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Table 2 Summary of findings for the main comparisons

From: Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis

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

  1. *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)
  2. CI: Confidence interval; MD: Mean difference; RR: Risk ratio
  3. GRADE Working Group grades of evidence
  4. High certainty: We are very confident that the true effect lies close to that of the estimate of the effect
  5. 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
  6. Low certainty: Our confidence in the effect estimate is limited: The true effect may be substantially different from the estimate of the effect
  7. 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