Evidence map›Paper›PMID 42266139›Full record

SynthesisEuropean journal of neurology2026

Pharmacological Interventions for Hereditary Transthyretin-Related Amyloidosis With Polyneuropathy: Systematic Review and Network Meta-Analysis.

Gonçalo S Duarte, Tiago Luís Gonçalves Neves Machado, Filipe B Rodrigues, Isabel Conceção, Davide Pareyson, João Nuno Marques Parracho Guerra da Costa, Task Force of the EAN/PNS Joint Guideline for the Management of Neurological Manifestations in Hereditary Transthyretin Amyloidosis

Erratum issuedAbstract readNetwork Meta-AnalysisSystematic Review
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in European journal of neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. Article
  5. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors.

Gonçalo S DuarteUnidade de Farmacologia Cínica, Unidade Local de Saúde de Santa Maria, Lisbon, Portugal.ORCID https://orcid.org/0000-0001-7802-1897
Tiago Luís Gonçalves Neves MachadoLaboratório de Farmacologia Clínica e Terapêutica, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.ORCID https://orcid.org/0000-0001-8930-4382
Filipe B RodriguesLaboratório de Farmacologia Clínica e Terapêutica, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.ORCID https://orcid.org/0000-0002-5567-7938
Isabel ConceçãoDepartamento de Neurociências e Saúde Mental, ULS de Santa Maria, CAML, Lisbon, Portugal.ORCID https://orcid.org/0000-0003-0934-9631
Davide PareysonUnit of Rare Neurological Diseases, Department of Clinical Neurosciences, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.ORCID https://orcid.org/0000-0001-6854-765X
João Nuno Marques Parracho Guerra da CostaLaboratório de Farmacologia Clínica e Terapêutica, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.
Task Force of the EAN/PNS Joint Guideline for the Management of Neurological Manifestations in Hereditary Transthyretin Amyloidosis

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundWe used network meta-analyses to evaluate the pharmacological interventions for Hereditary Transthyretin-related Amyloidosis with Polyneuropathy (ATTRv-PN).

methodsWe searched Medline, Embase, and Cochrane (June 2025) for randomized trials assessing pharmacological interventions in ATTRv-PN adults. Two reviewers independently screened, extracted data, and assessed risk of bias. Primary efficacy outcomes were mNIS+7 and Norfolk-QoL-DN. Primary safety outcome was serious adverse events (SAE). We used Bayesian hierarchical models. Evidence certainty was assessed using GRADE.

resultsSix trials (n = 989) were included (3 at high-risk of bias). Participant ages and disease duration ranged from 52.8 to 62.0 and 1.4 to 3.9 years, respectively. For mNIS+7, data were available for all interventions except tafamidis. All demonstrated statistically significant improvements versus placebo. Vutrisiran (standardized mean difference [SMD] vs. placebo: -1.66; 95% credible interval [CrI]: -2.13 to -1.17) and patisiran (SMD vs. placebo: -1.56; 95% CrI: -1.88 to -1.25) demonstrated improvements compared with diflunisal, eplontersen, and inotersen. For Norfolk-QoL-DN, data were available for all interventions except diflunisal. All except tafamidis demonstrated statistically significant improvements versus placebo. Patisiran (MD vs. placebo: -17.39; 95% CrI: -23.22 to -11.57), vutrisiran (MD vs. placebo: -16.99; 95% CrI: -25.24 to -8.72), and eplontersen (MD vs. placebo: -15.56; 95% CrI: -21.97 to -9.15) demonstrated improvements compared with tafamidis. For SAE, there were no differences between active interventions versus placebo. Confidence in the evidence varied from very low to moderate.

conclusionGene-silencing therapies were more efficacious, although these findings should be regarded as hypothesis-generating given the scarcity of data, lack of head-to-head trials, and clinical heterogeneity across trials.

Indexed as

Amyloid Neuropathies, FamilialPolyneuropathiesHumansRandomized Controlled Trials as TopicRNA, Small InterferingpatisiranRNA, Small Interferingclinical trialsfamilial amyloid polyneuropathynetwork meta‐analysis

Identifiers

PMID42266139
PMCPMC13250634

What OpenQuestion 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 OpenQuestion graph.