Societal costs and burden of hereditary transthyretin amyloidosis polyneuropathy

Inês, Mónica; Coelho, Teresa; Conceição, Isabel; Landeiro, Filipa; Carvalho, Mamede de; Costa, João

doi:10.1080/13506129.2019.1701429

Cited by 5 publications

(10 citation statements)

References 46 publications

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“…Similar to our study, Inês et al found the greatest proportion of patients with amyloidosis in the older age groups [28]. Additionally, the study reported the greatest disease burden for this group [28]. A fiscal cost model found that patients with ATTRv amyloidosis in the Netherlands pay less taxes and receive incrementally more in government intervention, with the conclusion that halting disease progression early would result in economic benefits beyond health benefits [14].…”

Section: Discussionsupporting

confidence: 87%

“…Two recent international articles examined societal and public costs of ATTRv amyloidosis. A 2016 study of patients with ATTRv polyneuropathy in Portugal found a mean cost (including direct and indirect costs) per patient of €28,152 with treatment costs contributing the largest portion (52%) [28]. Similar to our study, Inês et al found the greatest proportion of patients with amyloidosis in the older age groups [28].…”

Section: Discussionsupporting

confidence: 87%

“…A 2016 study of patients with ATTRv polyneuropathy in Portugal found a mean cost (including direct and indirect costs) per patient of €28,152 with treatment costs contributing the largest portion (52%) [28]. Similar to our study, Inês et al found the greatest proportion of patients with amyloidosis in the older age groups [28]. Additionally, the study reported the greatest disease burden for this group [28].…”

Section: Discussionsupporting

confidence: 86%

“…Third, this study examines only direct healthcare costs and does not include indirect costs such as reduced work productivity. Moreover, while we did not examine the effect of demographic factors, such as age, on the burden of illness, we would expect to find greater burden in certain populations similar to what has been found in other amyloidosis studies [14,28]. Additionally, costs prior to diagnosis, which may also contribute to burden, were not captured.…”

Section: Limitationsmentioning

confidence: 78%

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The Clinical and Economic Burden of Newly Diagnosed Hereditary Transthyretin (ATTRv) Amyloidosis: A Retrospective Analysis of Claims Data

et al. 2020

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Introduction: Little is known about the burden of hereditary transthyretin (ATTRv) amyloidosis, a genetic, progressive, and fatal disease caused by extracellular deposition of transthyretin amyloid fibrils. The study's aim was to estimate costs and disease burden associated with ATTRv amyloidosis in a real-world setting. Methods: Using IBM Ò MarketScan Ò Commercial and Medicare Supplemental data, we identified patients at least 18 years of age with newly diagnosed ATTRv amyloidosis. Diagnosis required at least one medical claim with relevant diagnosis code (International Classification of Diseases, 9th Revision, Clinical

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Section: Discussionsupporting

confidence: 87%

Section: Discussionsupporting

confidence: 87%

Section: Discussionsupporting

confidence: 86%

Section: Limitationsmentioning

confidence: 78%

See 2 more Smart Citations

The Clinical and Economic Burden of Newly Diagnosed Hereditary Transthyretin (ATTRv) Amyloidosis: A Retrospective Analysis of Claims Data

et al. 2020

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“…In recent years, a novel disease‐modifying strategy has emerged as a new therapeutic horizon to slow neurological deficits by stabilizing TTR structures or suppressing TTR production [5–8]. To maximize the effectiveness of these therapies, it is critical to identify biomarkers reflecting the onset of early and irreversible progression of ATTRv‐PN to guide the start of treatment [9].…”

Section: Introductionmentioning

confidence: 99%

Early changes of nerve integrity in preclinical carriers of hereditary transthyretin Ala117Ser amyloidosis with polyneuropathy

Chiang

Yeh

Sung

et al. 2021

Euro J of Neurology

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Background and purpose Disease‐modifying therapies provide new horizons for hereditary transthyretin amyloidosis with polyneuropathy (ATTRv‐PN) to slow neuropathic progression. Initiating treatment at the earliest time requires biomarkers reflecting both small‐ and large‐fiber degeneration in carriers. Methods This study included examinations of pathology (intraepidermal nerve fiber [IENF] density), physiology (nerve conduction studies, autonomic function test, and nerve excitability), and psychophysics (thermal thresholds) in carriers to compare to healthy controls and asymptomatic diabetic patients. Results There were 43 carriers (44.2 ± 11.4 years, p.Ala117Ser in 42 carriers), 43 controls (43.4 ± 12.7 years) including 26 noncarrier families, and 50 asymptomatic diabetic patients (58.1 ± 9.5 years). Carriers had lower IENF densities than controls and similar densities as diabetic patients. Median nerve conduction parameters, especially distal motor latency, were the most frequent neurophysiological abnormality in carriers, could differentiate carriers from controls and diabetic patients, were correlated with IENF densities in carriers but not in controls and diabetic patients, and were correlated with nerve excitability parameters in carriers but not in controls. Fifteen carriers (34.9%) with electrophysiological evidence of median nerve entrapment at the wrist had lower IENF densities and more abnormal conduction parameters than carriers without. We defined nerve dysfunction index—the ratio of median distal motor latency to IENF density—which differentiated carriers from controls. Conclusions In late‐onset ATTRv‐PN carriers with predominant p.Ala117Ser, median conduction parameters were the most common neurophysiological abnormalities and served as surrogate signatures of small‐ and large‐fiber impairment. Combination of median distal motor latency and IENF density can reflect early neuropathy in carriers.

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Pulmonary AL amyloidosis: A review and update on treatment options

Moy

Mirza

Moskal

et al. 2022

Annals of Medicine &Amp; Surgery

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Societal costs and burden of hereditary transthyretin amyloidosis polyneuropathy

Cited by 5 publications

References 46 publications

The Clinical and Economic Burden of Newly Diagnosed Hereditary Transthyretin (ATTRv) Amyloidosis: A Retrospective Analysis of Claims Data

The Clinical and Economic Burden of Newly Diagnosed Hereditary Transthyretin (ATTRv) Amyloidosis: A Retrospective Analysis of Claims Data

Early changes of nerve integrity in preclinical carriers of hereditary transthyretin Ala117Ser amyloidosis with polyneuropathy

Pulmonary AL amyloidosis: A review and update on treatment options

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