patients can benefit from disease‐modifying treatment in order to maximize quality of life and reduce the need for healthcare interactions 9,10 …”

Section: Introductionmentioning

confidence: 99%

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Healthcare resource use of patients with transthyretin amyloid cardiomyopathy

et al. 2022

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Aims Transthyretin amyloid cardiomyopathy (ATTR-CM) is the cardiac manifestation of transthyretin amyloidosis (ATTR).The aim of this study was to estimate healthcare resource use for ATTR-CM patients compared with heart failure (HF) patients, in Denmark, Finland, Norway, and Sweden. Methods and results Data from nationwide healthcare registers in the four countries were used. ATTR-CM patients were defined as individuals diagnosed with amyloidosis and cardiomyopathy or HF between 2008 and 2018. Patients in the ATTR-CM cohort were matched to patients with HF but without ATTR-CM diagnosis. Resource use included number of visits to specialty outpatient and inpatient hospital care. A total of 1831 ATTR-CM and 1831 HF patients were included in the analysis. The mean number of hospital-based healthcare contacts increased in both the ATTR-CM and HF cohort during 3 years pre-diagnosis and was consistently higher for the ATTR-CM cohort compared with the HF cohort, with 6.1 [

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Healthcare resource use of patients with transthyretin amyloid cardiomyopathy

et al. 2022

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“…Survival was significantly better among patients with AL amyloidosis diagnosed in < 6 months from symptom onset with >52% of patients surviving over the 5-year period of the study than for those whose diagnosis took longer who also had significantly increased risk of death as shown by >63% of patients dying during the study period ( 9 ). Undiagnosed and delayed diagnosis of CA results in high morbidity and high mortality whereas early diagnosis has both clinical and quality of life (QoL) benefits for patients with AL, ATTRwt, or ATTRv CA ( 10 – 12 ). Using a disease simulation model, early diagnosis and timely treatment have been shown to extend the calculated life expectancy from the onset of symptoms by more than 5 and more than 7 years among patients with ATTRwt and ATTRv amyloidosis, respectively ( 12 ).…”

Section: Introductionmentioning

confidence: 99%

Changing paradigm in the treatment of amyloidosis: From disease-modifying drugs to anti-fibril therapy

Quarta

Fontana

Damy

et al. 2022

Front. Cardiovasc. Med.

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Cardiac amyloidosis is a rare, debilitating, and usually fatal disease increasingly recognized in clinical practice despite patients presenting with non-specific symptoms of cardiomyopathy. The current standard of care (SoC) focuses on preventing further amyloid formation and deposition, either with anti-plasma cell dyscrasia (anti-PCD) therapies in light-chain (AL) amyloidosis or stabilizers of transthyretin (TTR) in transthyretin amyloidosis (ATTR). The SoC is supplemented by therapies to treat the complications arising from organ dysfunction; for example, heart failure, arrhythmia, and proteinuria. Advancements in treatments have improved patient survival, especially for those whose disease is detected and for whom treatment is initiated at an early stage. However, there still are many unmet medical needs, particularly for patients with severe disease for whom morbidity and mortality remain high. There currently are no approved treatments to reverse amyloid infiltration and deplete the amyloid fibrils already deposited in organs, which can continue to cause progressive dysfunction. Anti-fibril therapies aimed at removing the deposited fibrils are being investigated for safety and efficacy in improving outcomes for patients with severe disease. However, there is no clinical evidence yet that removing deposited amyloid fibrils will improve organ function, thereby improving quality of life or extending life. Nevertheless, anti-fibril therapies are actively being investigated in clinical trials to evaluate their ability to complement and synergize with current SoC.

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Improved long‐term survival with tafamidis treatment in patients with transthyretin amyloid cardiomyopathy and severe heart failure symptoms

Elliott

Gundapaneni

Sultan

et al. 2023

European J of Heart Fail

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AimThe value of disease‐modifying therapies (such as tafamidis) in patients with transthyretin amyloid cardiomyopathy (ATTR‐CM) and severe heart failure symptoms has been debated. This study assessed long‐term all‐cause survival in patients with New York Heart Association (NYHA) class III symptoms in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR‐ACT) long‐term extension (LTE) study.Methods and resultsAt the baseline of ATTR‐ACT, 55/176 (31.3%) patients receiving tafamidis 80 mg and 63/177 (35.6%) receiving placebo had NYHA class III symptoms. After 30 months of treatment, patients could join an ongoing LTE study to receive open‐label tafamidis. In an interim analysis of the LTE study (August 2021), all‐cause mortality was lower among patients with NYHA class III symptoms who received continuous tafamidis in ATTR‐ACT and the LTE study (hazard ratio 0.64; 95% confidence interval 0.41–0.99; median follow‐up: 60 months), as compared with those who received placebo in ATTR‐ACT and tafamidis in the LTE study (median follow‐up: 56 months). Similar findings were observed in patients with NYHA class I/II symptoms at baseline (0.50; 0.35–0.73; tafamidis 80 mg n = 121; placebo n = 114; median follow‐up of 61 and 60 months, respectively).ConclusionWe observed reduced all‐cause mortality with continuous tafamidis treatment compared with delayed tafamidis treatment (placebo then tafamidis) in patients with NYHA class III symptoms at baseline over a median follow‐up of ∼5 years. These findings demonstrate the value of tafamidis treatment in patients with ATTR‐CM and severe heart failure symptoms, and emphasize the importance of early treatment.Clinical Trial Registrations: ClinicalTrials.gov NCT01994889 and NCT02791230.

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Estimating the health benefits of timely diagnosis and treatment of transthyretin amyloid cardiomyopathy

Cited by 10 publications

References 32 publications

Healthcare resource use of patients with transthyretin amyloid cardiomyopathy

Healthcare resource use of patients with transthyretin amyloid cardiomyopathy

Changing paradigm in the treatment of amyloidosis: From disease-modifying drugs to anti-fibril therapy

Improved long‐term survival with tafamidis treatment in patients with transthyretin amyloid cardiomyopathy and severe heart failure symptoms

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