Evidence map›Paper›PMID 40974604›Full record

Trial reportAnnals of medicine2025

Impact of patisiran on polyneuropathy of hereditary transthyretin amyloidosis in patients with a V122I or T60A variant: a phase IV multicenter study.

Yessar Hussain, Saurabh Malhotra, Brett W Sperry, Francy Shu, Karthikeyan Ananthasubramaniam, Urvi Desai, Ugochukwu Egolum, Joel Fernandez, Miriam Freimer, Elizabeth Mauricio and 8 more

Abstract readMulticenter StudyClinical Trial, Phase IV
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Annals of medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

18 authors.

Yessar HussainAustin Neuromuscular Center, Austin, TX, USA.
Saurabh MalhotraDivision of Cardiology, Cook County Health and Rush Medical College, Chicago, IL, USA.ORCID 0000-0002-9396-9579
Brett W SperryDepartment of Cardiovascular Medicine, Saint Luke's Mid America Heart Institute, Kansas City, MO, USA.ORCID 0000-0002-8210-6401
Francy ShuDepartment of Neuroscience, Baylor University Medical Center/Baylor Scott and White Health, Dallas, TX, USA.ORCID 0000-0002-4670-1561
Karthikeyan AnanthasubramaniamDepartment of Cardiology, Henry Ford West Bloomfield Hospital, West Bloomfield Township, MI, USA.ORCID 0000-0001-5837-496X
Urvi DesaiDepartment of Neurology, Atrium Health Wake Forest University, Charlotte, NC, USA.ORCID 0000-0002-5169-3201
Ugochukwu EgolumNortheast Georgia Medical Center, Division of Cardiology, Georgia Heart Institute, Gainesville, GA, USA.ORCID 0000-0002-9606-1347
Joel FernandezDepartment of Cardiovascular Services, Morsani College of Medicine, University of South Florida, Tampa, FL, USA.ORCID 0000-0003-4008-4301
Miriam FreimerDepartment of Neurology, Wexner Medical Center, Ohio State University, Columbus, OH, USA.ORCID 0000-0001-8282-764X
Elizabeth MauricioDepartment of Neurology, Mayo Clinic, Jacksonville, FL, USA.ORCID 0000-0002-9493-9777
Dianna QuanDepartment of Neurology, University of Colorado Anschutz, Aurora, CO, USA.ORCID 0000-0002-3699-5252
Roy SmallDepartment of Cardiology, Lancaster General Health/Penn Medicine, Lancaster, PA, USA.
Kelley CapocelliAlnylam Pharmaceuticals, Cambridge, MA, USA.ORCID 0000-0002-8256-2896
Steven RoblinAlnylam Pharmaceuticals, Cambridge, MA, USA.
Shaun BenderAlnylam Pharmaceuticals, Cambridge, MA, USA.ORCID 0000-0002-7589-4534
Patrick Y JayAlnylam Pharmaceuticals, Cambridge, MA, USA.ORCID 0000-0001-9429-5492
Elena YurenevaAlnylam Pharmaceuticals, Cambridge, MA, USA.
Ronald ZoltyDepartment of Cardiology, University of Nebraska Medical Center, Omaha, NE, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThis study assessed the effectiveness and safety of patisiran in patients with V122I/T60A variant transthyretin (ATTRv) amyloidosis with polyneuropathy. These variants have been under-represented in previous trials of gene-silencing agents.

methodsThis was a multicenter, phase IV study conducted at 27 sites in the USA. Patients were ≥ 18 years, diagnosed with ATTRv amyloidosis with polyneuropathy and a documented V122I or T60A variant. Patisiran-treated patients were enrolled prospectively, ambispectively, and retrospectively. The primary endpoint was the proportion of patients with a stable or improved polyneuropathy disability (PND) score at 12 months

resultsSixty-seven patients were enrolled, of whom 58 received ≥ 1 dose of patisiran. In the efficacy population, 42/45 (93.3%) patients demonstrated stable or improved PND scores from baseline to Month 12. Patients also showed stable or improved quality of life, health status, autonomic symptoms, and cardiac function

conclusionsPatisiran demonstrated a consistent positive effect across multiple endpoints in patients with V122I/T60A ATTRv amyloidosis, including polyneuropathy manifestations.

Indexed as

Amyloid Neuropathies, FamilialPolyneuropathiesPrealbuminAdultAgedAged, 80 and overFemaleHumansMaleMiddle AgedProspective StudiesQuality of LifeRetrospective StudiesRNA, Small InterferingTreatment OutcomepatisiranPrealbuminRNA, Small InterferingATTRv amyloidosispatisiranpolyneuropathyRNA interferencetreatment

Identifiers

PMID40974604
PMCPMC12451958

What Socratic holds

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Registered trials

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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.