Literature DB >> 28063170

Assessing mNIS+7Ionis and international neurologists' proficiency in a familial amyloidotic polyneuropathy trial.

Peter J Dyck1, John C Kincaid2, P James B Dyck1, Vinay Chaudhry3, Namita A Goyal4, Christina Alves5, Hayet Salhi6, Janice F Wiesman7, Celine Labeyrie8, Jessica Robinson-Papp9, Márcio Cardoso5, Matilde Laura10, Katherine Ruzhansky11, Andrea Cortese12, Thomas H Brannagan13, Julie Khoury14, Sami Khella15, Márcia Waddington-Cruz16, João Ferreira17, Annabel K Wang18, Marcus V Pinto16, Samar S Ayache6, Merrill D Benson19, John L Berk20, Teresa Coelho5, Michael Polydefkis3, Peter Gorevic9, David H Adams8, Violaine Plante-Bordeneuve6, Carol Whelan10, Giampaolo Merlini21, Stephen Heitner14, Brian M Drachman15, Isabel Conceição17, Christopher J Klein1, Morie A Gertz1, Elizabeth J Ackermann22, Steven G Hughes22, Michelle L Mauermann1, Rito Bergemann23, Karen A Lodermeier1, Jenny L Davies1, Rickey E Carter1, William J Litchy1.   

Abstract

INTRODUCTION: Polyneuropathy signs (Neuropathy Impairment Score, NIS), neurophysiologic tests (m+7Ionis ), disability, and health scores were assessed in baseline evaluations of 100 patients entered into an oligonucleotide familial amyloidotic polyneuropathy (FAP) trial.
METHODS: We assessed: (1) Proficiency of grading neurologic signs and correlation with neurophysiologic tests, and (2) clinometric performance of modified NIS+7 neurophysiologic tests (mNIS+7Ionis ) and its subscores and correlation with disability and health scores.
RESULTS: The mNIS+7Ionis sensitively detected, characterized, and broadly scaled diverse polyneuropathy impairments. Polyneuropathy signs (NIS and subscores) correlated with neurophysiology tests, disability, and health scores. Smart Somatotopic Quantitative Sensation Testing of heat as pain 5 provided a needed measure of small fiber involvement not adequately assessed by other tests.
CONCLUSIONS: Specially trained neurologists accurately assessed neuropathy signs as compared to referenced neurophysiologic tests. The score, mNIS+7Ionis , broadly detected, characterized, and scaled polyneuropathy abnormality in FAP, which correlated with disability and health scores. Muscle Nerve 56: 901-911, 2017.
© 2017 Wiley Periodicals, Inc.

Entities:  

Keywords:  disability; familial amyloidotic polyneuropathy (FAP); neurophysiologic tests; oligonucleotide trials; peripheral neuropathy; polyneuropathy signs; proficiency

Mesh:

Substances:

Year:  2017        PMID: 28063170      PMCID: PMC5500439          DOI: 10.1002/mus.25563

Source DB:  PubMed          Journal:  Muscle Nerve        ISSN: 0148-639X            Impact factor:   3.217


  37 in total

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Journal:  Muscle Nerve       Date:  2014-01-28       Impact factor: 3.217

Review 4.  Nearly 200 X-ray crystal structures of transthyretin: what do they tell us about this protein and the design of drugs for TTR amyloidoses?

Authors:  S K Palaninathan
Journal:  Curr Med Chem       Date:  2012       Impact factor: 4.530

5.  Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30).

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6.  Structure of a complex of two plasma proteins: transthyretin and retinol-binding protein.

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7.  Structure of prealbumin: secondary, tertiary and quaternary interactions determined by Fourier refinement at 1.8 A.

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8.  The prevalence by staged severity of various types of diabetic neuropathy, retinopathy, and nephropathy in a population-based cohort: the Rochester Diabetic Neuropathy Study.

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9.  Diagnostic hallmarks and pitfalls in late-onset progressive transthyretin-related amyloid-neuropathy.

