Introduction: Long-term outcome data provide important insights into the clinical utility of enzyme replacement therapies. Such data are presented for velmanase alfa in the treatment of alpha-mannosidosis AM. Primary endpoints were changes in serum oligosaccharide and the 3-minute stair climb test 3MSCT. Results: Mean SD treatment exposure was Serum oligosaccharide levels were significantly reduced in the overall population at 12 months mean change: No treatment-emergent adverse events were reported leading to permanent treatment discontinuation.
Conclusions: Patients treated with velmanase alfa experienced improvements in biochemical and functional measures that were maintained for up to 4 years. Long term follow-up is important and further supports the use of velmanase alfa as an effective and well-tolerated treatment for AM. Based on the currently available data set, no baseline characteristic can be predictive of treatment outcome. Early treatment during paediatric age showed better outcome in functional endpoints.
Keywords: Alpha-mannosidosis; Enzyme replacement therapy; Integrated analysis; Lysosomal storage disorder; Recombinant human alpha-mannosidase; Velmanase alfa. Abstract Introduction: Long-term outcome data provide important insights into the clinical utility of enzyme replacement therapies.
Substances Recombinant Proteins alpha-Mannosidase.
