Table 2.
Estimates for LoA by onset group, and for loss of function of FARS E Items (for onset <15y). Items are arranged by median duration, including to the analysis of diagnosis of FRDA.
Onset subgroup | Item | Description | Median duration [y] | 25, 75 percentiles | N | Observed events | Left censored [%] | Truncation biasa [y] |
---|---|---|---|---|---|---|---|---|
Loss of ambulation, stratified by onset group | ||||||||
<15y | E7 | LoA/gait | 11.5 | 8.6, 16.2 | 703 | 148 | 33 | 2.9 |
15–24y | E7 | LoA/gait | 18.3 | 14.1, 27.5 | 211 | 45 | 25 | 7.0 |
>24y | E7 | LoA/gait | 23.5 | 18.0, 30.1 | 107 | 21 | 17 | 4.7 |
Loss of stance functions in the FARS E sub score, early onset group (<15y) | ||||||||
<15y | E5 | Stance dominant foot | 0.2 | 0.2, 0.2 | 703 | 14 | 97 | 7.3 |
<15y | E4 | Stance, tandem | 0.9 | 0.2, 2.0 | 703 | 46 | 92 | 4.8 |
<15y | E3B | Stance feet together, eyes closed |
1.2 | 0.5, 1.5 | 703 | 40 | 93 | 2.9 |
<15y | – | Diagnosis of FRDA | 3.0 | 1.0, 5.0 | 703 | 688 | 2 | – |
<15y | E2B | Stance feet apart, eyes closed |
4.1 | 2.5, 6.1 | 703 | 151 | 68 | 2.2 |
<15y | E3A | Stance, feet together | 5.8 | 3.6, 8.7 | 703 | 174 | 58 | 2.4 |
<15y | E2A | Stance, feet apart | 9.3 | 6.7, 12.2 | 703 | 180 | 39 | 1.5 |
<15y | FDS | Disease stage | 11.1 | 8.7, 15.6 | 703 | 149 | 33 | 3.3 |
<15y | E7 | LoA/gait | 11.5 | 8.6, 16.2 | 703 | 148 | 33 | 2.9 |
Difference between analyses all item-results were floored, e.g. 2.33 or 2.66 were set to 2; the highest number indicates maximum disability (‘unable’).