Medizinische Universität Graz Austria/Österreich - Forschungsportal - Medical University of Graz
Gewählte Publikation:
SHR Neuro Krebs Kardio Lipid Stoffw Microb
Harmatz, P; Giugliani, R; Schwartz, IV; Guffon, N; Teles, EL; Miranda, MC; Wraith, JE; Beck, M; Arash, L; Scarpa, M; Ketteridge, D; Hopwood, JJ; Plecko, B; Steiner, R; Whitley, CB; Kaplan, P; Yu, ZF; Swiedler, SJ; Decker, C; MPS VI Study Group.
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase.
Mol Genet Metab. 2008; 94(4):469-475
Doi: 10.1016/j.ymgme.2008.04.001
Web of Science
PubMed
FullText
FullText_MUG
- Co-Autor*innen der Med Uni Graz
- Plecko Barbara
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- Abstract:
- The objective of this study was to evaluate the long-term clinical benefits and safety of recombinant human arylsulfatase B (rhASB) treatment of mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome), a lysosomal storage disease. Fifty-six patients derived from 3 clinical studies were followed in open-label extension studies for a total period of 97-260 Weeks. All patients received weekly infusions of rhASB at 1 mg/kg. Efficacy was evaluated by (1) distance walked in a 12-minute walk test (12MWT) or 6-minute walk test (6MWT), (2) stairs climbed in the 3-minute stair climb (3MSC), and (3) reduction in urinary glycosaminoglycans (GAG). Safety was evaluated by compliance, adverse event (AE) reporting and adherence to treatment. A significant reduction in urinary GAG (71-79%) was sustained. For the 12MWT, subjects in Phase 2 showed improvement of 255+/-191 m (mean+/-SD) at Week 144; those in Phase 3 Extension demonstrated improvement from study baseline of 183+/-26 m (mean+/- SE) in the rhASB/rhASB group at Week 96 and from treatment baseline (Week 24) of 117+/-25 m in the placebo/rhASB group. The Phase 1/2 6MWT and the 3MSC from Phase 2 and 3 also showed sustained improvements through the final study measurements. Compliance was 98% overall. Only 560 of 4121 reported AEs (14%) were related to treatment with only 10 of 560 (2%) described as severe. rhASB treatment up to 5 years results in sustained improvements in endurance and has an acceptable safety profile.
- Find related publications in this database (using NLM MeSH Indexing)
- Adolescent -
- Adult -
- Child -
- Child, Preschool -
- Female -
- Follow-Up Studies -
- Glycosaminoglycans - urine
- Humans -
- Male -
- Motor Activity -
- Mucopolysaccharidosis VI - physiopathology
- Mucopolysaccharidosis VI - therapy
- N-Acetylgalactosamine-4-Sulfatase - administration & dosage
- N-Acetylgalactosamine-4-Sulfatase - adverse effects
- N-Acetylgalactosamine-4-Sulfatase - therapeutic use
- Recombinant Proteins - administration & dosage
- Recombinant Proteins - adverse effects
- Recombinant Proteins - therapeutic use
- Treatment Outcome -
- Walking -
- Find related publications in this database (Keywords)
- mucopolysaccharidosis VI
- N-acetylgalactosamine-4-sulfatase
- enzyme replacement therapy
- glycosaminoglycans
- clinical trial