title: Assessing mNIS+7Ionisand international neurologists' proficiency in a familial amyloidotic polyneuropathy trial.
abstract: BACKGROUND
  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+7Ionissensitively 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.
authors.affiliation: Department of Neurology, Mayo Clinic, 200 First Street SW, Rochester, Minnesota, 55905, USA.
authors.forename: Peter J
authors.initials: PJ
authors.lastname: Dyck
authors.affiliation: Indiana University, IU Health, Indianapolis, Indiana, USA.
authors.forename: John C
authors.initials: JC
authors.lastname: Kincaid
authors.affiliation: Department of Neurology, Mayo Clinic, 200 First Street SW, Rochester, Minnesota, 55905, USA.
authors.forename: P James B
authors.initials: PJB
authors.lastname: Dyck
authors.affiliation: Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
authors.forename: Vinay
authors.initials: V
authors.lastname: Chaudhry
authors.affiliation: University of California Irvine, Irvine, California, USA.
authors.forename: Namita A
authors.initials: NA
authors.lastname: Goyal
authors.affiliation: Hospital Santo António, Centro Hospitalar Porto, Porto, Portugal.
authors.forename: Christina
authors.initials: C
authors.lastname: Alves
authors.affiliation: Hôpitaux Universitaires Henri Mondor, Créteil, France.
authors.forename: Hayet
authors.initials: H
authors.lastname: Salhi
authors.affiliation: Boston Medical Center, Boston, Massachusetts, USA.
authors.forename: Janice F
authors.initials: JF
authors.lastname: Wiesman
authors.affiliation: CHU Bicêtre, French Reference Center for FAP (NNERF), Le Kremlin Bicêtre, France.
authors.forename: Celine
authors.initials: C
authors.lastname: Labeyrie
authors.affiliation: Icahn School of Medicine at Mount Sinai, New York, New York, USA.
authors.forename: Jessica
authors.initials: J
authors.lastname: Robinson-Papp
authors.affiliation: Hospital Santo António, Centro Hospitalar Porto, Porto, Portugal.
authors.forename: Márcio
authors.initials: M
authors.lastname: Cardoso
authors.affiliation: National Hospital for Neurology and Neurosurgery, London, United Kingdom.
authors.forename: Matilde
authors.initials: M
authors.lastname: Laura
authors.affiliation: Medical University of South Carolina, Charleston, South Carolina, USA.
authors.forename: Katherine
authors.initials: K
authors.lastname: Ruzhansky
authors.affiliation: C. Mondino National Neurological Institute, Pavia, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Cortese
authors.affiliation: Columbia University, New York, New York, USA.
authors.forename: Thomas H
authors.initials: TH
authors.lastname: Brannagan
authors.affiliation: Oregon Health & Science University, Portland, Oregon, USA.
authors.forename: Julie
authors.initials: J
authors.lastname: Khoury
authors.affiliation: Penn Presbyterian Medical Center, Philadelphia, Pennsylvania, USA.
authors.forename: Sami
authors.initials: S
authors.lastname: Khella
authors.affiliation: Clementino Fraga Filho University Hospital, Federal University of Rio de Janerio, Rio de Janerio, Brazil.
authors.forename: Márcia
authors.initials: M
authors.lastname: Waddington-Cruz
authors.affiliation: New University of Lisbon, Lisbon, Portugal.
authors.forename: João
authors.initials: J
authors.lastname: Ferreira
authors.affiliation: University of California-Irvine, Orange, California, USA.
authors.forename: Annabel K
authors.initials: AK
authors.lastname: Wang
authors.affiliation: Clementino Fraga Filho University Hospital, Federal University of Rio de Janerio, Rio de Janerio, Brazil.
authors.forename: Marcus V
authors.initials: MV
authors.lastname: Pinto
authors.affiliation: Hôpitaux Universitaires Henri Mondor, Créteil, France.
authors.forename: Samar S
authors.initials: SS
authors.lastname: Ayache
authors.affiliation: Indiana University School of Medicine, Indianapolis, Indiana, USA.
authors.forename: Merrill D
authors.initials: MD
authors.lastname: Benson
authors.affiliation: Boston University School of Medicine, Boston, Massachusetts, USA.
authors.forename: John L
authors.initials: JL
authors.lastname: Berk
authors.affiliation: Hospital Santo António, Centro Hospitalar Porto, Porto, Portugal.
