Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa
dc.contributor.author | Hughes, Derralynn A. | pt_BR |
dc.contributor.author | Giugliani, Roberto | pt_BR |
dc.contributor.author | Guffon, Nathalie | pt_BR |
dc.contributor.author | Jones, Simon A. | pt_BR |
dc.contributor.author | Mengel, Karl Eugen | pt_BR |
dc.contributor.author | Parini, Rossella | pt_BR |
dc.contributor.author | Matousek, R. | pt_BR |
dc.contributor.author | Hawley, Sara M. | pt_BR |
dc.contributor.author | Quartel, Adrian | pt_BR |
dc.date.accessioned | 2019-05-29T02:44:09Z | pt_BR |
dc.date.issued | 2017 | pt_BR |
dc.identifier.issn | 1750-1172 | pt_BR |
dc.identifier.uri | http://hdl.handle.net/10183/194742 | pt_BR |
dc.description.abstract | Background: This post hoc subanalysis examined outcomes in adult patients with Morquio A (mucopolysaccharidosis IVA) who received enzyme replacement therapy (ERT) with elosulfase alfa over a 120-weeks period. Patients ≥18 years of age evaluated in an open-label, long-term extension study of elosulfase alfa (modified per protocol [MPP], n = 32; intent-to-treat [ITT], n = 37; MOR-005; NCT01415427) were compared with the ≥18-year-old untreated population with 2-years follow-up from a Morquio A natural history study (n = 10; MorCAP; NCT00787995). The MOR-005 MPP population excluded patients who underwent orthopedic surgical procedures or were noncompliant with study protocol (defined as missing ≥20% of ERT infusions). No MorCAP patients underwent orthopedic surgical procedures during the relevant time period. Endurance was assessed by the 6-min walk test (6MWT) and 3-min stair climb test (3MSCT). Activities of daily living (ADLs) were assessed by the MPS Health Assessment Questionnaire (MPS HAQ). Results: Least squares (LS) mean (SE) 6MWT distances increased by 34.9 (11.7) m (MPP) and 30.5 (10.8) m (ITT) by week 120; LS mean (SE) change in 3MSCT at week 120 was 6.7 (1.8) stairs/min (MPP) and 5.9 (1.7) stairs/min (ITT). MorCAP patients showed no improvement in 6MWT distance or 3MSCT over a similar period of time. Pulmonary function measures remained unchanged in both MOR-005 and MorCAP adults. All MPS HAQ domain scores improved in MOR-005 adults, whereas MorCAP adults had unchanged caregiver assistance and mobility outcomes and worsened self-care outcomes. Conclusions: Long-term ERT in adult patients with Morquio A was associated with increased endurance and improvement in performance of ADLs. Trial registration: Trial Registration NCT01415427. Name of registry: Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome). Registered 8 August 2011, retrospectively registered. | en |
dc.format.mimetype | application/pdf | pt_BR |
dc.language.iso | eng | pt_BR |
dc.relation.ispartof | Orphanet journal of rare diseases. London. vol. 12 (2017), 98, 8 f. | pt_BR |
dc.rights | Open Access | en |
dc.subject | Mucopolissacaridose IV | pt_BR |
dc.subject | Morquio syndrome A | en |
dc.subject | Adults | en |
dc.subject | Terapia de reposição de enzimas | pt_BR |
dc.subject | Adulthood | en |
dc.subject | Advanced disease | en |
dc.subject | Long-term | en |
dc.subject | Enzyme replacement therapy | en |
dc.subject | Elosulfase alfa | en |
dc.subject | Mucopolysaccharidosis IVA | en |
dc.title | Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa | pt_BR |
dc.type | Artigo de periódico | pt_BR |
dc.identifier.nrb | 001089726 | pt_BR |
dc.type.origin | Estrangeiro | pt_BR |
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