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Table 3 Estimates of responder definition thresholds for NIS and NIS-LL total and subscale scores

From: The impact of inotersen on Neuropathy Impairment Score in patients with hereditary transthyretin amyloidosis with polyneuropathy

Instrument/Score

Scale Properties

Anchor-based estimates

Distribution-based estimates

Recommended RD thresholda

Reliability

SDbaseline

SDchange

Mean change

Linear regression

ROC

ES

SEM

SRM

NIS

 Total

0.99

25.3

14.6

11.8b

7.0b

6.9b

12.7

3.0

7.3

8.1

 Muscle weakness

0.99

17.0

10.7

–

–

–

8.5

1.9

5.4

5.3

 Sensation

0.92

6.5

4.3

3.3b

2.0b

1.8b

3.3

1.9

2.2

2.4

 Reflex

0.95

6.1

3.5

–

–

–

3.1

1.3

1.7

2.0

NIS-LL

 Total

0.98

15.7

7.8

7.0c

2.0c

5.3c

7.8

2.2

3.9

4.7

 Muscle weakness

0.98

11.2

6.3

5.2c

1.4c

3.8c

5.6

1.4

3.1

3.4

 Sensation

0.91

4.2

2.5

1.8b

1.2b

0.3b

2.1

1.3

1.2

1.3

 Reflex

0.88

2.4

1.6

–

–

–

1.2

0.8

0.8

0.9

  1. Abbreviations: ES effect size, LLF Lower Limb Function Test, NIS Neuropathy Impairment Score, NIS-LL Neuropathy Impairment Score – Lower Limb, PND Polyneuropathy Disability score, RD responder definition, ROC receiver operating characteristic, SD standard deviation, SEM standard error of measurement, SRM standardized response mean
  2. aRecommended RD threshold was calculated as the mean of RD estimates
  3. bRD threshold estimate was calculated using PND as the anchor measure
  4. cRD threshold estimate was calculated using LLF as the anchor measure