Table 1.
Demographic and clinical characteristics of the treatment groups.
| Parameter | All patients (n = 1,654) | IFNβ-1a 44 mcg (n = 467) | GA (n = 509) | IFNβ-1b (n = 199) | IFNβ-1a 30 mcg (n = 384) | Teriflunomide (n = 88) | Peg-IFNβ-1a (n = 9) | Statistical simultaneous comparison (except for peg-IFNβ-1a) | ||
|---|---|---|---|---|---|---|---|---|---|---|
| Test | P-value (corr.) | Effect size | ||||||||
| Age | 34.42 (9.79) | 32.832 (9.43) | 33.7981 (9.85) | 36.7914 (9.21) | 34.474 (10.05) | 41.12 (7.88) | 31.029 (7.07) | * | <0.001 | 0.04 |
| Disease duration (in months) | 5.323 (4.12) | 4.665 (3.55) | 5.5346 (4.39) | 5.9271 (4.01) | 5.196 (4.02) | 6.49 (4.96) | 9.059 (7.58) | ** | <0.001 | 0.02 |
| DMD treatment duration (in months) | 18.508 (7.12) | 18.528 (7.28) | 17.0362 (7.41) | 18.3597 (7.29) | 20.134 (6.21) | 20.51 (6.13) | 14.094 (4.99) | *** | <0.001 | 0.03 |
| EDSS score at baseline (0 M) | 1.853 (0.89) | 2.11 (0.98) | 1.78 (0.83) | 2.0151 (0.91) | 1.529 (0.71) | 1.97 (0.88) | 1.857 (0.8) | *** | <0.001 | 0.06 |
| EDSS score in the first year (12 M) | 1.886 (0.99) | 2.164 (1.08) | 1.7546 (0.91) | 2.018 (0.93) | 1.604 (0.89) | 2.08 (0.99) | 2 (1.15) | *** | <0.001 | 0.05 |
| EDSS score in the second year (24 M) | 1.98 (1.08) | 2.231 (1.18) | 1.8546 (1.02) | 2.1332 (1.08) | 1.716 (0.96) | 2.17 (1.01) | 2 (1.15) | *** | <0.001 | 0.04 |
| Absolute change in EDSS score after 1 year | 0.034 (0.61) | 0.047 (0.62) | −0.0092 (0.59) | 0.0052 (0.57) | 0.071 (0.66) | 0.11 (0.43) | 0.143 (0.38) | * | 0.295 | <0.01 |
| Absolute change in EDSS score after 2 years | 0.128 (0.72) | 0.121 (0.74) | 0.0747 (0.7) | 0.1181 (0.78) | 0.188 (0.74) | 0.24 (0.6) | 0.143 (0.38) | * | 0.924 | <0.01 |
| Time to CDP (in months) | 10.642 (4.84) | 11.96 (4.92) | 9.859 (5.55) | 10.2418 (3.39) | 10.331 (4.55) | 8.44 (4.4) | NA | ** | 0.675 | 0.03 |
| Number of relapses | 0.584 (0.96) | 0.707 (1.01) | 0.5069 (0.94) | 0.5327 (0.89) | 0.591 (1) | 0.5 (0.87) | 0.143 (0.38) | *** | 0.009 | 0.01 |
| ARR | 0.282 (0.46) | 0.341 (0.48) | 0.2453 (0.46) | 0.2579 (0.44) | 0.284 (0.47) | 0.24 (0.42) | 0.069 (0.18) | *** | 0.008 | 0.01 |
| Time to next relapse (in months) | 10.291 (7.12) | 9.895 (6.92) | 10.2243 (7.42) | 11.3047 (7.3) | 10.695 (7.18) | 9.03 (6.22) | 9.738 (NA) | *** | 0.663 | 0.01 |
| Follow-up duration (in months) | 24.671 (1.54) | 24.684 (1.49) | 24.6947 (1.56) | 24.7055 (1.53) | 24.63 (1.54) | 24.66 (1.73) | 23.415 (1.86) | *** | 0.939 | <0.01 |
| Gender | **** | 0.002 | 0.131 | |||||||
| Females | 1157 (69.95) | 312 (66.81) | 399 (78.39) | 125 (62.81) | 265 (69.01) | 53 (60.23) | 3 (42.86) | |||
| Males | 497 (30.05) | 155 (33.19) | 110 (21.61) | 74 (37.19) | 119 (30.99) | 35 (39.77) | 4 (57.14) | |||
| CDP | **** | 0.406 | 0.054 | |||||||
| No | 1566 (94.68) | 437 (93.58) | 489 (96.07) | 191 (95.98) | 360 (93.75) | 82 (93.18) | 7 (100) | |||
| Yes | 88 (5.32) | 30 (6.42) | 20 (3.93) | 8 (4.02) | 24 (6.25) | 6 (6.82) | 0 (0) | |||
| Relapse status during follow-up | **** | 0.034 | 0.089 | |||||||
| Relapse occurred | 603 (36.46) | 198 (42.4) | 165 (32.42) | 68 (34.17) | 142 (36.98) | 29 (32.95) | 1 (14.29) | |||
| Relapse free | 1051 (63.54) | 269 (57.6) | 344 (67.58) | 131 (65.83) | 242 (63.02) | 59 (67.05) | 6 (85.71) | |||
| Reason for treatment discontinuation | **** | 0.002 | 0.13 | |||||||
| Adverse Event | 80 (9.38) | 33 (12.64) | 13 (4.64) | 9 (8.49) | 23 (12.37) | 2 (10.53) | 0 | |||
| Lack of efficacy | 350 (41.03) | 122 (46.74) | 78 (27.86) | 55 (51.89) | 82 (44.09) | 13 (68.42) | 0 | |||
| Lack of tolerance | 313 (36.69) | 81 (31.03) | 153 (54.64) | 25 (23.58) | 52 (27.96) | 2 (10.53) | 0 | |||
| Pregnancy | 110 (12.9) | 25 (9.58) | 36 (12.86) | 17 (16.04) | 29 (15.59) | 2 (10.53) | 1 (100) | |||
| no info | 801 | 206 | 229 | 93 | 198 | 69 | 6 | |||
Numerical variables are expressed as the means (standard deviations), and categorical variables (gender, CDP, relapse-free status, reasons for treatment discontinuation) are expressed as frequency (percentage). The DMD groups were compared using parametric (*, ** after logarithmic transformation) or nonparametric ANOVA (***). Categorical variables were tested using the chi-squared test of independence. Reported P-values [(P value (corr.)] are corrected for multiple testing by Benjamini-Hochberg procedure.