Table 3.
Predicted progression from transition probabilities | Actual progression observed with treatment | Difference (actual less predicted) | Deviation score (%) | ||
---|---|---|---|---|---|
Without treatment | With treatment | ||||
Per protocol analysis (n=3686) | |||||
EDSS | 0.47 | 0.29 | 0.57 (0.544 to 0.603) | 0.28 | — |
Utility score | 0.0254 | 0.0158 | 0.0266 | — | 113 |
Including DMT switchers (n=3931) | |||||
EDSS | 0.48 | 0.30 | 0.59 (0.559 to 0.617) | 0.28 | — |
Utility score | 0.0258 | 0.0162 | 0.0271 | — | 113 |
Including SPMS converters on DMTs (n=3690) | |||||
EDSS | 0.48 | 0.30 | 0.58 (0.550 to 0.610) | 0.27 | — |
Utility score | 0.0261 | 0.0164 | 0.0275 | — | 114 |
Excluding extreme outliers (n=3650) | |||||
EDSS | 0.47 | 0.30 | 0.54 (0.514 to 0.570) | 0.25 | — |
Utility score | 0.0254 | 0.0158 | 0.0250 | — | 96 |
Subgroup analysis with year 2 confirmed scores (n=1479) | |||||
EDSS | 0.50 | 0.32 | 0.46 (0.422 to 0.507) | 0.14 | — |
Utility score | 0.0263 | 0.0162 | 0.0214 | — | 51 |
Predicted change in unconfirmed year 2 scores (n=3686) | |||||
EDSS | 0.0.47 | 0.29 | 0.48* | 0.19 | — |
Utility score | 0.0254 | 0.0158 | 0.0225 | — | 70 |
Imputing missing year 2 scores—best case scenario (n=3686) | |||||
EDSS | 0.53 | 0.33 | 0.60 (0.566 to 0.627) | 0.27 | — |
Utility score | 0.0290 | 0.0179 | 0.0285 | — | 95 |
Imputing missing year 2 scores—worst case scenario (n=3686) | |||||
EDSS | 0.53 | 0.33 | 0.66 (0.629 to 0.699) | 0.33 | — |
Utility score | 0.0290 | 0.0179 | 0.0351 | — | 156 |
Unadjusted baseline scores (n=3686) | |||||
EDSS | 0.49 | 0.31 | 0.20 (0.163 to 0.241) | −0.11 | — |
Utility score | 0.0257 | 0.0157 | 0.0073 | — | −84 |
DMTs=disease modifying treatments; EDSS=expanded disability status scale.
*95% confidence intervals cannot be calculated by standard methods because of indirect method used to estimate mean EDSS change.