Evidence map›Paper›PMID 33982288›Full record

ReviewEuropean journal of clinical investigation2021

Quality of life assessment in amyloid transthyretin (ATTR) amyloidosis.

Alberto Aimo, Claudio Rapezzi, Federico Perfetto, Francesco Cappelli, Giovanni Palladini, Laura Obici, Giampaolo Merlini, Gianluca Di Bella, Matteo Serenelli, Mattia Zampieri and 11 more

Registry-linked trialOpen access · hybridAbstract readReview
In one paragraph

Review in European journal of clinical investigation, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07080268 (Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis in Argentina.), which is not on this map. Cited by 19 papers.

0numbers the graph read from it
0cells of the map it votes in
19citing papers in PubMed
1.8field-weighted citation impact, top 15% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

NCT07080268 active not recruitingnot on this mapstarted 2025, after this paper: background citation

Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis in Argentina.

TypeobservationalSponsorHospital General de Niños Pedro de ElizaldeRan2025 to 2025Enrolled70ConditionsAmyloidosis in Transthyretin (TTR), Hereditary Amyloidosis, Transthyretin-Related
3 · Its place in the literature

Who cites it

19 citing papers in PubMed, 29 citations in OpenAlex.

  1. Article
  2. Article
  3. Review
  4. Review
  5. Review
  6. Article
  7. Review
  8. Review
  9. Review
  10. Quantitative muscle ultrasound as a disease biomarker in hereditary transthyretin amyloidosis with polyneuropathy.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024
    Article
  11. Review
  12. Review
  13. Article
  14. Article
  15. Article
  16. Accessibility to Occupational Therapy Services for Hereditary Transthyretin Amyloidosis.International journal of environmental research and public health · 2022
    Article
  17. Article
  18. Response to the comment by Dr Yarlas.European journal of clinical investigation · 2021
    Article
  19. Quality of life assessment in amyloid transthyretin (ATTR) amyloidosis.European journal of clinical investigation · 2021
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

21 authors at 5 institutions in 1 country.

Alberto AimoInstitute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.ORCID https://orcid.org/0000-0001-9129-9519
Claudio RapezziCardiology Division, University Hospital of Ferrara, Ferrara, Italy.
Federico PerfettoRegional Amyloid Centre, Azienda Ospedaliero Universitaria Careggi, Florence, Italy.
Francesco CappelliRegional Amyloid Centre, Azienda Ospedaliero Universitaria Careggi, Florence, Italy.
Giovanni PalladiniAmyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Laura ObiciAmyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Giampaolo MerliniAmyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Gianluca Di BellaCardiology Division, University of Messina, Messina, Italy.
Matteo SerenelliCardiology Division, University Hospital of Ferrara, Ferrara, Italy.
Mattia ZampieriRegional Amyloid Centre, Azienda Ospedaliero Universitaria Careggi, Florence, Italy.
Paolo MilaniAmyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Roberto LicordariCardiology Division, University of Messina, Messina, Italy.
Lucio TeresiInstitute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Nicolò RibarichInstitute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Vincenzo CastiglioneInstitute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Filippo QuattroneInstitute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Sabina De RosisManagement and Healthcare Laboratory, Institute of Management, Scuola Superiore Sant'Anna, Pisa, Italy.
Giuseppe VergaroInstitute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Giorgia PanichellaInstitute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Michele EmdinInstitute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Claudio PassinoInstitute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Scuola Superiore Sant'Anna · ITUniversity of Pavia · ITAzienda Ospedaliero-Universitaria Careggi · ITUniversity of Ferrara · ITUniversity of Messina · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundAmyloid transthyretin (ATTR) amyloidosis is caused by the systemic deposition of transthyretin molecules, either normal (wild-type ATTR, ATTRwt) or mutated (variant ATTR, ATTRv). ATTR amyloidosis is a disease with a severe impact on patients' quality of life (QoL). Nonetheless, limited attention has been paid to QoL so far, and no specific tools for QoL assessment in ATTR amyloidosis currently exist. QoL can be evaluated through patient-reported outcome measures (PROMs), which are completed by patients, or through scales, which are compiled by clinicians. The scales investigate QoL either directly or indirectly, i.e., by assessing the degree of functional impairment and limitations imposed by the disease.

designSearch for the measures of QoL evaluated in phase 2 and phase 3 clinical trials on ATTR amyloidosis.

resultsClinical trials on ATTR amyloidosis have used measures of general health status, such as the Short Form 36 Health Survey (SF-36), or tools developed in other disease settings such as the Kansas City Cardiomyopathy Questionnaire (KCCQ) or adaptations of other scales such as the modified Neuropathy Impairment Score +7 (mNIS+7).

conclusionsScales or PROMs for ATTR amyloidosis would be useful to better characterize newly diagnosed patients and to assess disease progression and response to treatment. The ongoing ITALY (Impact of Transthyretin Amyloidosis on Life qualitY) study aims to develop and validate 2 PROMs encompassing the whole phenotypic spectrum of ATTRwt and ATTRv amyloidosis, that might be helpful for patient management and may serve as surrogate endpoints for clinical trials.

Indexed as

Quality of LifeAmyloid NeuropathiesAmyloid Neuropathies, FamilialCardiomyopathiesHumansPatient Reported Outcome MeasuresATTR amyloidosisPROMsquality of lifescalestransthyretin

Identifiers

PMID33982288
PMCPMC8596396
OpenAlexW3161053378

What OpenQuestion holds

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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.