Table 4.
Baseline patient characteristics for the NPP subgroup analysis.
| Masitinib-naïve PBO (N = 53) | NPP-M4.5 (N = 29) | Deltab (%) | NPP-M(pooled) (N = 59) | Deltab (%) | NPP-ALL (N = 84) | Deltab (%) | |
|---|---|---|---|---|---|---|---|
| Sex; n (%) | |||||||
| Male | 33 (62.3) | 20 (69.0) | +6.7 | 39 (66.1) | +3.8 | 55 (65.5) | +3.2 |
| ΔFS < 1.1; n (%) | |||||||
| Yes | 48 (90.6) | 28 (96.6) | +6.0 | 57 (96.6) | +6.0 | 80 (95.2) | +4.6 |
| Average ΔFS (points/month) | |||||||
| Mean ± SD | 0.62 ± 0.7 | 0.5 ± 0.5 | −19.4 | 0.46 ± 0.39 | −25.8 | 0.46 ± 0.38 | −25.8 |
| ALSFRS-R score | |||||||
| Mean ± SD | 38.5 ± 5.1 | 40.4 ± 5.6 | +4.9 | 40.1 ± 4.8 | +4.2 | 40.2 ± 4.8 | +4.4 |
| Age (years) | |||||||
| Mean ± SD | 54.0 ± 10.8 | 50.0 ± 9.7 | −7.4 | 50.7 ± 9 .7 | −6.1 | 50.9 ± 10.0 | −5.7 |
| ALS diagnosis; n (%) | |||||||
| Definite | 34 (64.2) | 13 (44.8) | −19.4 | 29 (49.2) | −15.0 | 36 (42.9) | −21.3 |
| Probable | 12 (22.6) | 14 (48.3) | +25.7 | 22 (37.3) | +14.7 | 38 (45.2) | +22.6 |
| Probable, lab | 7 (13.2) | 2 (6.9) | −6.3 | 8 (13.6) | +0.4 | 10 (11.9) | −1.3 |
| Disease durationa | |||||||
| Mean ± SD | 9.5 ± 7.1 | 10.0 ± 10.0 | +5.3 | 11.2 ± 9.3 | +17.9 | 10.7 ± 8.7 | +12.6 |
| FVC (% predicted) | |||||||
| Mean ± SD | 91.7 ± 20.6 | 93.9 ± 15.9 | +2.4 | 91.4 ± 15.7 | −0.3 | 92.7 ± 15.4 | +1.1 |
| Site of onset; n (%) | |||||||
| Bulbar | 6 (11.3) | 6 (20.7) | +9.4 | 10 (16.9) | +5.6 | 14 (16.7) | +5.4 |
| Region; n (%) | |||||||
| Other countries | 21 (39.6) | 10 (34.5) | −5.1 | 23 (39.0) | −0.6 | 25 (29.8) | −9.8 |
| Eastern Europe | 6 (11.3) | 2 (6.9) | −4.4 | 2 (3.4) | −7.9 | 2 (2.4) | −8.9 |
| North America/Western Europe | 26 (49.1) | 17 (58.6) | +9.5 | 34 (57.6) | +8.5 | 57 (67.9) | +18.8 |
Average disease duration from time of diagnosis.
Delta, relative difference between treatment arms (%) with respect to masitinib-naïve PBO.
ALSFRS-R, Amyotrophic Lateral Sclerosis Functional Rating Scale-revised; FVC, forced vital capacity; masitinib-naïve PBO, cohort from AB10015 placebo arm (regardless of baseline ΔFS or baseline ALSFRS-R scores) who were alive at 1 November 2017 and did not enter the NPP; NPP M4.5, cohort from study AB10015 M4.5 treatment arm (regardless of baseline ΔFS or baseline ALSFRS-R scores) who continued receiving masitinib as part of NPP; NPP, Named Patient Program; SD, standard deviation; ΔFS, ALSFRS-R progression rate calculated from disease onset to baseline.