{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T21:16:47Z","timestamp":1793049407825,"version":"4.5.0"},"reference-count":48,"publisher":"SAGE Publications","issue":"5","license":[{"start":{"date-parts":[[2020,2,3]],"date-time":"2020-02-03T00:00:00Z","timestamp":1580688000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"DOI":"10.13039\/100007721","name":"Cure SMA","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007721","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["J Child Neurol"],"published-print":{"date-parts":[[2020,4]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Spinal muscular atrophy is an autosomal-recessive, progressive neuromuscular disease associated with extensive morbidity. Children with spinal muscular atrophy have potentially increased life spans due to improved nutrition, respiratory support, and novel pharmaceuticals.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives:<\/jats:title>\n                    <jats:p>To report on the quality of life and family experience for children with spinal muscular atrophy with attentiveness to patient- and proxy-concordance and to stratify quality of life reports by spinal muscular atrophy type and medical interventions.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>A prospective, crossover survey study inclusive of 58 children (26 spinal muscular atrophy type I, 23 type II, 9 type III) and their family caregivers at a free-standing Midwestern children\u2019s hospital. Twenty-eight families completed the 25-item PedsQL 3.0 Neuromuscular Module. Forty-four participants completed the 36-item PedsQL Family Impact Module and 47 completed the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaire.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>The PedsQL Family Impact Module demonstrated significant differences between spinal muscular atrophy types I and II in functioning domains including physical, emotional, social, and family relations ( P &lt; .03). Child self-report and proxy report surveys demonstrated significant differences between spinal muscular atrophy types in the communication domains ( P &lt; .003). Children self-reported their quality of life higher than proxy report of child quality of life. Gastrostomy tube ( P = .001) and ventilation support ( P = .029) impacted proxy-reported quality of life perspectives, whereas nusinersen use did not. Spinal surgery was associated with improved parental quality of life and family impact ( P &lt; .03).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>The measurement and monitoring of quality of life for children with spinal muscular atrophy and their families represents an implementable priority for care teams.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/0883073819900463","type":"journal-article","created":{"date-parts":[[2020,2,3]],"date-time":"2020-02-03T05:37:10Z","timestamp":1580708230000},"page":"322-330","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":18,"title":["A Prospective, Crossover Survey Study of Child- and Proxy-Reported Quality of Life According to Spinal Muscular Atrophy Type and Medical Interventions"],"prefix":"10.1177","volume":"35","author":[{"given":"Meaghann S.","family":"Weaver","sequence":"first","affiliation":[{"name":"Children\u2019s Hospital of Omaha, University of Nebraska Medical Center, Omaha, NE, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rewais","family":"Hanna","sequence":"additional","affiliation":[{"name":"School of Medicine and Public Health, University of Wisconsin\u2013Madison, Madison, WI, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Scott","family":"Hetzel","sequence":"additional","affiliation":[{"name":"School of Medicine and Public Health, University of Wisconsin\u2013Madison, Madison, WI, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karen","family":"Patterson","sequence":"additional","affiliation":[{"name":"School of Medicine and Public Health, University of Wisconsin\u2013Madison, Madison, WI, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alice","family":"Yuroff","sequence":"additional","affiliation":[{"name":"School of Medicine and Public Health, University of Wisconsin\u2013Madison, Madison, WI, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Sund","sequence":"additional","affiliation":[{"name":"School of Medicine and Public Health, University of Wisconsin\u2013Madison, Madison, WI, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Meredith","family":"Schultz","sequence":"additional","affiliation":[{"name":"AveXis, Inc, Bannockburn, IL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary","family":"Schroth","sequence":"additional","affiliation":[{"name":"Cure SMA, Chicago, IL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew A.","family":"Halanski","sequence":"additional","affiliation":[{"name":"Children\u2019s Hospital of Omaha, University of Nebraska Medical Center, Omaha, NE, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2020,2,3]]},"reference":[{"key":"bibr1-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(12)70061-3"},{"key":"bibr2-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)60921-6"},{"key":"bibr3-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1055\/s-0037-1603517"},{"key":"bibr4-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1177\/0883073807305662"},{"key":"bibr5-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.004"},{"key":"bibr6-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.005"},{"key":"bibr7-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1097\/PCC.0b013e3182388ad1"},{"key":"bibr8-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejpn.2016.08.008"},{"key":"bibr9-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2016.04.062"},{"key":"bibr10-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24497"},{"key":"bibr11-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.0417-15.2015"},{"key":"bibr12-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2017.52"},{"key":"bibr13-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1038\/nrneurol.2018.4"},{"key":"bibr14-0883073819900463","doi-asserted-by":"publisher","DOI":"10.1177\/104990910201900109"},{"issue":"9","key":"bibr15-0883073819900463","first-page":"S218","volume":"13","author":"Khanna D","year":"2007","journal-title":"Am J Manag Care"},{"key":"bibr16-0883073819900463","first-page":"354","volume":"17","author":"What quality of life? 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