11. Oral valproic acid versus placebo outcomes (Kissel 2014).
| Valproic acid | Placebo | Difference (95% CI)a | P value | |
| Number (%) of participants randomised first 6 months (cross‐over setting) | 16 (100%) | 17 (100%) | — | — |
| Number (%) of participants evaluable for analysis after 12 months | 30 (91%) | 30 (91%) | — | — |
| Analysis after 6 months before cross‐over | ||||
| Number (%) of participants evaluable for analysis SMAFRS | 14 (88%) | 17 (100%) | — | — |
| Mean (SD) change in SMAFRS | ‐0.29 (1.59) | ‐0.35 (2.32) | 0.06 (‐1.32 to 1.44) | 0.93 |
| Number (%) of participants evaluable for analysis upper extremity | 14 | 16 | — | — |
| Mean (SD) change in MVICT upper extremity (Newton) | ‐0.24 (1.17) | ‐0.01 (1.05) | ‐0.23 (‐1.03 to 0.57) | 0.54 |
| Number (%) of participants evaluable for analysis lower extremity | 13 | 16 | — | — |
| Mean (SD) change in lower extremity MVICT (Newton) | ‐0.02 (0.65) | 0.35 (1.30) | ‐0.37 (‐1.09 to 0.35) | 0.36 |
| Number (%) of participants evaluable for analysis pulmonary function (FVC) | 14 (88%) | 17 (100%) | — | — |
| Mean (SD; range) change in pulmonary function (FVC improvement in litres) | ‐0.04 (0.35) | 0.30 (1.20) | ‐0.34 (‐0.94 to 0.26) | 0.31 |
| Number of participants evaluable for QoL analysis | 14 | 17 | — | — |
| Mean (SD) change in mini‐SIP for QoL | ‐0.19 (2.80) | 0.91 (4.78) | ‐1.1 (‐3.8 to 1.6) | 0.53 |
| Valproic acid | Placebo | Risk ratio (95% CI)a | P value | |
| Number of adverse events | 30 | 66 | — | — |
| Number of participants with adverse events | 12 | 15 | 0.8 (0.44 to 1.44) | |
| Number of severe adverse events | 2 | 3 | — | — |
| Number of participants with severe adverse events | 2 | 2 | 1.0 (0.15 to 6.69) | — |
| Number (%) of participants discontinued intervention | NA | NA | — | — |
CI: confidence interval; FVC: forced vital capacity; mini‐SIP: mini‐Sickness Illness Profile; MVICT: maximum voluntary isometric contraction; NA: not available; QoL: quality of life; SD: standard deviation; SMAFRS: Spinal Muscular Atrophy Functional Rating Scale.
aCalculated with available data.