Evidence map›Paper›PMID 36829087›Full record

ArticleActa neurologica Belgica2023

A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium.

Jan L De Bleecker, Kristl G Claeys, Stéphanie Delstanche, Vinciane Van Parys, Jonathan Baets, Sébastien Tilleux, Gauthier Remiche

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Acta neurologica Belgica, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed, 1 pooled it
1.5field-weighted citation impact, top 18% 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.

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

7 citing papers in PubMed, 1 synthesis or guideline pooled it, 10 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Article
  4. 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 · 2024
    Observational
  5. Review
  6. Article
  7. 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

7 authors at 7 institutions in 2 countries.

Jan L De BleeckerDepartment of Neurology, University Hospital Ghent, Ghent, Belgium.ORCID http://orcid.org/0000-0002-1328-1812
Kristl G ClaeysDepartment of Neurology, University Hospitals Leuven, Laboratory for Muscle Diseases and Neuropathies, KU Leuven, Leuven, Belgium.ORCID http://orcid.org/0000-0001-9937-443X
Stéphanie DelstancheDepartment of Neurology, Centre Hospitalier Universitaire de Liège, Liège, Belgium.ORCID http://orcid.org/0000-0003-4481-579X
Vinciane Van ParysDepartment of Neurology, Cliniques Universitaires Saint-Luc, Brussels, Belgium.
Jonathan BaetsDepartment of Neurology, Antwerp University Hospital, Faculty of Medicine and Health Sciences, Translational Neurosciences, UAntwerpen, Antwerp, Belgium.ORCID http://orcid.org/0000-0001-8295-5385
Sébastien TilleuxAlnylam Belgium BVBA, Schumanplein 6, 1040, Brussels, Belgium. stilleux@alnylam.com.ORCID http://orcid.org/0000-0002-0094-5712
Gauthier RemicheDepartment of Neurology, Hôpital Erasme, Université Libre de Bruxelles (ULB), Brussels, Belgium.ORCID http://orcid.org/0000-0002-4882-3570
Alnylam Pharmaceuticals (United States) · USCentre Hospitalier Universitaire de Liège · BECliniques Universitaires Saint-Luc · BEGhent University Hospital · BEKU Leuven · BEUniversité Libre de Bruxelles · BEUniversity of Antwerp · BE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionHereditary transthyretin-mediated (hATTR) amyloidosis, a genetic disease caused by mutations in the transthyretin gene, leads to progressive sensory and autonomic neuropathy and/or cardiomyopathy and is associated with renal and ophthalmologic manifestations and a poor prognosis.

methodsThis is a retrospective study based on data collected from the medical records of patients with hATTR amyloidosis treated with patisiran between 01 July 2018 and 01 February 2021. Six Belgian neuromuscular reference centers participated, covering all patisiran-treated hATTR amyloidosis patients at the study time. This study was conducted to collect data requested in the context of the reimbursement of patisiran in Belgium.

resultsThirty-one patients were diagnosed with hATTR amyloidosis with polyneuropathy, Coutinho stage 1 or 2, and eligible for active treatment during the data collection period. Of the hATTR amyloidosis patients treated with patisiran (n = 12), seven and five had polyneuropathy stages 1 and 2, respectively. Six patients had cardiac symptoms (New York Heart Association class 2 or above). Follow-up information was available for nine patients. Following patisiran treatment, eight patients showed stable or improved assessments for most neurological or cardiological parameters. Only one patient presented with worsening statuses at the end of the data collection period.

conclusionsThe patients with hATTR amyloidosis in Belgium have similar baseline demographics and disease characteristics to those studied in the patisiran APOLLO study and show a similar therapeutic response in the real-world, altering the expected disease progression in most patients.

Indexed as

Amyloid Neuropathies, FamilialPolyneuropathiesBelgiumHumansPrealbuminRetrospective StudiesRNA, Small InterferingpatisiranPrealbuminRNA, Small InterferinghATTRPatisiranPolyneuropathyReal-world evidence

Identifiers

PMID36829087
PMCPMC10238330
OpenAlexW4321782374

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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 OpenQuestion graph.