Authors:  Maike F Dohrn; Christoph Röcken; Jan L De Bleecker; Jean-Jacques Martin; Matthias Vorgerd; Peter Y Van den Bergh; Andreas Ferbert; Katrin Hinderhofer; J Michael Schröder; Joachim Weis; Jörg B Schulz; Kristl G Claeys
Journal:  J Neurol       Date:  2013-10-08       Impact factor: 4.849

10.  Retrospective study of a TTR FAP cohort to modify NIS+7 for therapeutic trials.

Authors:  N Suanprasert; J L Berk; M D Benson; P J B Dyck; C J Klein; J A Gollob; B R Bettencourt; V Karsten; P J Dyck
Journal:  J Neurol Sci       Date:  2014-06-27       Impact factor: 3.181

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  14 in total

Review 1.  Autonomic involvement in hereditary transthyretin amyloidosis (hATTR amyloidosis).

Authors:  Alejandra Gonzalez-Duarte
Journal:  Clin Auton Res       Date:  2018-03-06       Impact factor: 4.435

2.  Muscle quantitative MRI as a novel biomarker in hereditary transthyretin amyloidosis with polyneuropathy: a cross-sectional study.

Authors:  Elisa Vegezzi; Andrea Cortese; Laura Obici; Anna Pichiecchio; Niels Bergsland; Roberta Mussinelli; Matteo Paoletti; Francesca Solazzo; Riccardo Currò; Lucia Ascagni; Ilaria Callegari; Ilaria Quartesan; Alessandro Lozza; Xeni Deligianni; Francesco Santini; Enrico Marchioni; Giuseppe Cosentino; Enrico Alfonsi; Cristina Tassorelli; Stefano Bastianello; Giampaolo Merlini; Giovanni Palladini
Journal:  J Neurol       Date:  2022-09-06       Impact factor: 6.682

3.  Somatotopic heat pain thresholds and intraepidermal nerve fibers in health.

Authors:  Jenny L Davies; Janean K Engelstad; Linde E Gove; Linda K Linbo; Rickey E Carter; Christopher Lynch; Nathan P Staff; Christopher J Klein; P James B Dyck; David N Herrmann; Peter J Dyck
Journal:  Muscle Nerve       Date:  2018-04-20       Impact factor: 3.217

Review 4.  Transthyretin familial amyloid polyneuropathy: an update.

Authors:  Violaine Plante-Bordeneuve
Journal:  J Neurol       Date:  2017-12-16       Impact factor: 4.849

5.  Pharmacological treatment for familial amyloid polyneuropathy.

Authors:  Francesca Magrinelli; Gian Maria Fabrizi; Lucio Santoro; Fiore Manganelli; Giampietro Zanette; Tiziana Cavallaro; Stefano Tamburin
Journal:  Cochrane Database Syst Rev       Date:  2020-04-20

Review 6.  Novel RNA-targeted therapies for hereditary ATTR amyloidosis and their impact on the autonomic nervous system.

Authors:  Isabel Conceição
Journal:  Clin Auton Res       Date:  2019-08-09       Impact factor: 4.435

7.  The use of validated and nonvalidated surrogate endpoints in two European Medicines Agency expedited approval pathways: A cross-sectional study of products authorised 2011-2018.

Authors:  Catherine Schuster Bruce; Petra Brhlikova; Joseph Heath; Patricia McGettigan
Journal:  PLoS Med       Date:  2019-09-10       Impact factor: 11.069

Review 8.  Expert consensus recommendations to improve diagnosis of ATTR amyloidosis with polyneuropathy.

Authors:  David Adams; Yukio Ando; João Melo Beirão; Teresa Coelho; Morie A Gertz; Julian D Gillmore; Philip N Hawkins; Isabelle Lousada; Ole B Suhr; Giampaolo Merlini
Journal:  J Neurol       Date:  2020-01-06       Impact factor: 4.849

9.  Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy.

Authors:  David Adams; Ole B Suhr; Peter J Dyck; William J Litchy; Raina G Leahy; Jihong Chen; Jared Gollob; Teresa Coelho
Journal:  BMC Neurol       Date:  2017-09-11       Impact factor: 2.474

10.  Somatostatin analogues for refractory diarrhoea in familial amyloid polyneuropathy.

Authors:  Michael Collins; Anna Pellat; Guillemette Antoni; Hélène Agostini; Céline Labeyrie; David Adams; Franck Carbonnel
Journal:  PLoS One       Date:  2018-08-30       Impact factor: 3.240

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