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Development of measures of polyneuropathy impairment in hATTR amyloidosis: From NIS to mNIS + 7.

Abstract：

:Hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) is a rare, life-threatening disease, caused by point mutations in the transthyretin gene. It is a heterogeneous, multisystem disease with rapidly progressing polyneuropathy (including sensory, motor, and autonomic impairments) and cardiac dysfunction. Measures used to assess polyneuropathy in other diseases have been tested as endpoints in hATTR amyloidosis clinical trials (i.e. Neuropathy Impairment Score [NIS], NIS-lower limb, and NIS + 7), yet the unique nature of the polyneuropathy in this disease has necessitated modifications to these scales. In particular, the heterogeneous impairment and the aggressive disease course have been key drivers in developing scales that better capture the disease burden and progression of polyneuropathy in hATTR amyloidosis. The modified NIS + 7 (mNIS + 7) scale was specifically designed to assess polyneuropathy impairment in patients with hATTR amyloidosis, and has been the primary endpoint in two recent, phase III studies in this disease. The mNIS + 7 uses highly standardized, quantitative, and referenced assessments to quantify decreased muscle weakness, muscle stretch reflexes, sensory loss, and autonomic impairment. Physicians using this scale in clinical trials should be specifically trained and monitored to minimize variability. This article discusses the different scales that have been/are being used to assess polyneuropathy in patients with hATTR amyloidosis, their correlation with other disease assessments, and reflects on how and why scales have evolved to the latest iteration of mNIS + 7.

journal_name

J Neurol Sci

journal_title

Journal of the neurological sciences

authors

Dyck PJB,González-Duarte A,Obici L,Polydefkis M,Wiesman JF,Antonino I,Litchy WJ,Dyck PJ

doi

10.1016/j.jns.2019.116424

subject

Has Abstract

pub_date

2019-10-15 00:00:00

pages

116424

eissn

0022-510X

issn

1878-5883

pii

S0022-510X(19)30356-9

journal_volume

405

pub_type

杂志文章,评审

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