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Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome.
- Author(s): Hendriksz, Christian J;
- Berger, Kenneth I;
- Parini, Rossella;
- AlSayed, Moeenaldeen D;
- Raiman, Julian;
- Giugliani, Roberto;
- Mitchell, John J;
- Burton, Barbara K;
- Guelbert, Norberto;
- Stewart, Fiona;
- Hughes, Derralynn A;
- Matousek, Robert;
- Jurecki, Elaina;
- Decker, Celeste;
- Harmatz, Paul R
- et al.
Published Web Locationhttps://doi.org/10.1007/s10545-016-9973-6
ObjectiveTo 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.
MethodsIn 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).
ResultsMaximum 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.
ConclusionsLong-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.
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