Thirty-two studies were included in the review: 23 open-label studies (n=682) and nine randomised placebo-controlled studies (n=311, range six to 82).
Agalsidase beta and agalsidase alpha were associated with significant decreases in mean plasma GL-3 levels in three out of four RCTs and four out of nine open-label studies.
Normalisation of GL-3 in renal cells using agalsidase beta (1.0mg/kg every other week) was reported in one of two RCTs and four out of four open-label studies; no treatment effect of agalsidase alpha was found in one RCT.
Small, non-significant reductions in urinary GL-3 excretion were reported in three out of three open-label studies.
Cardiac GL-3 was significantly reduced with agalsidase beta in one RCT that reported this endpoint; agalsidase alpha did not significantly reduce cardiac GL-3 (one RCT).
Agalsidase beta was associated with positive effects on GL-3 clearance in the skin in two RCTs and three open-label studies.
The most common adverse events with enzyme replacement therapy were mild to moderate infusion reactions.
Further results (including results for important subgroups) were reported in the paper.