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

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 mNIS+7Ionis and its subscores and correlation with disability and health scores. Results The modified Neuropathy Impairment Score + 7 neurophysiologic tests (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. Discussion 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.

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