Evidence map›Paper›PMID 37357518›Full record

ArticleCurrent neuropharmacology2023

Does Patisiran Reduce Ocular Transthyretin Synthesis? A Pilot Study of Two Cases.

Chiara Cambieri, Marco Marenco, Tania Colasanti, Carmine Mancone, Alessandro Corsi, Mara Riminucci, Laura Libonati, Federica Moret, Cristina Chimenti, Alessandro Lambiase and 4 more

Open access · greenAbstract readCase Reports
PubMed Full text at PMC Publisher
In one paragraph

Article in Current neuropharmacology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
0.9field-weighted citation impact, top 25% 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

5 citing papers in PubMed, 6 citations in OpenAlex.

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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

14 authors at 3 institutions in 1 country.

Chiara CambieriDepartment of Human Neuroscience, Centre for Rare Neuromuscular Disease, Sapienza University of Rome, Rome, Italy.
Marco MarencoDepartment of Sense Organs, Sapienza University of Rome, Rome, Italy.
Tania ColasantiDepartment of Clinical Internal, Rheumatology Unit, Anesthetic and Cardiovascular Sciences, Sapienza University of Rome, Rome, Italy.
Carmine ManconeDepartment of Molecular Medicine, Sapienza University of Rome, Rome, Italy.
Alessandro CorsiDepartment of Molecular Medicine, Sapienza University of Rome, Rome, Italy.
Mara RiminucciDepartment of Molecular Medicine, Sapienza University of Rome, Rome, Italy.
Laura LibonatiDepartment of Human Neuroscience, Centre for Rare Neuromuscular Disease, Sapienza University of Rome, Rome, Italy.
Federica MoretDepartment of Human Neuroscience, Centre for Rare Neuromuscular Disease, Sapienza University of Rome, Rome, Italy.
Cristina ChimentiDepartment of Clinical, Internal, Anesthesiologist and Cardiovascular Sciences, Sapienza University of Rome, Rome, Italy.
Alessandro LambiaseDepartment of Sense Organs, Sapienza University of Rome, Rome, Italy.
Fabrizio ContiDepartment of Molecular Medicine, Sapienza University of Rome, Rome, Italy.
Matteo GaribaldiDepartment of Neuroscience, Mental Health, and Sensory Organs (NESMOS), Sant'Andrea Hospital, Sapienza University, Rome, Italy.
Maurizio InghilleriDepartment of Human Neuroscience, Centre for Rare Neuromuscular Disease, Sapienza University of Rome, Rome, Italy.
Marco CeccantiDepartment of Human Neuroscience, Centre for Rare Neuromuscular Disease, Sapienza University of Rome, Rome, Italy.
Sapienza University of Rome · ITAzienda Ospedaliera Sant'Andrea · ITIstituto Nazionale per le Malattie Infettive Lazzaro Spallanzani · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundVariant transthyretin-mediated amyloidosis (ATTR-v) is a well-characterized disease affecting the neurologic and cardiovascular systems. Patisiran has been approved for neurologic involvement as it reduces hepatic synthesis of transthyretin (TTR). Eye involvement is a lateonset feature increasing the risk of glaucoma and cataracts in patients.

aimsThe aim of this case series was to assess whether patisiran can effectively reduce TTR synthesis in such a barrier-protected organ as the eye.

methodsTwo patisiran-treated ATTR-v patients underwent serum and aqueous humor sampling to measure TTR levels detected by SDS-PAGE and immunoblotting. Serum samples were compared to healthy control (HC), whereas aqueous humor samples were compared to non-amyloidotic subjects affected by cataracts and glaucoma.

resultsSerum TTR levels representative of hepatic synthesis were sharply lower in treated patients if compared to the HC (-87.5% and -93.75%, respectively). Aqueous humor TTR levels showed mild-tono reduction in treated patients compared to non-amyloidotic subjects with cataracts (-34.9% and +8.1%, respectively) and glaucoma (-41.1% and -2.1%).

conclusionPatisiran does not seem to be as effective in inhibiting ocular TTR synthesis as it is in inhibiting hepatic synthesis. Re-engineering the envelope could allow the drug to target RPE cells thus avoiding any ocular involvement.

Indexed as

CataractGlaucomaAmyloid Neuropathies, FamilialHumansPilot ProjectsPrealbuminRNA, Small InterferingpatisiranPrealbuminRNA, Small InterferingAmyloidosisATTRATTR-vocularpatisiranRNA interferencesiRNAtransthyretin

Identifiers

PMID37357518
PMCPMC10616919
OpenAlexW4382010747

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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.