Cochrane · systematic review · CD006281
Drug treatment for spinal muscular atrophy type I
Wadman, RI, van der Pol, WL, Bosboom, WMJ, et al. · Cochrane Database of Systematic Reviews · 2019 · Cochrane Library
NeuromuscularAMSTAR 2: not assessed
SoF tableOnly the outcomes this review put in its Summary of Findings table are listed (the Cochrane Handbook caps them at about seven per comparison). Its other analyses are not shown here.
Questions
14
current version
GRADE rated
11
No studies
3
Included studies
NR
No studies 3 · 21.4%Ungraded 0 · 0%Very low 4 · 28.6%Low 0 · 0%Moderate 7 · 50%High 0 · 0%
Ungraded means an effect was reported without a GRADE rating; it is not a gap. No studies means the review found nothing to answer the question. The six categories are a display order, not a score.
Questions and results
Intrathecal injected nusinersen compared to sham procedure for infants with SMA and 2 SMN2 copies
Population: infants with SMA and 2 SMN2 copies
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Time from birth until death or full‐time ventilationa Intrathecal injected nusinersen vs sham procedure | HR 0.53 [0.32, 0.89] | 1 study · 121 participants | Moderate Downgraded: risk of bias, imprecision |
Acquisition of head control within one year after the onset of treatment Intrathecal injected nusinersen vs sham procedure | RR 16.95 [1.04, 274.84] | 1 study · 110 participants | Moderate Downgraded: risk of bias, imprecision |
Acquisition of the ability to sit within one year after the onset of treatment Intrathecal injected nusinersen vs sham procedure | RR 6.68 [0.39, 115.38] | 1 study · 110 participants | Moderate Downgraded: risk of bias, imprecision |
Acquisition of the ability to stand within one year after the onset of treatment Intrathecal injected nusinersen vs sham procedure | RR 1.54 [0.06, 36.92] | 1 study · 110 participants | Moderate Downgraded: risk of bias, imprecision |
Change in motor disability score ‐ response on HINE‐2 within one year after the onset of treatmente Intrathecal injected nusinersen vs sham procedure | RR 38.51 [2.43, 610.14] | 1 study · 110 participants | Moderate Downgraded: risk of bias, imprecision |
Adverse events attributable to treatment Measured as adverse events (all) Intrathecal injected nusinersen vs sham procedure | RR 0.99 [0.92, 1.05] | 1 study · 121 participants | Moderate Downgraded: imprecision |
Severe adverse events attributable to treatment Measured as severe adverse events (all) Intrathecal injected nusinersen vs sham procedure | RR 0.7 [0.55, 0.89] | 1 study · 121 participants | Moderate Downgraded: imprecision |
Riluzole compared to placebo for children with SMA type I
Population: children with SMA type I
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Time from birth until death or full‐time ventilation Riluzole vs placebo | no estimate extracted | 1 study · 10 participants | Very low Downgraded: risk of bias, imprecision |
Acquisition of head control Riluzole vs placebo | no estimate extracted | NR | No studies |
Acquisition of the ability to sit Riluzole vs placebo | no estimate extracted | 1 study · 10 participants | Very low Downgraded: risk of bias, imprecision |
Acquisition of the ability to stand Riluzole vs placebo | no estimate extracted | NR | No studies |
Change in motor disability score Riluzole vs placebo | no estimate extracted | NR | No studies |
Adverse events attributable to treatment Riluzole vs placebo | no estimate extracted | 1 study · 10 participants | Very low Downgraded: risk of bias, imprecision |
Severe adverse events attributable to treatment Riluzole vs placebo | no estimate extracted | 1 study · 10 participants | Very low Downgraded: risk of bias, imprecision |