Table 1.
Baseline characteristics of the cohort
| Whole cohort (N = 75) | No need for tube feeding (N = 34) | Tube feeding and oral (N = 9) | Tube feeding without oral (N = 32) | |
|---|---|---|---|---|
| Age | ||||
| Mean (SD) | 1.34 (1.19) | 1.18 (0.98) | 1.08(1.14) | 1.58 (1.39) |
| Median [range] | 0.81 [0.06–4.98] | 0.83 [0.06–4.98] | 0.63 [0.23–3.73] | 0.92 [0.24–4.78] |
| Gender | ||||
| F, n (%) | 38 (51) | 16 (47) | 5 (56) | 17 (53) |
| M, n (%) | 37 (49) | 18 (53) | 4 (44) | 15 (47) |
| SMN2 copies | ||||
| 1, n (%) | 1 (1) | 0 (0) | 0 (0) | 1 (3) |
| 2, n (%) | 63 (84) | 27 (79) | 8 (89) | 28 (88) |
| 3, n (%) | 11 (15) | 7 (21) | 1 (11) | 3 (9) |
| SMA subtype | ||||
| 1.1, n (%) | 13 (17) | 4 (12) | 2 (22) | 7 (22) |
| 1.5, n (%) | 41 (55) | 16 (47) | 6 (67) | 19 (59) |
| 1.9, n (%) | 21 (28) | 14 (41) | 1 (11) | 6 (19) |
| Tube feeding | ||||
| Yes, n (%) | 30 (40) | 0 (0) | 4 (44) | 26 (81) |
| No, n (%) | 45 (60) | 34 (100) | 5 (56) | 6 (19) |
| Tracheostomy | ||||
| Yes, n (%) | 16 (21) | 0 (0) | 0 (0) | 16 (50) |
| No, n (%) | 59 (79) | 34 (100) | 9 (100) | 16 (50) |
| Treatment | ||||
| Nusinersen, n (%) | 69 (92) | 29 (85) | 9 (100) | 31 (97) |
| OA, n (%) | 6 (8) | 5 (15) | 0 (0) | 1 (3) |
| Risdiplam, n (%) | 0 (0) | 0 (0) | 0 (0) | 0 (0) |
| Switch | ||||
| Nus. + OA, n (%) | 18 (24) | 14 (41) | 2 (22) | 2 (6) |
| Nus. + Risd, n (%) | 5 (7) | 0 (0) | 0 (0) | 5 (16) |