Evidence map›Paper›PMID 40580033›Full record

ArticleJournal of the peripheral nervous system : JPNS2025

Skin Biopsy as a Diagnostic Tool for ATTRv Amyloid Neuropathy in the UK.

Luke F O'Donnell, Victor Zhang, Roy Carganillo, Alexander M Rossor, Matilde Laura, Mariola Skorupinska, Janet A Gilbertson, Dorota Rowczenio, Yousuf Razvi, Julian D Gillmore and 1 more

Abstract read
In one paragraph

Article in Journal of the peripheral nervous system : JPNS, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Article
  2. Review
  3. Skin Biopsy as a Diagnostic Tool for ATTRv Amyloid Neuropathy in the UK.Journal of the peripheral nervous system : JPNS · 2025
    Article
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

11 authors.

Luke F O'DonnellCentre for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.ORCID https://orcid.org/0000-0001-9514-1516
Victor ZhangCentre for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.
Roy CarganilloCentre for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.
Alexander M RossorCentre for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.ORCID https://orcid.org/0000-0003-4648-2896
Matilde LauraCentre for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.ORCID https://orcid.org/0000-0001-8264-8676
Mariola SkorupinskaCentre for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.
Janet A GilbertsonNational Amyloidosis Centre, University College London, London, UK.
Dorota RowczenioNational Amyloidosis Centre, University College London, London, UK.
Yousuf RazviNational Amyloidosis Centre, University College London, London, UK.
Julian D GillmoreNational Amyloidosis Centre, University College London, London, UK.
Mary M ReillyCentre for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveGene silencing therapy for ATTRv has revolutionised treatment. In minimally symptomatic, early neuropathic disease, skin biopsy can aid in the diagnosis of ATTRv-PN, assessing both amyloid deposition and IENFD. Our aim was to study the value of performing skin biopsies in the diagnosis of ATTRv-PN in UK patients and to assess the influence of this on accessing gene silencing treatment.

methodsSeventy-three patients had skin biopsies performed between July 2021 and October 2023. These were stained for amyloid, typed by immunohistochemistry, and analysed for IENFD.

resultsThe Thr60Ala (30%), Val122Ile (23%) and Val30Met (22%) variants represented the largest number of cases. Normal/equivocal neurophysiology was demonstrated in 78% of cases. 40% of patients had abnormal IENFD, 33% had positive amyloid and 16% had both. This allowed 33% of patients to start gene silencing therapy, 75% of whom had a preceding amyloid cardiomyopathy diagnosed.

conclusionsSkin biopsy is a useful, minimally invasive method for diagnosing ATTRv-PN. It allowed a substantial number of patients to commence gene silencing treatment. As Thr60Ala and Val122Ile are the commonest TTR variants in the UK and patients often present with cardiomyopathy, early diagnosis of ATTRv-PN is critical for treatment decisions.

Indexed as

Amyloid Neuropathies, FamilialSkinAdultAgedAged, 80 and overBiopsyFemaleHumansMaleMiddle AgedPrealbuminUnited KingdomPrealbuminTTR protein, humanamyloidskin biopsyTTR

Identifiers

PMID40580033
PMCPMC12205485

What Socratic holds

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Registered trials

None linked

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 Socratic graph.