5. Intrathecal injected nusinersen versus placebo outcomes (Mercuri 2018).
| Nusinersen | Placebo | Difference (95% CI) | P value | |
| Number of participants randomised | 84 | 42 | — | — |
| Interim analysisa | 84b | 42b | — | — |
| Mean change in HFMSE | 4.0 | ‐1.9 | 5.9 (3.7 to 8.1) | < 0.001 |
| Final analysis | 84c | 42c | — | — |
| Mean change in HFMSE | 3.9 | ‐1.0 | 4.9 (3.1 to 6.7) | — |
| Nusinersen | Placebo | Difference (95% CI) | P value | |
| Percentage of participants with 3‐point‐change on HFMSE (%) | 57 | 26 | 30.5 (12.7 to 48.3) | < 0.001 |
| Percentage (number) of children who achieved ≥ 1 new WHO motor milestone (% (number)) | 20 (13) | 6 (2) | 14 (‐7 to 34) | 0.08 |
| Nusinersen | Placebo | Risk ratio (95% CI) | P value | |
| Percentage (number) of children who achieved ability to stand alone | 2 (1) | 3 (1) | 0.5 (0.03 to 7.80) | — |
| Percentage (number) of children who achieved ability to walk with assistance | 2 (1) | 0 (0) | 1.5 (0.06 to 36.1) | — |
| Number of participants with adverse events | 78 | 42 | 0.9 (0.9 to 1.0) | 0.01 |
CI: confidence interval; HFMSE: Hammersmith Functional Motor Scale Expanded; WHO: World Health Organization.
a Conducted in children who completed at least six months of the trial. Data of children who had not yet completed the 15‐month period were imputed. b Included 35 participants in the nusinersen group and 19 participants in the control group who completed the 15‐month assessment. Data for 49 participants in the nusinersen group and 23 participants in the control group were imputed. c The number of children with observed data for the 15‐month assessment was 66 in the nusinersen group and 34 in the control group, and the number of children with imputed data was 18 in the nusinersen group and eight in the control group.