authors.forename: Teresa
authors.initials: T
authors.lastname: Coelho
authors.affiliation: Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
authors.forename: Michael
authors.initials: M
authors.lastname: Polydefkis
authors.affiliation: Icahn School of Medicine at Mount Sinai, New York, New York, USA.
authors.forename: Peter
authors.initials: P
authors.lastname: Gorevic
authors.affiliation: CHU Bicêtre, French Reference Center for FAP (NNERF), Le Kremlin Bicêtre, France.
authors.forename: David H
authors.initials: DH
authors.lastname: Adams
authors.affiliation: Hôpitaux Universitaires Henri Mondor, Créteil, France.
authors.forename: Violaine
authors.initials: V
authors.lastname: Plante-Bordeneuve
authors.affiliation: National Hospital for Neurology and Neurosurgery, London, United Kingdom.
authors.forename: Carol
authors.initials: C
authors.lastname: Whelan
authors.affiliation: Foundation IRCCS Policlinico San Matteo, University of Pavia, Italy.
authors.forename: Giampaolo
authors.initials: G
authors.lastname: Merlini
authors.affiliation: Oregon Health & Science University, Portland, Oregon, USA.
authors.forename: Stephen
authors.initials: S
authors.lastname: Heitner
authors.affiliation: Penn Presbyterian Medical Center, Philadelphia, Pennsylvania, USA.
authors.forename: Brian M
authors.initials: BM
authors.lastname: Drachman
authors.affiliation: New University of Lisbon, Lisbon, Portugal.
authors.forename: Isabel
authors.initials: I
authors.lastname: Conceição
authors.affiliation: Department of Neurology, Mayo Clinic, 200 First Street SW, Rochester, Minnesota, 55905, USA.
authors.forename: Christopher J
authors.initials: CJ
authors.lastname: Klein
authors.affiliation: Department of Neurology, Mayo Clinic, 200 First Street SW, Rochester, Minnesota, 55905, USA.
authors.forename: Morie A
authors.initials: MA
authors.lastname: Gertz
authors.affiliation: Ionis Pharmaceuticals, Inc, Carlsbad, California, USA.
authors.forename: Elizabeth J
authors.initials: EJ
authors.lastname: Ackermann
authors.affiliation: Ionis Pharmaceuticals, Inc, Carlsbad, California, USA.
authors.forename: Steven G
authors.initials: SG
authors.lastname: Hughes
authors.affiliation: Department of Neurology, Mayo Clinic, 200 First Street SW, Rochester, Minnesota, 55905, USA.
authors.forename: Michelle L
authors.initials: ML
authors.lastname: Mauermann
authors.affiliation: GlaxoSmithKline, Middlesex, United Kingdom.
authors.forename: Rito
authors.initials: R
authors.lastname: Bergemann
authors.affiliation: Department of Neurology, Mayo Clinic, 200 First Street SW, Rochester, Minnesota, 55905, USA.
authors.forename: Karen A
authors.initials: KA
authors.lastname: Lodermeier
authors.affiliation: Department of Neurology, Mayo Clinic, 200 First Street SW, Rochester, Minnesota, 55905, USA.
authors.forename: Jenny L
authors.initials: JL
authors.lastname: Davies
authors.affiliation: Department of Neurology, Mayo Clinic, 200 First Street SW, Rochester, Minnesota, 55905, USA.
authors.forename: Rickey E
authors.initials: RE
authors.lastname: Carter
authors.affiliation: Department of Neurology, Mayo Clinic, 200 First Street SW, Rochester, Minnesota, 55905, USA.
authors.forename: William J
authors.initials: WJ
authors.lastname: Litchy
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D028227:Amyloid Neuropathies, Familial / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D015331:Cohort Studies; D003943:Diagnostic Techniques, Neurological; D004185:Disability Evaluation; D005260:Female; D006801:Humans; D007391:International Cooperation; D008297:Male; D008875:Middle Aged; D009431:Neural Conduction / Q000187:drug effects / Q000502:physiology; D000072141:Neurologists; D009841:Oligonucleotides / Q000627:therapeutic use*; D017063:Outcome Assessment, Health Care; D012720:Severity of Illness Index
chemical_list: D009841:Oligonucleotides
keywords: disability; familial amyloidotic polyneuropathy (FAP); neurophysiologic tests; oligonucleotide trials; peripheral neuropathy; polyneuropathy signs; proficiency