caa a22 4500 605544743 CHVBK 20210128100932.0 cr unu---uuuuu 210128e20150801xx s 000 0 eng 10.1007/s10897-014-9791-7 doi (NATIONALLICENCE)springer-10.1007/s10897-014-9791-7 Health-Related Quality of Life in Patients with MPS II [Elektronische Daten] [Mary Needham, Wendy Packman, Natasha Quinn, Maxwell Rappoport, Christa Aoki, Alan Bostrom, Matthew Cordova, Sandra Macias, Cynthia Morgan, Seymour Packman] 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. National Society of Genetic Counselors, Inc., 2014 MPS II nationallicence Hunter syndrome nationallicence Quality of life nationallicence HRQOL nationallicence Psychology nationallicence Needham Mary Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut Packman Wendy Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut Quinn Natasha Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut Rappoport Maxwell Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut Aoki Christa Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut Bostrom Alan University of California, San Francisco, USA aut Cordova Matthew Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut Macias Sandra Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut Morgan Cynthia University of California, San Francisco, USA aut Packman Seymour University of California, San Francisco, USA aut Journal of Genetic Counseling Springer US; http://www.springer-ny.com 24/4(2015-08-01), 635-644 1059-7700 24:4<635 2015 24 10897 https://doi.org/10.1007/s10897-014-9791-7 text/html Onlinezugriff via DOI BK010053 XK010053 XK010000 Metadata rights reserved Springer special CC-BY-NC licence nationallicence 1 research-article jats NATIONALLICENCE NATIONALLICENCE NL-springer NATIONALLICENCE 856 40 https://doi.org/10.1007/s10897-014-9791-7 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Needham Mary Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut NATIONALLICENCE 700 1- Packman Wendy Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut NATIONALLICENCE 700 1- Quinn Natasha Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut NATIONALLICENCE 700 1- Rappoport Maxwell Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut NATIONALLICENCE 700 1- Aoki Christa Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut NATIONALLICENCE 700 1- Bostrom Alan University of California, San Francisco, USA aut NATIONALLICENCE 700 1- Cordova Matthew Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut NATIONALLICENCE 700 1- Macias Sandra Pacific Graduate School of Psychology at Palo Alto University, University of California, San Francisco, USA aut NATIONALLICENCE 700 1- Morgan Cynthia University of California, San Francisco, USA aut NATIONALLICENCE 700 1- Packman Seymour University of California, San Francisco, USA aut NATIONALLICENCE 773 0- Journal of Genetic Counseling Springer US; http://www.springer-ny.com 24/4(2015-08-01), 635-644 1059-7700 24:4<635 2015 24 10897