ArticleMuscle & nerve2019
Burden of hereditary transthyretin amyloidosis on quality of life.
Article in Muscle & nerve, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
31 citing papers in PubMed, 1 synthesis or guideline pooled it, 63 citations in OpenAlex.
- Rate of neuropathic progression in hereditary transthyretin amyloidosis with polyneuropathy and other peripheral neuropathies: a systematic review and meta-analysis.BMC neurology · 2021Pooled it
- The impact of inotersen on Neuropathy Impairment Score in patients with hereditary transthyretin amyloidosis with polyneuropathy.BMC neurology · 2023Trial
- Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis.Journal of neurology · 2020Trial
- Disease-Modifying Therapies for Hereditary Transthyretin Amyloidosis with Polyneuropathy: Current Status and Future Perspectives.Neurology and therapy · 2026Review
- The patient's voice: real-world experiences of a transthyretin amyloidosis focus group.BMC health services research · 2026Article
- Functional Capacity Evaluation and Rehabilitation Strategies in Cardiac Amyloidosis: A Comprehensive Review.Journal of clinical medicine · 2025Review
- Realizing the therapeutic potential of rapid knockdown of transthyretin via RNA interference in transthyretin amyloidosis.Molecular therapy. Nucleic acids · 2025Review
- Reliability and validity of the Transthyretin Amyloidosis - Quality of Life (ATTR-QOL) Questionnaire impact scales.Journal of patient-reported outcomes · 2025Article
- Predictors of disease burden in patients with untreated transthyretin amyloid cardiomyopathy and their caregivers: aFrontiers in cardiovascular medicine · 2025Article
- Effectiveness of patisiran after switching from tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy.European journal of neurology · 2024Article
- Long-term treatment of hereditary transthyretin amyloidosis with patisiran: multicentre, real-world experience in Italy.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024Observational
- Impact of Vutrisiran on Quality of Life and Physical Function in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy.Neurology and therapy · 2023Article
- Review
- Article
- Burden of untreated transthyretin amyloid cardiomyopathy on patients and their caregivers by disease severity: results from a multicenter, non-interventional, real-world study.Frontiers in cardiovascular medicine · 2023Article
- Amylo-AFFECT-QOL, a self-reported questionnaire to assess health-related quality of life and to determine the prognosis in cardiac amyloidosis.Frontiers in cardiovascular medicine · 2023Article
- Neuropathic pain experience in symptomatic and presymptomatic subjects carrying a transthyretin gene mutation.Frontiers in neurology · 2023Article
- Real-life experience with inotersen in hereditary transthyretin amyloidosis with late-onset phenotype: Data from an early-access program in Italy.European journal of neurology · 2022Observational
- Screening for ATTR amyloidosis in the clinic: overlapping disorders, misdiagnosis, and multiorgan awareness.Heart failure reviews · 2022Review
- Responder analysis for neuropathic impairment and quality-of-life assessment in patients with hereditary transthyretin amyloidosis with polyneuropathy in the NEURO-TTR study.Journal of neurology · 2022Article
Corrections and comments
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Authors and funding
11 authors at 7 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionHereditary transthyretin (hATTR) amyloidosis is a progressive, degenerative disease, with peripheral neuropathy, cardiomyopathy, and other clinical manifestations. In this study we examine the impact of hATTR amyloidosis on quality of life (QOL).
methodsNeuropathy-specific QOL, measured with the Norfolk QOL-Diabetic Neuropathy questionnaire, was compared between patients with hATTR amyloidosis and patients with type 2 diabetes, whereas generic QOL, measured with the 36-item Short Form Health Survey version 2 (SF-36v2), was compared between patients with hATTR amyloidosis, the general population, and patients with chronic diseases.
resultsNeuropathy-specific QOL for patients with hATTR amyloidosis was nearly equivalent to that of patients with type 2 diabetes with diabetic neuropathy accompanied by a history of ulceration, gangrene, or amputation. Generic QOL was worse than that seen in the general population, with physical functioning worse than that for patients with multiple sclerosis and congestive heart failure. DISCUSSION: Patients with hATTR amyloidosis show significant burden on QOL, particularly in physical functioning. Muscle Nerve 60: 169-175, 2019.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.