Twenty-one studies were included (368 patients; range one to 90; 27 children and 251 adults).
Of 138 patients, creatine kinase levels decreased in 69.6%, stabilized in 10.9%, and increased in 19.6% of patients, relative to baseline. Of 122 patients, the results of the 6-min walking test improved in 77.9%, stabilised in 8.2%, and declined in 13.9%. Of the nine studies (115 patients) that reported ambulatory status, eight reported improvement in at least one patient, and one reported no overall change; most studies did not report changes in ambulatory status from baseline. Of 124 patients, forced vital capacity improved in 51.6%, stabilised in 13.7%, and declined in 34.7%. Of 66 patients, the need for ventilatory support decreased in 59.1%, stabilised in 36.4% and increased in 4.5%. Further results for subgroups of patients and different time-points for outcome measurement were reported.
In terms of quality of life (measured using the short-form-36 in 156 patients), 8.3% reported an overall improvement, 23.1% reported improvements in both mental and physical component scores, 7.7 % reported improvements only in the physical component score, 15.4% showed improved mental component scores, and 23.1 % reported improved bodily pain; there were no details as to which scores improved in 2.6% of patients.
Adverse events were reported for 38 patients; 34 patients experienced mild to moderately severe events and four experienced severe or serious events (fatal tracheal haemorrhage; severe emphysema and/or pneumothorax; severe tongue oedema). Of 128 patients for whom data on the development of antibodies to alglucosidase alfa were available, 121 converted to low IgG titers, three had anaphylactic reactions and two developed antibodies to alglucosidase alfa.