Summary

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• This summary is based on the review of one randomized controlled trial. ^[1]
• In children under 6 years with alpha-mannosidosis (AM), velmanase alfa (VA) was associated with reductions in serum oligosaccharide concentrations, improvements in hearing, enhanced immunological profiles, and better quality of life outcomes.
• The abstract suggests that early treatment with VA in young children may yield greater clinical benefits over time compared to starting treatment later, though no direct comparisons with older patients were provided.
• No comparative effectiveness data between VA and other treatments were provided in the abstract.
• Four out of five children (80%) treated with velmanase alfa experienced adverse drug reactions, totaling 16 events. Most adverse events (99.5%) were mild or moderate, and none resulted in study discontinuation.
• Two children had infusion-related reactions (12 events in total), and four developed antidrug antibodies, three of which were neutralizing, but these did not require stopping the treatment.
• The study focused on children under 6 years of age with alpha-mannosidosis, with a gender distribution of 3 males and 2 females. It suggests that early treatment with VA in this population resulted in improvements in serum oligosaccharide concentrations, hearing, immunological profiles, and quality of life, with no significant adverse events leading to study discontinuation. The benefits observed suggest potential advantages of initiating treatment at a younger age, though the small sample size limits broader conclusion.