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01-08-2015 | Original Research

Health-Related Quality of Life in Patients with MPS II

Authors: Mary Needham, Wendy Packman, Natasha Quinn, Maxwell Rappoport, Christa Aoki, Alan Bostrom, Matthew Cordova, Sandra Macias, Cynthia Morgan, Seymour Packman

Published in: Journal of Genetic Counseling | Issue 4/2015

Abstract

Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a chronic and progressive X-linked lysosomal disease that mainly affects males. The National MPS Society (2013) reports that MPS II affects 1 in 100,000 to 1 in 150,000 males worldwide. Two distinct forms of the disease are based on age of onset and clinical course: attenuated and severe. MPS II affects many organ systems including the nervous, cardiovascular, gastrointestinal and respiratory systems. Clinical manifestations can include progressive hearing loss, mental impairment, and enlarged liver and spleen. This study focuses on the health-related quality of life of individuals (HRQOL) with MPS II as measured by the parent and self-report versions of the Pediatric Quality of Life Inventory (PedsQL™). Both parents of patients with MPS II as well as patients themselves reported lower scores on all domains of the PedsQL™ (physical, emotional, social and school functioning) indicating that children with MPS II have an overall lower HRQOL when compared to a healthy sample. When compared with patients with other chronic illnesses (cancer, MSUD, galactosemia,), the MPS II sample had significantly lower scores on a number of PedsQL™ scales, suggesting an overall lower HRQOL. No significant relationships were found using scores from parent or self report PedsQL™ measures and length of time on ERT.

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Title: Health-Related Quality of Life in Patients with MPS II
Authors: Mary Needham
Wendy Packman
Natasha Quinn
Maxwell Rappoport
Christa Aoki
Alan Bostrom
Matthew Cordova
Sandra Macias
Cynthia Morgan
Seymour Packman
Publication date: 01-08-2015
Publisher: Springer US
Published in: Journal of Genetic Counseling / Issue 4/2015
Print ISSN: 1059-7700
Electronic ISSN: 1573-3599
DOI: https://doi.org/10.1007/s10897-014-9791-7

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Health-Related Quality of Life in Patients with MPS II

Abstract

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