Table 2.
Summary of findings for the main comparisons
| Summary of findings: | |||||
|---|---|---|---|---|---|
| Exon skipping compared to placebo for Duchenne muscular dystrophy | |||||
| Patient or population: Duchenne muscular dystrophy | |||||
| Setting: | |||||
| Intervention: Exon skipping | |||||
| Comparison: Placebo | |||||
| OutcomeNo of participants(studies) | Relative effect(95% CI) | Anticipated absolute effects (95% CI) | Certainty | ||
| Without Exon skipping | With Exon skipping | Difference | |||
| 6MWT at week 24 (change from baseline,metres)№ of participants: 291(4 RCTs) | – | The mean 6MWT at week 24 (change from baseline,metres) ranged from −29.11 to −3.6 m | – | MD 9.16 m lower(21.94 lower to 3.62 higher) | ⨁⨁◯◯LOW a,b,c,d |
| NSAA at week24 (change from baseline)№ of participants: 116(3 RCTs) | – | The mean NSAA at week24 (change from baseline) ranged from −0.6 to 3.3 | – | MD 1.2 higher(2.35 lower to 4.75 higher) | ⨁◯◯◯VERY LOW a,b,c,d,e,f,g,h,i,j |
| Adverse events: injection site reaction№ of participants: 322(5 RCTs) | RR 2.54(0.95 to 6.81) | 14.4% | 36.6%(13.6 to 98.2) | 22.2% more(0.7 fewer to 83.8 more) | ⨁◯◯◯VERY LOW a,b,c,d,f,h |
| Adverse events: renal toxicity№ of participants: 322(5 RCTs) | RR 1.72(1.07 to 2.78) | 16.3% | 28.1%(17.5 to 45.4) | 11.8% more(1.1 more to 29.1 more) | ⨁◯◯◯VERY LOW a,b,c,d,i,j |
| 6MWT at week 48 (change from baseline,meters)№ of participants: 226(2 RCTs) | – | The mean 6MWT at week 48 (change from baseline,meters) ranged from −52.65 to −24.7 | – | MD 19.11 lower(39.88 lower to 1.66 higher) | ⨁⨁◯◯LOW a,b,c,d |
| Timed test: 4stair climb at week 24 (change from baseline, seconds)№ of participants: 116(3 RCTs) | – | The mean timed test: 4stair climb at week 24 (change from baseline, seconds) ranged from −1.22 to 0.59 s | – | MD 0.17 s lower(0.97 lower to 0.62 higher) | ⨁◯◯◯VERY LOW a,b,c,d,f |
| QOL at week 48 (PedsQL),patient (change from baseline)№ of participants: 219(2 RCTs) | – | The mean QOL at week 48 (PedsQL),patient (change from baseline) ranged from 0.37 to 0.52 | – | MD 1.53 lower(4.69 lower to 1.63 higher) | ⨁⨁◯◯LOW a,b,c,d |
*The risk in the intervention group (and its 95% confidence interval) is based on the assumed risk in the comparison group and the relative effect of the intervention (and its 95% CI)
CI: Confidence interval; MD: Mean difference; RR: Risk ratio
GRADE Working Group grades of evidence
High certainty: We are very confident that the true effect lies close to that of the estimate of the effect
Moderate certainty: We are moderately confident in the effect estimate: The true effect is likely to be close to the estimate of the effect, but there is a possibility that it is substantially different
Low certainty: Our confidence in the effect estimate is limited: The true effect may be substantially different from the estimate of the effect
Very low certainty: We have very little confidence in the effect estimate: The true effect is likely to be substantially different from the estimate of effect