The DOI matches its Crossref record; 32 of the 32 references it cites were found in a registry or an index.
Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy
Open accessAbstract
Tafamidis, a transthyretin (TTR) kinetic stabilizer, delayed neuropathic progression in patients with Val30Met TTR familial amyloid polyneuropathy (TTR-FAP) in an 18-month randomized controlled trial (study Fx-005). This 12-month, open-label extension study evaluated the long-term safety, tolerability, and efficacy of tafamidis 20 mg once daily in 86 patients who earlier received blinded treatment with tafamidis or placebo. Efficacy measures included the Neuropathy Impairment Score in the Lower Limbs (NIS-LL), Norfolk Quality of Life-Diabetic Neuropathy total quality of life (TQOL) score, and changes in neurologic function and nutritional status. We quantified the monthly rates of change in efficacy measures, and TTR stabilization, and monitored adverse events (AEs). Patients who continued on tafamidis had stable rates of change in NIS-LL (from 0.08 to 0.11/month; p = 0.60) and TQOL (from -0.03 to 0.25; p = 0.16). In patients switched from placebo, the monthly rate of change in NIS-LL declined (from 0.34 to 0.16/month; p = 0.01), as did TQOL score (from 0.61 to -0.16; p < 0.001). Patients treated with tafamidis for 30 months had 55.9 % greater preservation of neurologic function as measured by the NIS-LL than patients in whom tafamidis was initiated later. Plasma TTR was stabilized in 94.1 % of patients treated with tafamidis for 30 months. AEs were similar between groups; no patients discontinued because of an AE. Long-term tafamidis was well tolerated, with the reduced rate of neurologic deterioration sustained over 30 months. Tafamidis also slowed neurologic impairment in patients previously given placebo, but treatment benefits were greater when tafamidis was begun earlier.
Overview
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Citations
Cites
A Double-Blind, Delayed-Start Trial of Rasagiline in Parkinson's Disease
Olanow, C. W., Rascol, O., Hauser, R., Feigin, P. D., Jankovic, J., Lang, A., Langston, W., Melamed, E., Poewe, W., Stocchi, F., et al. (2009) · New England Journal of Medicine · doi:10.1056/nejmoa0809335 · Cited by 901
Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial
Coelho, T., Maia, L. F., Martins da Silva, A., Waddington Cruz, M., Planté-Bordeneuve, V., Lozeron, P., Suhr, O. B., Campistol, J. M., Conceição, I. M., Schmidt, H. H.-J., et al. (2012) · Neurology · doi:10.1212/wnl.0b013e3182661eb1 · Cited by 816
Self-citationTafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade
Bulawa, C. E., Connelly, S., DeVit, M., Wang, L., Weigel, C., Fleming, J. A., Packman, J., Powers, E. T., Wiseman, R. L., Foss, T. R., et al. (2012) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1121005109 · Cited by 742
Self-citationHolmgren, G., Steen, L., Suhr, O., Ericzon, B.-G., Groth, C.-G., Andersen, O., Wallin, B. G., Seymour, A., Richardson, S., Hawkins, P. N., et al. (1993) · The Lancet · doi:10.1016/0140-6736(93)93127-m · Cited by 579
Self-citationPrevention of Transthyretin Amyloid Disease by Changing Protein Misfolding Energetics
Hammarström, P., Wiseman, R. L., Powers, E. T., & Kelly, J. W. (2003) · Science · doi:10.1126/science.1079589 · Cited by 525
Self-citationTabulation of human transthyretin (TTR) variants, 2003
Connors, L. H., Lim, A., Prokaeva, T., Roskens, V. A., & Costello, C. E. (2003) · Amyloid · doi:10.3109/13506120308998998 · Cited by 505
A Controlled, Randomized, Delayed-Start Study of Rasagiline in Early Parkinson Disease
(2004) · Archives of Neurology · doi:10.1001/archneur.61.4.561 · Cited by 485
Transthyretin-Related Familial Amyloidotic Polyneuropathy
Ando, Y., Nakamura, M., & Araki, S. (2005) · Archives of Neurology · doi:10.1001/archneur.62.7.1057 · Cited by 416
Structure of a complex of two plasma proteins: transthyretin and retinol-binding protein
Monaco, H., Rizzi, M., & Coda, A. (1995) · Science · doi:10.1126/science.7754382 · Cited by 409
Dyck, P. J., Davies, J. L., Litchy, W. J., & O'Brien, P. C. (1997) · Neurology · doi:10.1212/wnl.49.1.229 · Cited by 382
Cited by
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
Adams, D., Gonzalez-Duarte, A., O’Riordan, W. D., Yang, C.-C., Ueda, M., Kristen, A. V., Tournev, I., Schmidt, H. H., Coelho, T., Berk, J. L., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1716153 · Cited by 3,047
Self-citationGenotype and Phenotype of Transthyretin Cardiac Amyloidosis
Maurer, M. S., Hanna, M., Grogan, M., Dispenzieri, A., Witteles, R., Drachman, B., Judge, D. P., Lenihan, D. J., Gottlieb, S. S., Shah, S. J., et al. (2016) · Journal of the American College of Cardiology · doi:10.1016/j.jacc.2016.03.596 · Cited by 538
Self-citationDiagnosis, Prognosis, and Therapy of Transthyretin Amyloidosis
Gertz, M. A., Benson, M. D., Dyck, P. J., Grogan, M., Coelho, T., Cruz, M., Berk, J. L., Plante-Bordeneuve, V., Schmidt, H. H. J., & Merlini, G. (2015) · Journal of the American College of Cardiology · doi:10.1016/j.jacc.2015.09.075 · Cited by 475
Self-citationSuhr, O. B., Coelho, T., Buades, J., Pouget, J., Conceicao, I., Berk, J., Schmidt, H., Waddington-Cruz, M., Campistol, J. M., Bettencourt, B. R., et al. (2015) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-015-0326-6 · Cited by 321
Self-citationAdvances in the treatment of hereditary transthyretin amyloidosis: a review
Gertz, M. A., Mauermann, M. L., Grogan, M., & Coelho, T. (2019) · Brain and Behavior · doi:10.1002/brb3.1371 · Cited by 102
Self-citation
Metadata
- Authors
- Coelho, T., Maia, L. F., da Silva, A. M., Cruz, M. W., Planté-Bordeneuve, V., Suhr, O. B., Conceiçao, I., Schmidt, H. H.-J., Trigo, P., Kelly, J. W., Labaudinière, R., Chan, J., Packman, J., & Grogan, D. R.
- Venue
- Journal of Neurology
- Published
- 2013-08-22
- Volume
- 260
- Issue
- 11
- Pages
- 2802-2814
- ISSN
- 0012-1037 (ISSN-L)
- 0340-5354
- 1432-1459
- OpenAlex
- W2080914865
- Cited by
- 344
- References
- 32
- FWCI
- 13.18
- Type
- journal article
- Language
- en
- Open access
- hybrid
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
How to cite
Cite CoChat for the checks on this page, and the paper for its own findings.
- This page
- CoChat. (2026). Citation check: Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy. https://app.cochat.ai/papers/10.1007/s00415-013-7051-7
- The paper
- Coelho, T., Maia, L. F., da Silva, A. M., Cruz, M. W., Planté-Bordeneuve, V., Suhr, O. B., Conceiçao, I., Schmidt, H. H.-J., Trigo, P., Kelly, J. W., Labaudinière, R., Chan, J., Packman, J., & Grogan, D. R. (2013). Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy. Journal of Neurology. https://doi.org/10.1007/s00415-013-7051-7
Data sources
Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
