Cochrane · systematic review · CD006282
Drug treatment for spinal muscular atrophy types II and III
Wadman, RI, van der Pol, WL, Bosboom, WMJ, et al. · Cochrane Database of Systematic Reviews · 2020 · 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
75
current version
GRADE rated
57
No studies
18
Included studies
NR
No studies 18 · 24%Ungraded 0 · 0%Very low 8 · 10.7%Low 32 · 42.7%Moderate 17 · 22.7%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
Oral creatine compared to placebo for children with SMA types II and III
Population: children with SMA types II and III
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in disability score Oral creatine vs placebo | no estimate extracted | 1 study · 40 participants | Moderate Downgraded: imprecision |
Change in total muscle strength (total muscle strength) Oral creatine vs placebo | no estimate extracted | 1 study · 22 participants | Low Downgraded: imprecision, inconsistency |
Acquiring the ability to stand or walk Oral creatine vs placebo | no estimate extracted | NR | No studies |
Change in quality of life Oral creatine vs placebo | no estimate extracted | 1 study · 38 participants | Low Downgraded: imprecision, inconsistency |
Change in pulmonary function Oral creatine vs placebo | no estimate extracted | 1 study · 23 participants | Low Downgraded: imprecision, inconsistency |
Time from beginning of treatment until death or full‐time ventilation Oral creatine vs placebo | no estimate extracted | 1 study · 40 participants | Moderate Downgraded: imprecision |
Adverse events related to treatment Oral creatine vs placebo | no estimate extracted | 1 study · 40 participants | Low Downgraded: risk of bias, imprecision |
Oral gabapentin compared to placebo for adults with SMA types II and III
Population: adults with SMA types II and III
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in disability score Oral gabapentin vs placebo | no estimate extracted | 1 study · 66 participants | Low Downgraded: risk of bias, imprecision |
Change in muscle strength assessed as: % change in total muscle strength from baseline Oral gabapentin vs placebo | no estimate extracted | 1 study · 50 participants | Low Downgraded: risk of bias, imprecision |
Acquiring ability to walk Oral gabapentin vs placebo | no estimate extracted | 1 study · 73 participants | Low Downgraded: risk of bias, imprecision |
Change in quality of life Oral gabapentin vs placebo | no estimate extracted | 1 study · 67 participants | Low Downgraded: risk of bias, imprecision |
Change in pulmonary function Oral gabapentin vs placebo | no estimate extracted | 1 study · 65 participants | Low Downgraded: risk of bias, imprecision |
Time from beginning of treatment to death or full‐time ventilation Oral gabapentin vs placebo | no estimate extracted | 1 study · 84 participants | Moderate Downgraded: imprecision |
Adverse events related to treatment Oral gabapentin vs placebo | no estimate extracted | 1 study · 65 participants | Low Downgraded: imprecision |
Oral hydroxyurea compared to placebo for children and adults with SMA types II and III
Population: children and adults with SMA types II and III
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in disability score Oral hydroxyurea vs placebo | no estimate extracted | 1 study · 57 participants | Low Downgraded: imprecision |
Change in disability score Oral hydroxyurea vs placebo | no estimate extracted | 1 study · 38 participants | Moderate Downgraded: imprecision |
Change in muscle strength Oral hydroxyurea vs placebo | no estimate extracted | 1 study · 57 participants | Moderate Downgraded: imprecision |
Acquiring the ability to stand or walk Oral hydroxyurea vs placebo | no estimate extracted | NR | No studies |
Change in quality of life Oral hydroxyurea vs placebo | no estimate extracted | NR | No studies |
Change in pulmonary function Oral hydroxyurea vs placebo | no estimate extracted | 1 study · 57 participants | Low Downgraded: imprecision, indirectness |
Time from beginning of treatment until death or full‐time ventilation Oral hydroxyurea vs placebo | no estimate extracted | 1 study · 57 participants | Moderate Downgraded: imprecision |
Adverse events related to treatment Oral hydroxyurea vs placebo | no estimate extracted | 1 study · 57 participants | Moderate Downgraded: imprecision |
Intrathecal injected nusinersen compared to sham procedure for children with SMA type II
Population: children with SMA type II
