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Evaluation of the Long-Term Treatment Effects of Intravenous Idursulfase in Patients With Mucopolysaccharidosis II (MPS II) Using Statistical Modeling: Data From the Hunter Outcome Survey (HOS)

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source: Orphanet journal of rare diseases

year: 2021

authors: Muenzer J,Botha J,Harmatz P,Giugliani R,Kampmann C,Burton BK

summary/abstract:

Mucopolysaccharidosis II (MPS II; Hunter syndrome) is a rare, life-limiting lysosomal storage disease caused by deficient iduronate-2-sulfatase activity. Enzyme replacement therapy (ERT) with intravenous (IV) idursulfase can stabilize or improve many somatic manifestations, but there remains a need for further analysis of long-term treatment outcomes. Using data from patients with MPS II enrolled in the Hunter Outcome Survey (HOS), mixed modeling was performed to evaluate and predict the effects of IV idursulfase treatment on selected clinical parameters for up to 8 years following treatment start. The modeling population comprised male patients followed prospectively in HOS who had received IV idursulfase for at least 5 years and who had data available for two or more time points (at least one post-ERT). Age at ERT start and time since ERT start were included as covariates.

organization: University of North Carolina at Chapel Hill, 101 Manning Drive CB# 7487, Medical School Wing E Room 117, Chapel Hill, NC, 27599-7487, USA. [email protected].

DOI: 10.1186/s13023-021-02052-4

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