Evidence map›Paper›PMID 39096004›Full record

ArticleAnnals of clinical and translational neurology2024

Diflunisal versus tafamidis on neuropathy and cardiomyopathy in hereditary transthyretin amyloidosis.

Chi-Chao Chao, Shiou-Ru Tzeng, Ming-Chang Chiang, Hsueh-Wen Hsueh, Wan-Jen Hsieh, Yuan-Chun Chao, Mei-Fang Cheng, Yen-Hung Lin, Mao-Yuan Su, Chun-Hsiang Huang and 4 more

Abstract readComparative Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Annals of clinical and translational neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

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2 · The registry

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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

2 citing papers in PubMed.

  1. Review
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4 · The record

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5 · Who and what money

Authors and funding

14 authors.

Chi-Chao ChaoDepartment of Neurology, National Taiwan University Hospital, Taipei, Taiwan.ORCID 0000-0001-6499-5789
Shiou-Ru TzengInstitute of Biochemistry and Molecular Biology, College of Medicine, National Taiwan University, Taipei, Taiwan.ORCID 0000-0002-7180-0190
Ming-Chang ChiangDepartment of Biomedical Engineering, National Yang Ming Chiao Tung University, Taipei, Taiwan.ORCID 0000-0003-4286-6032
Hsueh-Wen HsuehDepartment of Neurology, National Taiwan University Hospital, Taipei, Taiwan.ORCID 0000-0003-4707-1861
Wan-Jen HsiehDepartment of Neurology, National Taiwan University Hospital, Taipei, Taiwan.
Yuan-Chun ChaoDepartment of Medicine, National Yang Ming Chiao Tung University, Taipei, Taiwan.
Mei-Fang ChengDepartment of Nuclear Medicine, National Taiwan University Hospital, Taipei, Taiwan.
Yen-Hung LinDepartment of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan.
Mao-Yuan SuDepartment of Medical Imaging, National Taiwan University Hospital, Taipei, Taiwan.
Chun-Hsiang HuangProtein Diffraction Group, Experimental Facility Division, National Synchrotron Radiation Research Center, Hsinchu, Taiwan.
Yi-Shiang WangInstitute of Biochemistry and Molecular Biology, College of Medicine, National Taiwan University, Taipei, Taiwan.
Ming-Fang HsiehInstitute of Biochemistry and Molecular Biology, College of Medicine, National Taiwan University, Taipei, Taiwan.
Ping-Huei TsengDepartment of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan.
Sung-Tsang HsiehDepartment of Neurology, National Taiwan University Hospital, Taipei, Taiwan.ORCID 0000-0002-3188-8482

Funding

Ministry of Science and Technology, Taiwan 107-2314-B-002-072-MY2Ministry of Science and Technology, Taiwan 109-2320-B-002-024Ministry of Science and Technology, Taiwan 110-2320-B-002-072Ministry of Science and Technology, Taiwan 110-2320-B-002-075Ministry of Science and Technology, Taiwan 111-2320-B-002-078National Taiwan University Hospital UN109-004
6 · The paper itself

Abstract

objectivesHereditary transthyretin (TTR) amyloidosis (ATTRv) is frequently complicated by polyneuropathy (ATTRv-PN) and cardiomyopathy (ATTRv-CM). The long-term efficacy of diflunisal on both polyneuropathy and cardiomyopathy in ATTRv patients, especially those with non-V30M genotypes, has not been fully investigated and compared with that of tafamidis.

methodsWe compared the structural and biochemical characteristics of A97S-TTR complexed with tafamidis with those of diflunisal, and prospectively followed up and compared the progression of polyneuropathy and cardiomyopathy between ATTRv-PN patients taking diflunisal and those taking tafamidis.

resultsBoth diflunisal and tafamidis effectively bind to the two thyroxine-binding sites at the A97S-TTR dimer-dimer interface and equally and almost sufficiently reduce amyloid fibril formation. Thirty-five ATTRv-PN patients receiving diflunisal and 22 patients receiving tafamidis were enrolled. Compared with no treatment, diflunisal treatment significantly delayed the transition of FAP Stage 1 to 2 and Stage 2 to 3 and decreased the deterioration in parameters of the ulnar nerve conduction study (NCS). The progression of FAP stage or NCS parameters did not differ between patients treated with diflunisal and those treated with tafamidis. Both diflunisal and tafamidis treatments significantly decreased radiotracer uptake on INTERPRETATIONS: The binding patterns of both tafamidis and diflunisal to A97S-TTR closely resembled those observed in the wild type. Diflunisal can effectively delay the progression of polyneuropathy and cardiomyopathy with similar efficacy to tafamidis and may become a cost-effective alternative treatment for late-onset ATTRv-PN.

Indexed as

Amyloid Neuropathies, FamilialBenzoxazolesCardiomyopathiesDiflunisalAdultAgedFemaleHumansMaleMiddle AgedPolyneuropathiesPrealbuminBenzoxazolesDiflunisalPrealbumintafamidis

Identifiers

PMID39096004
PMCPMC11537138

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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.