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in disability score Intrathecal injected nusinersen vs sham procedure | MD 5.9 [3.7, 8.1] | 1 study · 126 participants | Moderate Downgraded: imprecision |
Change in disability score (3 point‐change) Intrathecal injected nusinersen vs sham procedure | RR 1.8 [0.99, 3.1] | 1 study · 126 participants | Moderate Downgraded: imprecision |
Change in muscle strength Intrathecal injected nusinersen vs sham procedure | no estimate extracted | NR | No studies |
Acquiring the ability to stand or walk Intrathecal injected nusinersen vs sham procedure | RR 0.5 [0.03, 7.8] | 1 study · 126 participants | Low Downgraded: imprecision |
Change in quality of life Intrathecal injected nusinersen vs sham procedure | no estimate extracted | NR | No studies |
Change in pulmonary function Intrathecal injected nusinersen vs sham procedure | no estimate extracted | NR | No studies |
Time from beginning of treatment until death or full‐time ventilation Intrathecal injected nusinersen vs sham procedure | no estimate extracted | NR | No studies |
Adverse events related to treatment Intrathecal injected nusinersen vs sham procedure | RR 0.9 [0.9, 1] | 1 study · 126 participants | Moderate Downgraded: imprecision |
Oral olesoxime compared to placebo for non‐ambulatory children and adolescents with SMA types II and III
Population: non‐ambulatory children and adolescents with SMA types II and III
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in disability score Oral olesoxime vs placebo | no estimate extracted | 1 study · 160 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Change in disability score Oral olesoxime vs placebo | no estimate extracted | 1 study · 160 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Change in disability score Oral olesoxime vs placebo | RR 1.43 | 1 study · 160 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Change in disability score Oral olesoxime vs placebo | no estimate extracted | 1 study · 160 participants | Low Downgraded: risk of bias, imprecision |
Change in muscle strength Oral olesoxime vs placebo | no estimate extracted | NR | No studies |
Acquiring the ability to stand or walk Oral olesoxime vs placebo | no estimate extracted | NR | No studies |
Change in quality of life Oral olesoxime vs placebo | no estimate extracted | 1 study · 108 participants | Low Downgraded: risk of bias, imprecision |
Change in pulmonary function Oral olesoxime vs placebo | no estimate extracted | 1 study · 102 participants | Low Downgraded: risk of bias, imprecision |
Time from beginning of treatment until death or full‐time ventilation Oral olesoxime vs placebo | no estimate extracted | 1 study · 160 participants | Moderate Downgraded: imprecision |
Adverse events related to treatment Oral olesoxime vs placebo | RR 0.95 [0.91, 0.99] | 1 study · 165 participants | Moderate Downgraded: imprecision |
Oral phenylbutyrate compared to placebo for children with SMA type II
Population: children with SMA type II
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in disability score Oral phenylbutyrate vs placebo | no estimate extracted | 1 study · 90 participants | Low Downgraded: risk of bias, imprecision |
Change in muscle strength Oral phenylbutyrate vs placebo | no estimate extracted | 1 study · 70 participants | Low Downgraded: risk of bias, imprecision |
Acquiring the ability to stand or walk Oral phenylbutyrate vs placebo | no estimate extracted | NR | No studies |
Change in quality of life Oral phenylbutyrate vs placebo | no estimate extracted | NR | No studies |
Change in pulmonary function Oral phenylbutyrate vs placebo | no estimate extracted | 1 study · 67 participants | Low Downgraded: risk of bias, imprecision |
Time from beginning of treatment until death or full‐time ventilation Oral phenylbutyrate vs placebo | no estimate extracted | 1 study · 107 participants | Low Downgraded: risk of bias, imprecision |
Adverse events related to treatment Oral phenylbutyrate vs placebo | RR 3.1 [1.25, 7.84] | 1 study · 107 participants | Moderate Downgraded: imprecision |
Subcutaneous somatotropin compared to placebo for children and adults with SMA types II and III
Population: children and adults with SMA types II and III
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in disability score Subcutaneous somatotropin vs placebo | no estimate extracted | 19 participants | Very low Downgraded: risk of bias, imprecision |
Change in muscle strength Subcutaneous somatotropin vs placebo | no estimate extracted | 19 participants | Low Downgraded: imprecision |
