10. Oral valproic acid plus acetylcarnitine versus placebo outcomes (Swoboda 2010).
| Valproic acid + acetyl‐L‐carnitine | Placebo | Difference (95% CI) | P value | |
| All ages | ||||
| Number of participants randomised | 31 | 30 | — | — |
| Number (%) of participants evaluable for analysis disability (MHFMS) | 28 (90%) | 28 (93%) | — | — |
| Mean change (SD) in disability score (MHFSM) at 6 months | 0.82 (2.88) | 0.18 (3.98) | 0.64 (‐1.1 to 2.38) | 0.50 |
| Number (%) of participants evaluable for analysis of nerve innervation value (CMAP) | 19 (61%) | 19 (63%) | — | — |
| Mean (SD) change in total amplitude CMAP from baseline to 6 months | 0.02 (0.70) | ‐0.10 (0.66) | 0.12 (‐0.33 to 0.57) | 0.59 |
| Number (%) of participants evaluable for analysis of quality of life (PedsQL) from baseline to 12 months | 27 (87%) | 27 (90%) | — | — |
| Mean (SD) change in quality of life (PedsQL) from baseline to 12 months | ‐1.9 (13.6) | 0.3 (12.9) | ‐2.2 (‐9.27 to 4.87) | 0.54 |
| Age < 3 years old | ||||
| Number (%) of participants evaluable for analysis disability (MHFMS) | 12 (52%) | 11 (48%) | — | — |
| Mean change (SD) in disability score (MHFSM) from baseline to 6 months | 1.33 (2.27) | 1.09 (5.37) | 0.24 (‐3.28 to 3.76) | 0.89 |
| Aged 3–8 years | ||||
| Number (%) of participants evaluable for analysis disability (MHFSM) | 18 (47%) | 17 (45%) | — | — |
| Mean change (SD) in disability score (MHFSM) from baseline to 6 months | 0.44 (3.29) | ‐0.41 (2.79) | 0.85 (‐1.25 to 2.95) | 0.42 |
| Aged ≥ 5 years | ||||
| Number of participants evaluable for analysis muscle strength in arms (myometry) | 7 | 7 | — | — |
| Mean (SD) change in arm muscle strength (myometry) from baseline to 6 months (kg) | 0.64 (0.6) | 0.07 (1.04) | 0.57 (‐0.45 to 1.58) | 0.23a |
| Number of participants evaluable for analysis muscle strength in legs (myometry) | 6 | 4 | — | — |
| Mean (SD) change in leg muscle strength (myometry) from baseline to 6 months (kg) | 0.55 (0.83) | ‐0.85 (2.22) | 1.40 (‐1.98 to 4.79) | 0.19a |
| Number of participants evaluable for analysis muscle strength in both arms and legs (myometry) | 7 | 8 | — | — |
| Mean (SD) change in total muscle strength (myometry) from baseline to 6 months (kg) | 1.18 (0.91) | ‐0.25 (2.47) | 1.43 (0.69 to 3.56) | 0.21 |
| Number of participants evaluable for analysis pulmonary function (FVC in % of predicted) | NA | NA | NAa | NAa |
| Mean (SD) change in pulmonary function (FVC in % of predicted) from baseline to 6 months | NA | NA | — | — |
| Valproic acid + acetyl‐L‐carnitine | Placebo | Risk ratio (95% CI) | P value | |
| All ages | ||||
| Number (%) of participants with adverse events (6 months) | 23 (77%) | 18 (58%) | 1.32 (0.92 to 1.89) | — |
| Number (%) of participants with severe adverse events (6 months) | 6 (20%) | 2 (6%) | 3.1 (0.60 to 12.1) | — |
CI: confidence interval; CMAP: compound maximum action potential; FVC: forced vital capacity; MHFMS: Modified Hammersmith Functional Motor Scale; PedsQL: Pediatric Quality of Life Inventory; SD: standard deviation.
a Underpowered.