1. Scores included as outcome measures.
| Instrument name | Abbreviation | Score (range) | Direction of response | References | Data analysed as |
| Stage of disease based on disability or disability | |||||
| Clinical staging of TTR‐FAP | FAP stage | 1 to 3 | Higher scores indicate greater disease severity | Coutinho 1980 | Ordinal |
| Polyneuropathy Disability Score | PDS | 0 to 5 | Higher scores indicate greater walking disability | Steen 1983 | Ordinal |
| Modified Norris Test Score | MNT | 75 to 0 | Lower scores indicate greater disability | Lacomblez 1989 | Continuous |
| Portuguese classification system | PCS | 0 to 6 | Higher scores indicate greater disease severity | Sales‐Luís 1990 | Ordinal |
| Kumamoto Score | KS | 0 to 96 | Higher scores indicate greater disease severity | Tashima 1999 | Continuous |
| Yamamoto Score | YS | 0 to 4 | Higher scores indicate greater disease severity | Yamamoto 2007 | Ordinal |
| Rasch‐built Overall Disability Scale | R‐ODS | 0 to 48 | Lower scores indicate greater disability | van Nes 2011 | Continuous |
| Impairment due to nerve function | |||||
| Neuropathy Impairment Score | NIS | 0 to 244 | Higher scores indicate greater deficits | Dyck 1995 | Continuous |
| Neuropathy Impairment Score in the lower limbs | NIS‐LL | 0 to 88 | Higher scores indicate greater deficits | Bril 1999; Dyck 1997 | Continuous |
| Neuropathy Impairment Score in the upper limbs | NIS‐UL | 0 to 116 | Higher scores indicate greater deficits | Lozeron 2013 | Continuous |
| Neuropathy Impairment Score plus 7 nerve tests | NIS+7 | 0 to 270 | Higher scores indicate greater deficits | Berk 2013 | Continuous |
| Modified Neuropathy Impairment Score plus 7 nerve tests (Alnylam version) | mNIS+7Alnylam | 0 to 304 | Higher scores indicate greater deficits | Suanprasert 2014; Adams 2018 | Continuous |
| Modified Neuropathy Impairment Score plus 7 nerve tests (Ionis version) | mNIS+7Ionis | ‐22.3 to 346.3 | Higher scores indicate greater deficits | Suanprasert 2014; Dyck 2017; Benson 2018 | Continuous |
| Charcot‐Marie Tooth Neuropathy Score | CMTNS | 0 to 36 | Higher scores indicate greater deficits | Shy 2005 | Continuous |
| Charcot‐Marie Tooth Neuropathy Score 2nd version | CMTNS2 | 0 to 36 | Higher scores indicate greater deficits | Murphy 2011 | Continuous |
| Neuropathy Disability Score revised version | rNDS | 0 to 10 | Higher scores indicate greater deficits | Abbott 2002 | Continuous |
| Compound Autonomic Dysfunction Test | CADT | 0 to 16 | Higher scores indicate greater autonomic impairment | Denier 2007 | Continuous |
| Wasting and autonomic gastrointestinal function | |||||
| Modified body mass index | mBMI | Product of the BMI (kg/m2) and serum albumin concentration (g/L) |
Lower scores indicate greater wasting (worse nutritional status) | Suhr 1984 | Count |
| Quality of life | |||||
| Short Form 36 Health Survey Questionnaire | SF‐36 | 0 to 100 | Lower scores indicate worse status |
Ware 1992 | Continuous |
| Norfolk Quality of Life–Diabetic Neuropathy Questionnairea | Norfolk QOL‐DN | ‐2 to 138 ‐4 to 136 |
Higher scores indicate worse status |
Vinik 2005 Adams 2018 Benson 2018 |
Continuous |
| EuroQoL Quality of Life Scale | EQ‐5D | 5 to 15 | Higher scores indicate worse status |
Rabin 2001 | Continuous |
| Karnofsky Performance Status | Karnofsky | 0 to 100 | Lower scores indicate worse status |
Yates 1980 | Continuous |
| Depression | |||||
| Beck Depression Inventory 2nd version | BDI‐II | 0 to 63 | Higher total scores indicate more severe depressive symptoms | Beck 1988 | Continuous |
| Hamilton Depression Rating Scale for Depression | HAM‐D | 0 to 50 | Higher total scores indicate more severe depressive symptoms | Hamilton 1960 | Continuous |
a Slightly different score ranges are reported in different RCTs (range (‐2 to 138) in Coelho 2012 and range (‐4 to 136) in Adams 2018 and Benson 2018), although the direction of the scores is the same.