Acquiring the ability to stand or walk Subcutaneous somatotropin vs placebo | no estimate extracted | NR | No studies |
Change in quality of life Subcutaneous somatotropin vs placebo | no estimate extracted | 19 participants | Very low Downgraded: risk of bias, imprecision |
Change in pulmonary function Subcutaneous somatotropin vs placebo | no estimate extracted | 19 participants | Low Downgraded: imprecision |
Time from beginning of treatment until death or full‐time ventilation Subcutaneous somatotropin vs placebo | no estimate extracted | 19 participants | Low Downgraded: imprecision |
Adverse events related to treatment Subcutaneous somatotropin vs placebo | RR 1.57 [0.78, 3.17] | 19 participants | Low Downgraded: imprecision |
Intravenous thyrotropin releasing hormone compared to placebo for children with SMA types II and III
Population: children with SMA types II and III
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in disability score Intravenous thyrotropin releasing hormone vs placebo | no estimate extracted | NR | No studies |
Change in muscle strength Intravenous thyrotropin releasing hormone vs placebo | no estimate extracted | 1 study · 9 participants | Very low Downgraded: risk of bias, imprecision |
Acquiring the ability to stand or walk Intravenous thyrotropin releasing hormone vs placebo | no estimate extracted | NR | No studies |
Change in quality of life Intravenous thyrotropin releasing hormone vs placebo | no estimate extracted | NR | No studies |
Change in pulmonary function Intravenous thyrotropin releasing hormone vs placebo | no estimate extracted | NR | No studies |
Time to death or full‐time ventilation Intravenous thyrotropin releasing hormone vs placebo | no estimate extracted | 1 study · 9 participants | Low Downgraded: risk of bias, imprecision |
Adverse events related to treatment Intravenous thyrotropin releasing hormone vs placebo | no estimate extracted | 1 study · 9 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Oral valproic acid plus acetyl‐L‐carnitine compared to placebo for non‐ambulatory children with SMA types II and III
Population: non‐ambulatory children with SMA types II and III
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in disability score Oral valproic acid plus acetyl‐L‐carnitine vs placebo | no estimate extracted | 1 study · 61 participants | Moderate Downgraded: imprecision |
Change in muscle strength Oral valproic acid plus acetyl‐L‐carnitine vs placebo | no estimate extracted | 1 study · 16 participants | Low Downgraded: imprecision |
Acquiring the ability to stand or walk Oral valproic acid plus acetyl‐L‐carnitine vs placebo | no estimate extracted | NR | No studies |
Change in quality of life Oral valproic acid plus acetyl‐L‐carnitine vs placebo | no estimate extracted | 1 study · 54 participants | Very low Downgraded: risk of bias, imprecision, inconsistency |
Change in pulmonary function Oral valproic acid plus acetyl‐L‐carnitine vs placebo | no estimate extracted | 1 study · 24 participants | Low Downgraded: risk of bias, imprecision |
Time from beginning of treatment until death or full‐time ventilation Oral valproic acid plus acetyl‐L‐carnitine vs placebo | no estimate extracted | 1 study · 61 participants | Moderate Downgraded: imprecision |
Adverse events related to treatment Oral valproic acid plus acetyl‐L‐carnitine vs placebo | RR 1.32 [0.92, 1.89] | 1 study · 61 participants | Moderate Downgraded: imprecision |
Oral valproic acid compared to placebo for ambulatory adults with SMA type III
Population: ambulatory adults with SMA type III
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in disability score Oral valproic acid vs placebo | no estimate extracted | 31 participants | Low Downgraded: imprecision |
Change in muscle strength Oral valproic acid vs placebo | no estimate extracted | 30 participants | Low Downgraded: imprecision |
Acquiring the ability to stand or walk Oral valproic acid vs placebo | no estimate extracted | NR | No studies |
Change in quality of life Oral valproic acid vs placebo | no estimate extracted | 28 participants | Moderate |
Change in pulmonary function Oral valproic acid vs placebo | no estimate extracted | 24 participants | Low Downgraded: imprecision |
Time from beginning of treatment until death or full‐time ventilation Oral valproic acid vs placebo | no estimate extracted | 33 participants | Low Downgraded: imprecision |
Adverse events related to treatment Oral valproic acid vs placebo | RR 0.8 [0.44, 1.44] | 33 participants | Low Downgraded: imprecision |