Table 1.
Baseline characteristics of the datasets analysed, by multiple sclerosis phenotypes (means and standard deviations; counts and proportions)
| Full dataset (N = 27 328) | Phase 3 trials + extensions (n = 8346) | Phase 3 placebo-controlled trials (n = 4970) | |||||||
|---|---|---|---|---|---|---|---|---|---|
| RRMS n = 24 469 |
SPMS n = 1873 |
PPMS n = 986 |
RRMS n = 5623 |
SPMS n = 1753 |
PPMS n = 970 |
RRMS n = 2355 |
SPMS n = 1645 |
PPMS n = 970 |
|
| Agea (years) [min, max] |
n′ = 24 440 (99.9%) 39.4 ± 10.5 [10, 86] |
n′ = 1873 (100%) 47.6 ± 8.2 [21, 78] |
n′ = 986 (100%) 48.5 ± 8.5 [24, 67] |
n′ = 5623 (100%) 36.9 ± 9.8 [10, 59] |
n′ = 1753 (100%) 47.8 ± 8.0 [21, 63] |
n′ = 970 (100%) 48.4 ± 8.5 [24, 66] |
n′ = 2355 (100%) 38.7 ± 8.9 [16, 59] |
n′ = 1645 (100%) 48.0 ± 8.0 [21, 63] |
n′ = 970 (100%) 48.4 ± 8.5 [24, 66] |
| Females (%) |
n′ = 24 467 (99.99%) 17 490 (71.5%) |
n′ = 1873 (100%) 1123 (60.0%) |
n′ = 986 (100%) 477 (48.4%) |
n′ = 5623 (100%) 3938 (70.0%) |
n′ = 1753 (100%) 1050 (59.9%) |
n′ = 970 (100%) 469 (48.4%) |
n′ = 2355 (100%) 1733 (73.6%) |
n′ = 1645 (100%) 987 (60.0%) |
n′ = 970 (100%) 469 (48.4%) |
| Caucasian (%) |
n′ = 19 218 (78.5%) 16 400 (85.3%) |
n′ = 1860 (99.3%) 1728 (92.9%) |
n′ = 986 (100%) 945 (95.8%) |
n′ = 5607 (99.7%) 5132 (91.5%) |
n′ = 1742 (99.4%) 1661 (95.4%) |
n′ = 970 (100%) 933 (96.2%) |
n′ = 2355 (100%) 2175 (92.4%) |
n′ = 1635 (99.4%) 1559 (95.4%) |
n′ = 970 (100%) 933 (96.2%) |
| Duration of multiple sclerosis since first symptoms, years (%) | n′ = 21 943 (89.7%) | n′ = 1826 (97.5%) | n′ = 984 (99.8%) | n′ = 5622 (99.9%) | n′ = 1750 (99.8%) | n′ = 969 (99.9%) | n′ = 2355 (100%) | n′ = 1642 (99.8%) | n′ = 969 (99.9%) |
| Median categoryb | 5 to <10 | 10 to <30 | 5 to <10 | 5 to <10 | 10 to <30 | 5 to <10 | 5 to <10 | 10 to <30 | 5 to <10 |
| 0 to <2 years | 3213 (14.6%) | 5 (0.3%) | 7 (0.7%) | 1125 (20.0%) | 5 (0.3%) | 5 (0.5%) | 333 (14.1%) | 4 (0.2%) | 5 (0.5%) |
| 2 to <5 years | 4397 (20.0%) | 99 (5.4%) | 387 (39.3%) | 1235 (22.0%) | 94 (5.4%) | 386 (39.8%) | 457 (19.4%) | 90 (5.5%) | 386 (39.8%) |
| 5 to <10 years | 5867 (26.7%) | 334 (18.3%) | 554 (56.3%) | 1484 (26.4%) | 315 (18.0%) | 550 (56.8%) | 682 (29.0%) | 295 (18.0%) | 550 (56.8%) |
| 10 to <30 years | 8033 (36.6%) | 1265 (69.3%) | 36 (3.7%) | 1714 (30.5%) | 1214 (69.4%) | 28 (2.9%) | 843 (35.8%) | 1134 (69.1%) | 28 (2.9%) |
| ≥30 years | 433 (2.0%) | 123 (6.7%) | 0 (0.0%) | 64 (1.1%) | 122 (7.0%) | 0 (0.0%) | 40 (1.7%) | 119 (7.2%) | 0 (0.0%) |
| Previously treated (%) |
n′ = 15 935 (65.1%) 12 343 (77.5%) |
