No abstract

The search found no trials comparing the effectiveness and safety of enzyme replacement therapy to another intervention, no intervention or placebo. One small randomized controlled trial provided no robust evidence for which dosing schedule of alglucosidase alfa was more effective to treat infantile-onset Pompe disease. It is not deemed ethical to proceed with new placebo-controlled trials, therefore a randomized controlled trial with a large sample size comparing different dosing schedules of enzyme replacement therapy is needed. The main clinical outcomes (i.e. cardiac function, invasive ventilation, survival, motor development, adverse events (e.g. the development of antibodies)) should be standardized when evaluated and reported.

No abstract

(i) A management guideline for off-label drug use is urgently needed, with which we can guide medical institutions to establish the management regulations of off-label drug use. (ii) Pediatric clinical research should be promoted actively, and pharmaceutical enterprises should be encouraged to completely provide pediatric drug information. (iv) Academic organizations should be invited to join in for best professional drug use. (v) Pediatric patients and their families should receive the education on rational drug use.