RT Journal Article
T1 Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis.
A1 Lund, Allan M
A1 Borgwardt, Line
A1 Cattaneo, Federica
A1 Ardigo, Diego
A1 Geraci, Silvia
A1 Gil-Campos, Mercedes
A1 De Meirleir, Linda
A1 Laroche, Cecile
A1 Dolhem, Philippe
A1 Cole, Duncan
A1 Tylki-Szymanska, Anna
A1 Lopez-Rodriguez, Monica
A1 Guillen-Navarro, Encarna
A1 Dali, Christine I
A1 Heron, Benedicte
A1 Fogh, Jens
A1 Muschol, Nicole
A1 Phillips, Dawn
A1 Van den Hout, J M Hannerieke
A1 Jones, Simon A
A1 Amraoui, Yasmina
A1 Harmatz, Paul
A1 Guffon, Nathalie
K1 Alpha-mannosidosis
K1 Enzyme replacement therapy
K1 Integrated analysis
K1 Lysosomal storage disorder
K1 Recombinant human alpha-mannosidase
K1 Velmanase alfa
AB 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). Patient data (n = 33; 14 adults, 19 paediatric) from the clinical development programme for velmanase alfa were integrated in this prospectively-designed analysis of long-term efficacy and safety. Patients who participated in the phase I/II or phase III trials and were continuing to receive treatment after completion of the trials were invited to participate in a comprehensive evaluation visit to assess long-term outcomes. Primary endpoints were changes in serum oligosaccharide and the 3-minute stair climb test (3MSCT). Mean (SD) treatment exposure was 29.3 (15.2) months. Serum oligosaccharide levels were significantly reduced in the overall population at 12 months (mean change: -72.7%, P  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.
PB John Wiley & Sons
YR 2018
FD 2018-03-18
LK http://hdl.handle.net/10668/12421
UL http://hdl.handle.net/10668/12421
LA en
NO Lund AM, Borgwardt L, Cattaneo F, Ardigò D, Geraci S, Gil-Campos M, et al. Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis. J Inherit Metab Dis. 2018 Nov;41(6):1225-1233
DS RISalud
RD Apr 11, 2025