n′ = 1832 (97.8%) 1435 (78.3%) |
n′ = 977 (99.1%) 211 (21.6%) |
n′ = 5622 (99.9%) 3201 (56.9%) |
n′ = 1753 (100%) 1374 (78.4%) |
n′ = 969 (99.9%) 205 (21.2%) |
n′ = 2354 (99.9%) 1330 (56.5%) |
n′ = 1645 (100%) 1289 (78.4%) |
n′ = 969 (99.9%) 205 (21.2%) |
| Number of relapses in previous year |
n′ = 22 040 (90.1%) 1.2 ± 1.0 |
n′ = 1827 (97.5%) 0.3 ± 0.6 |
n′ = 983 (99.7%) 0.0 ± 0.1 |
n′ = 5623 (100%) 1.4 ± 0.8 |
n′ = 1751 (99.9%) 0.3 ± 0.6 |
n′ = 970 (100%) 0.0 ± 0.0 |
n′ = 2355 (100%) 1.5 ± 0.8 |
n′ = 1643 (99.9%) 0.3 ± 0.5 |
n′ = 970 (100%) 0.0 ± 0.0 |
| EDSS at baseline |
n′ = 20 535 (83.9%) 2.7 ± 1.6 |
n′ = 1852 (98.9%) 5.4 ± 1.1 |
n′ = 976 (99.0%) 4.7 ± 1.0 |
n′ = 5621 (99.9%) 2.5 ± 1.3 |
n′ = 1753 (100%) 5.4 ± 1.1 |
n′ = 970 (100%) 4.7 ± 1.0 |
n = 2355 (100%) 2.4 ± 1.3 |
n′ = 1645 (100%) 5.4 ± 1.1 |
n′ = 970 (100%) 4.7 ± 1.0 |
| Patients with Gd-enhancing lesions (%) |
n′ = 10 227 (41.8%) 3884 (38.0%) |
n′ = 1732 (92.5%) 391 (22.6%) |
n′ = 967 (98.1%) 124 (12.8%) |
n′ = 5578 (99.2%) 2122 (38.0%) |
n′ = 1701 (97.0%) 378 (22.2%) |
n′ = 967 (99.7%) 124 (12.8%) |
n′ = 2342 (99.4%) 863 (36.8%) |
n′ = 1593 (96.8%) 350 (22.0%) |
n′ = 967 (99.7%) 124 (12.8%) |
| T2 lesion volume (mm3) at baselinec |
n′ = 6178 (25.2%) 8375 ± 10 764 |
n′ = 1738 (92.8%) 15 717 ± 16 227 |
n′ = 967 (98.1%) 9764 ± 12 014 |
n′ = 5580 (99.2%) 8214 ± 10 545 |
n′ = 1707 (97.4%) 15 654 ± 16 238 |
n′ = 967 (99.7%) 9764 ± 12 014 |
n′ = 2342 (99.4%) 5890 ± 7752 |
n′ = 1599 (97.2%) 15 298 ± 16 010 |
n′ = 967 (99.7%) 9764 ± 12 014 |
n refers to the analysis set totals; n refers to the total number of subjects in the specific column; n′ (%) refers to the number and proportion of patients with the specific baseline feature evaluated; and the numbers in bold refer to the mean ± standard deviation or the number (proportion) of patients with the specific characteristic out of n′.
To preserve anonymization of the data, the age of patients has been randomly jittered, thus the age range may not be fully aligned with the inclusion criteria of individual studies. The phase 3 RRMS studies included adult RRMS patients per protocol inclusion criteria. Baseline characteristics of the paediatric phase 3 study (PARADIGMS) have previously been reported.27
To preserve anonymization of the data, exact duration of multiple sclerosis has been categorized.
MRI methods differ between different trials and MRI reading centers, which may introduce systematic biases for between-phenotype comparisons22; cross-phenotype comparisons should be done cautiously.