Table 2.
12-month CHOP-INTEND results on the sample of 46 patients of Wilcoxon test and Kruskal–Wallis-test/Mann–Whitney test according to the subgroups of stratification.
| Stratification subgroups | Time | Total (N) | Median (IQR) | Mean (SD) | P-value |
Median change |
Mean change |
P-value |
|---|---|---|---|---|---|---|---|---|
| Wilcoxon | T12–T0 (IQR) | T12–T0 (SD) | Kruskal–Wallis | |||||
| Age at OA treatment | ||||||||
| <6 months | T0 | 15 | 30 (20) | 34.4 (14.5) | P = 0.001b | 17 (12) | 16.7 (7.56) | Chi square = 15.13. P = 0.0005b, df = 2 |
| T12 | 15 | 51 (19) | 51.1 (10.8) | |||||
| 7–24 months | T0 | 12 | 42.5 (5.75) | 42.1 (8.27) | P = 0.003b | 6 (12) | 9.75 (8.4) | |
| T12 | 12 | 52.5 (9.5) | 51.8 (7.07) | |||||
| >24 months | T0 | 19 | 50 (8.5) | 47.1 (10.3) | P < 0.001b | 3 (6.5) | 5.11 (5.20) | |
| T12 | 19 | 53 (8.0) | 52.2 (9.15) | |||||
| Sex | ||||||||
| Female | T0 | 14 | 43.5 (22.8) | 41.2 (14.5) | P = 0.001b | 8.5 (813.8) | 10.8 (9.23) | Chi square = 0.16,529. P = 0.68, df = 1 |
| T12 | 14 | 53 (8.5) | 52 (7.81) | |||||
| Male | T0 | 32 | 43.5 (13.2) | 41.8 (11.7) | P < 0.001b | 8.5 (14) | 9.78 (8.16) | |
| T12 | 32 | 52 (11.5) | 51.6 (9.69) | |||||
| Weight | ||||||||
| <8500 gr | T0 | 20 | 36.5 (20) | 36.7 (13.4) | P = 0.001b | 17 (13.2) | 15 (8.48) | Chi square = 11.31 P < 0.001b, df = 1 |
| T12 | 20 | 51.5 (14.2) | 51.8 (9.64) | |||||
| >8500 gr | T0 | 26 | 47 (10.8) | 45.5 (10.4) | P < 0.001b | 4 (6.75) | 6.27 (6.16) | |
| T12 | 26 | 53.5 (10.2) | 51.7 (8.81) | |||||
| SMN2 copy number | ||||||||
| 2 | T0 | 42 | 42.5 (19) | 40.4 (12.0) | P < 0.001b | 8.5 (14.8) | 10.3 (8.44) | N/A |
| T12 | 42 | 52.0 (13.2) | 50.8 (8.83) | |||||
| 3 | T0 | 4 | 55.5 (18) | 54.5 (11.1) | N/A | 6.5 (14) | 7.5 (8.81) | |
| T12 | 4 | 64 (2.0) | 62.0 (4.0) | |||||
| SMA 1 severity of the disease | ||||||||
| 1.1 | T0 | 4 | 30 (13) | 29.5 (8.19) | N/A | 14 (9.5) | 15.5 (6.81) | Chi square = 6.31 P = 0.04b, df = 2 |
| T12 | 4 | 44.5 (11) | 45 (12.0) | |||||
| 1.5 | T0 | 32 | 43 (19) | 39.8 (11.7) | P < 0.001b | 10.5 (15.2) | 11.3 (8.56) | |
| T12 | 32 | 52 (10.2) | 51.1 (8.31) | |||||
| 1.9 | T0 | 8 | 51 (8) | 50 (8.18) | P = 0.035b | 3 (4.5) | 4.62 (5.78) | |
| T12 | 8 | 54 (14) | 54.6 (9.02) | |||||
| Pre-treatment with nusinersena | ||||||||
| No | T0 | 7 | 30 (9.5) | 30.4 (9.03) | P = 0.022b | 17 (9.5) | 16.6 (6.32) | Chi square = 0.030, P = 0.8617, df = 1 |
| T12 | 7 | 47 (9.5) | 47 (10.3) | |||||
| Yes | T0 | 8 | 38.5 (25.8) | 37.9 (18.0) | P = 0.022b | 19.5 (11.2) | 16.8 (8.94) | |
| T12 | 8 | 59 (14.0) | 54.6 (10.6) |
a
Computed only in patients <6 months at OA treatment.
b
Reached statistical significance (P < 0.05).