Unknown

Dataset Information

0

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

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.

SUBMITTER: Hughes D 

PROVIDER: S-EPMC5442692 | biostudies-literature | 2017 May

REPOSITORIES: biostudies-literature

Json Xml
altmetric image

Publications

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

Hughes D D   Giugliani R R   Guffon N N   Jones S A SA   Mengel K E KE   Parini R R   Matousek R R   Hawley S M SM   Quartel A A  

Orphanet journal of rare diseases 20170523 1

<h4>Background</h4>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  ...[more]

Similar Datasets

Unknown Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome.
| S-EPMC5065598 | biostudies-literature
Unknown Clinical Utility of Elosulfase Alfa in the Treatment of Morquio A Syndrome.
| S-EPMC8759989 | biostudies-literature
Unknown Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England.
| S-EPMC7818902 | biostudies-literature
Unknown Pharmacokinetic and pharmacodynamic evaluation of elosulfase alfa, an enzyme replacement therapy in patients with Morquio A syndrome.
| S-EPMC4243006 | biostudies-literature
Unknown Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y.
| S-EPMC4700045 | biostudies-literature
Unknown Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open-label, phase 2 study.
| S-EPMC5298029 | biostudies-literature
Unknown Elosulfase alfa for mucopolysaccharidosis type IVA: Real-world experience in 7 patients from the Spanish Morquio-A early access program.
| S-EPMC6047108 | biostudies-literature
Unknown Elosulfase alfa enzyme replacement therapy attenuates disease progression in a non-ambulatory Japanese patient with Morquio A syndrome (case report).
| S-EPMC5604956 | biostudies-literature
Unknown Cardiopulmonary Exercise Testing Reflects Improved Exercise Capacity in Response to Treatment in Morquio A Patients: Results of a 52-Week Pilot Study of Two Different Doses of Elosulfase Alfa.
| S-EPMC6226396 | biostudies-literature
Unknown Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study.
| S-EPMC4744659 | biostudies-literature