Cochrane · systematic review · CD012993
Enzyme replacement therapy for late‐onset Pompe disease
Dalmia, S, Sharma, R, Ramaswami, U, et al. · Cochrane Database of Systematic Reviews · 2023 · Cochrane Library
Cystic Fibrosis and Genetic DisordersAMSTAR 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
43
current version
GRADE rated
23
No studies
20
Included studies
NR
No studies 20 · 46.5%Ungraded 0 · 0%Very low 7 · 16.3%Low 11 · 25.6%Moderate 5 · 11.6%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
Alglucosidase alfa versus placebo
Population: 90 people with LOPD
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change from baseline in 6MWT (metres) Alglucosidase alfa vs placebo | no estimate extracted | 1 study · 90 participants | Moderate Downgraded: risk of bias |
Change in FVC (% predicted) Alglucosidase alfa vs placebo | no estimate extracted | 1 study · 90 participants | Moderate Downgraded: risk of bias |
Infusion reactions Alglucosidase alfa vs placebo | RR 1.21 [0.57, 2.61] | 1 study · 90 participants | Low Downgraded: risk of bias, imprecision |
Need for respiratory support Alglucosidase alfa vs placebo | no estimate extracted | NR | No studies |
Use of a walking aid/wheelchair Alglucosidase alfa vs placebo | no estimate extracted | NR | No studies |
QoL (PCS in SF‐36 (measured on a scale from 0 to 100)) Alglucosidase alfa vs placebo | no estimate extracted | 1 study · 90 participants | Low Downgraded: risk of bias, imprecision |
Treatment‐related AEs Alglucosidase alfa vs placebo | RR 0.94 [0.64, 1.39] | 1 study · 90 participants | Low Downgraded: risk of bias, imprecision |
Alglucosidase alfa plus clenbuterol compared to alglucosidase alfa plus placebo
Population: 13 people with LOPD
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in 6MWT from baseline (metres) Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo | no estimate extracted | 1 study · 10 participants | Very low Downgraded: risk of bias, imprecision |
Change in FVC from baseline (% predicted) Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo | no estimate extracted | 1 study · 11 participants | Very low Downgraded: risk of bias, imprecision |
Infusion reactions Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo | no estimate extracted | NR | No studies |
Need for respiratory support Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo | no estimate extracted | NR | No studies |
Use of a walking aid / wheelchair Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo | no estimate extracted | NR | No studies |
QoL Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo | no estimate extracted | NR | No studies |
Treatment‐related adverse events Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo | no estimate extracted | NR | No studies |
Alglucosidase alfa plus albuterol compared to alglucosidase alfa plus placebo
Population: 13 people with LOPD
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change in 6MWT from baseline (metres) Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo | no estimate extracted | 1 study · 5 participants | Very low Downgraded: risk of bias, imprecision |
Change in FVC from baseline (% predicted) Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo | no estimate extracted | 1 study · 5 participants | Very low Downgraded: risk of bias, imprecision |
Infusion reactions Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo | no estimate extracted | NR | No studies |
Need for respiratory support Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo | no estimate extracted | NR | No studies |
Use of a walking aid / wheelchair Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo | no estimate extracted | NR | No studies |
QoL Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo | no estimate extracted | NR | No studies |
Adverse events Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo | RR 0.67 [0.38, 1.18] | 1 study · 13 participants | Very low Downgraded: risk of bias, imprecision |
VAL‐1221 compared to alglucosidase alfa
Population: adults with LOPD
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Change from baseline in 6MWT (metres) VAL‐1221 vs alglucosidase alfa | no estimate extracted | 1 study · 12 participants | Very low Downgraded: risk of bias, imprecision |
Change in FVC (% predicted) VAL‐1221 vs alglucosidase alfa | no estimate extracted | NR | No studies |
Infusion reactions VAL‐1221 vs alglucosidase alfa | OR 2.8 | 1 study · 12 participants | Very low Downgraded: risk of bias, imprecision |
Need for respiratory support VAL‐1221 vs alglucosidase alfa | no estimate extracted | NR | No studies |
Use of a walking aid/wheelchair VAL‐1221 vs alglucosidase alfa | no estimate extracted | NR | No studies |
QoL VAL‐1221 vs alglucosidase alfa | no estimate extracted | NR | No studies |
Treatment‐related adverse events VAL‐1221 vs alglucosidase alfa | no estimate extracted | NR | No studies |
Cipaglucosidase plus miglustat versus alglucosidase alfa plus placebo
Population: adults with LOPD
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
6MWT: mean change from baseline in 6MWT (metres) Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo | no estimate extracted | 1 study · 117 participants | Low Downgraded: risk of bias, imprecision |
Change in sitting FVC (% predicted) Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo | no estimate extracted | 1 study · 133 participants | Moderate Downgraded: risk of bias |
Change in SNIP (% predicted) Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo | no estimate extracted | 1 study · 121 participants | Low Downgraded: risk of bias, imprecision |
Infusion reactions Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo | RR 0.94 [0.49, 1.8] | 1 study · 123 participants | Low Downgraded: risk of bias, imprecision |
Need for respiratory support Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo | no estimate extracted | NR | No studies |
Use of a walking aid / wheelchair Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo | no estimate extracted | NR | No studies |
QoL: change from baseline to PROMIS score (physical function score and fatigue score) Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo | no estimate extracted | 1 study · 121 participants | Low Downgraded: risk of bias, imprecision |
Treatment‐related AEs: total treatment‐emergent adverse events potentially related to treatment Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo | RR 0.83 [0.49, 1.4] | 1 study · 123 participants | Low Downgraded: risk of bias, imprecision |
Avalglucosidase alfa versus alglucosidase alfa
Population: adults with LOPD
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
6MWT: mean change from baseline (metres) Avalglucosidase alfa vs alglucosidase alfa | no estimate extracted | 1 study · 100 participants | Moderate Downgraded: risk of bias |
Change in FVC (% predicted) Avalglucosidase alfa vs alglucosidase alfa | no estimate extracted | 1 study · 100 participants | Moderate Downgraded: risk of bias |
Infusion reactions Avalglucosidase alfa vs alglucosidase alfa | RR 0.78 [0.42, 1.45] | 1 study · 100 participants | Low Downgraded: risk of bias, imprecision |
Need for respiratory support Avalglucosidase alfa vs alglucosidase alfa | no estimate extracted | NR | No studies |
Use of a walking aid / wheelchair Avalglucosidase alfa vs alglucosidase alfa | no estimate extracted | NR | No studies |
QoL: change from baseline in EQ‐5D‐5L score Avalglucosidase alfa vs alglucosidase alfa | no estimate extracted | 1 study · 100 participants | Low Downgraded: risk of bias, imprecision |
Treatment‐related AEs: total treatment‐emergent adverse events potentially related to treatment Avalglucosidase alfa vs alglucosidase alfa | RR 0.92 [0.61, 1.4] | 1 study · 100 participants | Low Downgraded: risk of bias, imprecision |