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Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome

Hendriksz, CJ; Berger, KI; Parini, R; AlSayed, MD; Raiman, J; Giugliani, R; Mitchell, JJ; ... Harmatz, PR; + view all (2016) Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome. Journal of Inherited Metabolic Disease , 39 (6) pp. 839-847. 10.1007/s10545-016-9973-6. Green open access

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Abstract

OBJECTIVE: To present long-term respiratory function outcomes from an open-label, multi-center, phase 3 extension study (MOR-005) of elosulfase alfa enzyme replacement therapy (ERT) in patients with Morquio A syndrome. METHODS: In part 1 of MOR-005, patients initially randomized to ERT in the 24-week pivotal study (MOR-004) remained on their regimen (2.0 mg/kg/week or every other week); placebo patients were re-randomized to one of the two regimens. During part 2, all patients received elosulfase alfa 2.0 mg/kg/week. Respiratory function was one of the efficacy endpoints evaluated in MOR-005. Change from MOR-004 baseline to 120 weeks of treatment for the combined population was determined and compared with results from untreated patients from a Morquio A natural history study (MorCAP). RESULTS: Maximum voluntary ventilation (MVV) improved up to week 72 and then stabilized; forced vital capacity (FVC) and forced expiratory volume in 1 s (FEV1) increased continuously over 120 weeks. Mean increases in the modified per-protocol population was 9.2 % for FVC, 8.8 % for FEV1, and 6.1 % for MVV after 120 weeks. All patients ≤14 years showed respiratory improvements, presumably in part related to growth; however, these were greater in treated patients. For those >14 years, treated patients showed improvements, while deterioration occurred in untreated. Altogether, the improvements were significantly greater (P < 0.05) in treated patients. CONCLUSIONS: Long-term ERT is associated with sustained improvements in respiratory function in Morquio A. In younger patients (≤14 years), some improvement may be ascribed to growth. In older patients, other mechanisms, e.g., decreased glycosaminoglycan storage, are likely involved.

Type: Article
Title: Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome
Location: Netherlands
Open access status: An open access version is available from UCL Discovery
DOI: 10.1007/s10545-016-9973-6
Publisher version: http://doi.org/10.1007/s10545-016-9973-6
Language: English
Additional information: © The Author(s) 2016. This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http:// creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made.
Keywords: Adolescent, Adult, Aged, Child, Child, Preschool, Chondroitinsulfatases, Double-Blind Method, Enzyme Replacement Therapy, Female, Forced Expiratory Volume, Humans, Long-Term Care, Male, Middle Aged, Mucopolysaccharidosis IV, Respiration, Respiratory Function Tests, Young Adult
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: https://discovery.ucl.ac.uk/id/eprint/10039584
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