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Table 3 Change in mNIS+7 by components and subgroups at 24 months of patisiran treatment

From: A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis

 

Change from baseline to Month 24

n

Mean (SEM)

Median (range)

mNIS+7 component

 Total

26

−6.95 (2.03)

−7.00 (−34.63 to 15.38)

 NIS-Weakness

26

1.23 (1.43)

0.00 (−13.50 to 24.38)

 NIS-Reflexes

26

−0.48 (0.53)

0.00 (−6.00 to 7.00)

 QST

26

−7.4 (2.04)

−6.0 (−40.0 to 16.0)

 NCS ∑5

26

−0.19 (0.18)

−0.25 (−2.00 to 2.50)

 Postural BP

26

−0.10 (0.06)

0.00 (−1.00 to 0.50)

mNIS+7 by TTR tetramer stabilizer use

 Patisiran alone

7

−6.75 (5.24)

−8.50 (−28.50 to 15.38)

 Patisiran + TTR tetramer stabilizer

19

−7.03 (2.11)

−6.63 (−34.63 to 3.88)

  1. Abbreviations: BP blood pressure, mNIS+7 modified Neuropathy Impairment Score +7, NCS nerve conduction studies, NIS Neuropathy Impairment Score, QST quantitative sensory testing, SEM standard error of the mean, TTR transthyretin