evidence/gaps
Cochrane · systematic review · CD009354

Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I

Jameson, E, Jones, S, Remmington, T · Cochrane Database of Systematic Reviews · 2019 · 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
7
No studies
0
Included studies
NR
No studies 0 · 0%Ungraded 0 · 0%Very low 1 · 14.3%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

Summary of findings

Population: adults and children with mucopolysaccharidosis I
7 questions
OutcomeEffectEvidenceStatus / GRADE
Change in urinary GAG excretion from baseline
no estimate extracted1 study · 43 participantsLow
Downgraded: risk of bias, imprecision
Mean percentage change from baseline in FVC (% predicted)
no estimate extracted1 study · 45 participantsLow
Downgraded: risk of bias, imprecision
Mean change from baseline in 6MWT (metres)
no estimate extracted1 study · 45 participantsLow
Downgraded: risk of bias, imprecision
Infusion‐related reactions (number of participants with one or more reaction)
OR 0.51 [0.15, 1.71]1 study · 45 participantsLow
Downgraded: risk of bias, imprecision
Antibody production: number of participants developing IgG antibodies
OR 385.4 [17.47, 8500.51]1 study · 45 participantsVery low
Downgraded: risk of bias, imprecision
Quality of life
no estimate extracted1 study · 45 participantsLow
Downgraded: risk of bias, imprecision
Mortality: number of deaths
no estimate extracted1 study · 45 participantsLow
Downgraded: risk of bias, imprecision