Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa

Detalhes bibliográficos
Autor(a) principal: Hughes, Derralynn A.
Data de Publicação: 2017
Outros Autores: Giugliani, Roberto, Guffon, Nathalie, Jones, Simon A., Mengel, Karl Eugen, Parini, Rossella, Matousek, R., Hawley, Sara M., Quartel, Adrian
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da UFRGS
Texto Completo: http://hdl.handle.net/10183/194742
Resumo: 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.
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spelling Hughes, Derralynn A.Giugliani, RobertoGuffon, NathalieJones, Simon A.Mengel, Karl EugenParini, RossellaMatousek, R.Hawley, Sara M.Quartel, Adrian2019-05-29T02:44:09Z20171750-1172http://hdl.handle.net/10183/194742001089726Background: 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.application/pdfengOrphanet journal of rare diseases. London. vol. 12 (2017), 98, 8 f.Mucopolissacaridose IVTerapia de reposição de enzimasMorquio syndrome AAdultsAdulthoodAdvanced diseaseLong-termEnzyme replacement therapyElosulfase alfaMucopolysaccharidosis IVAClinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfaEstrangeiroinfo:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/openAccessreponame:Repositório Institucional da UFRGSinstname:Universidade Federal do Rio Grande do Sul (UFRGS)instacron:UFRGSTEXT001089726.pdf.txt001089726.pdf.txtExtracted Texttext/plain36417http://www.lume.ufrgs.br/bitstream/10183/194742/2/001089726.pdf.txtf9f70f95fc3e46d67324f3da1ca81c11MD52ORIGINAL001089726.pdfTexto completo (inglês)application/pdf744128http://www.lume.ufrgs.br/bitstream/10183/194742/1/001089726.pdf64a1bd573ca192b86bd6d4cba37e02ffMD5110183/1947422019-05-30 02:40:29.206135oai:www.lume.ufrgs.br:10183/194742Repositório de PublicaçõesPUBhttps://lume.ufrgs.br/oai/requestopendoar:2019-05-30T05:40:29Repositório Institucional da UFRGS - Universidade Federal do Rio Grande do Sul (UFRGS)false
dc.title.pt_BR.fl_str_mv Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa
title Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa
spellingShingle Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa
Hughes, Derralynn A.
Mucopolissacaridose IV
Terapia de reposição de enzimas
Morquio syndrome A
Adults
Adulthood
Advanced disease
Long-term
Enzyme replacement therapy
Elosulfase alfa
Mucopolysaccharidosis IVA
title_short Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa
title_full Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa
title_fullStr Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa
title_full_unstemmed Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa
title_sort Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa
author Hughes, Derralynn A.
author_facet Hughes, Derralynn A.
Giugliani, Roberto
Guffon, Nathalie
Jones, Simon A.
Mengel, Karl Eugen
Parini, Rossella
Matousek, R.
Hawley, Sara M.
Quartel, Adrian
author_role author
author2 Giugliani, Roberto
Guffon, Nathalie
Jones, Simon A.
Mengel, Karl Eugen
Parini, Rossella
Matousek, R.
Hawley, Sara M.
Quartel, Adrian
author2_role author
author
author
author
author
author
author
author
dc.contributor.author.fl_str_mv Hughes, Derralynn A.
Giugliani, Roberto
Guffon, Nathalie
Jones, Simon A.
Mengel, Karl Eugen
Parini, Rossella
Matousek, R.
Hawley, Sara M.
Quartel, Adrian
dc.subject.por.fl_str_mv Mucopolissacaridose IV
Terapia de reposição de enzimas
topic Mucopolissacaridose IV
Terapia de reposição de enzimas
Morquio syndrome A
Adults
Adulthood
Advanced disease
Long-term
Enzyme replacement therapy
Elosulfase alfa
Mucopolysaccharidosis IVA
dc.subject.eng.fl_str_mv Morquio syndrome A
Adults
Adulthood
Advanced disease
Long-term
Enzyme replacement therapy
Elosulfase alfa
Mucopolysaccharidosis IVA
description 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.
publishDate 2017
dc.date.issued.fl_str_mv 2017
dc.date.accessioned.fl_str_mv 2019-05-29T02:44:09Z
dc.type.driver.fl_str_mv Estrangeiro
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dc.identifier.uri.fl_str_mv http://hdl.handle.net/10183/194742
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001089726
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dc.relation.ispartof.pt_BR.fl_str_mv Orphanet journal of rare diseases. London. vol. 12 (2017), 98, 8 f.
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