Cochrane · systematic review · CD011539
Enzyme replacement therapy for infantile‐onset Pompe disease
Chen, M, Zhang, L, Quan, S · Cochrane Database of Systematic Reviews · 2017 · 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
7
current version
GRADE rated
6
No studies
0
Included studies
NR
No studies 0 · 0%Ungraded 1 · 14.3%Very low 0 · 0%Low 6 · 85.7%Moderate 0 · 0%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
Enzyme replacement therapy for infantile‐onset Pompe disease
Population: infantile‐onset Pompe disease
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Cardiac function | no estimate extracted | 1 study · 18 participants | Low Downgraded: risk of bias |
Time to ventilation | no estimate extracted | 1 study · 18 participants | Low Downgraded: risk of bias |
Survival | 61 | 1 study · 18 participants | Low Downgraded: risk of bias |
Motor development | no estimate extracted | 1 study · 18 participants | Low Downgraded: risk of bias |
Quality of life | no estimate extracted | 1 study · 18 participants | Ungraded |
Infusion‐related events | RR 0.83 [0.4, 1.76] | 1 study · 18 participants | Low Downgraded: risk of bias, imprecision |
Other adverse events | no estimate extracted | 1 study · 18 participants | Low Downgraded: risk of bias |