{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T21:16:28Z","timestamp":1793049388841,"version":"4.5.0"},"reference-count":38,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"3","content-domain":{"domain":["lww.com","ovid.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2010,3]]},"abstract":"\n Objective:<\/jats:title>\n To evaluate the reliability and validity of the PedsQL\u2122 3.0 Neuromuscular Module (NMM) in assessing health-related quality of life in the Duchenne muscular dystrophy (DMD) population for use as a secondary outcome measure in phase III clinical trials.<\/jats:p>\n <\/jats:sec>\n \n Background:<\/jats:title>\n DMD is the most common genetic form of muscular dystrophy in childhood. Clinical trials are underway to evaluate modalities of treatment. The NMM was developed based on interviews of patients with DMD and spinal muscular atrophy. To determine the PedsQL\u2122 reliability and validity, we administered the NMM to patients with DMD and their caregivers.<\/jats:p>\n <\/jats:sec>\n \n Design\/Methods:<\/jats:title>\n Boys 8 to 18 years old with DMD were recruited from a neuromuscular disease clinic. At baseline, the child and caregiver completed the NMM and the PedsQL\u2122 4.0 Generic Core Scales (GC). The NMM was repeated 2 to 6 weeks later. Reliability was assessed using Cronbach's coefficient alpha (internal consistency) and intraclass correlation (ICC) (test-retest consistency). Construct validity was assessed by comparing baseline child and caregiver NMM total scores with the GC Total Score, forced vital capacity, cardiac ejection fraction, and ambulatory status.<\/jats:p>\n <\/jats:sec>\n \n Results:<\/jats:title>\n \n Forty-four children and their caregivers completed the study. Internal consistency reliability of the total scale score of the NMM was demonstrated (Child \u03b1 = 0.85; Caregiver \u03b1 = 0.87). Test-retest reliability of the NMM was also demonstrated (Child ICC = 0.75,\n P<\/jats:italic>\n = 0.001; Caregiver ICC = 0.85,\n P<\/jats:italic>\n < 0.001). Validity of the total scale score of the NMM when compared with the GC Total Scale Score was supported (Child\n r<\/jats:italic>\n (41) = 0.63,\n P<\/jats:italic>\n < 0.001; Caregiver\n r<\/jats:italic>\n (42) = 0.64,\n P<\/jats:italic>\n < 0.001). Validity of the NMM compared with forced vital capacity was also supported (Child\n r<\/jats:italic>\n (38) = 0.35,\n P<\/jats:italic>\n = 0.032; Caregiver\n r<\/jats:italic>\n (39) = 0.41,\n P<\/jats:italic>\n = 0.01). The NMM parent-proxy-report and child self-report \u201cAbout My Child's Neuromuscular Disease\u201d scale was significantly related to wheelchair use (\n P<\/jats:italic>\n < 0.008 and 0.016, respectively); the GC \"Child Self-Report \u201cPhysical Health\u201d scale was also significantly related to wheelchair use (\n P<\/jats:italic>\n < 0.001). We were unable to conduct any analysis with ejection fraction because of the small number of children across all categories.\n <\/jats:p>\n <\/jats:sec>\n \n Conclusions:<\/jats:title>\n The PedsQL\u2122 NMM is a reliable measure of disease-specific health-related quality of life in the DMD population and may be used as an outcome measure in clinical trials.<\/jats:p>\n <\/jats:sec>","DOI":"10.1097\/cnd.0b013e3181c5053b","type":"journal-article","created":{"date-parts":[[2010,2,27]],"date-time":"2010-02-27T01:03:34Z","timestamp":1267232614000},"page":"97-109","update-policy":"https:\/\/doi.org\/10.1097\/lww.0000000000001000","source":"Crossref","is-referenced-by-count":118,"title":["The PedsQL\u2122 in Pediatric Patients with Duchenne Muscular Dystrophy: Feasibility, Reliability, and Validity of the Pediatric Quality of Life Inventory Neuromuscular Module and Generic Core Scales"],"prefix":"10.1097","volume":"11","author":[{"given":"Sarah E","family":"Davis","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda S","family":"Hynan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine A","family":"Limbers","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C Mariam","family":"Andersen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Medrith C","family":"Greene","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James W","family":"Varni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan T","family":"Iannaccone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","reference":[{"key":"R1-1-20240808","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1016\/0960-8966(91)90039-U","article-title":"Population frequencies of inherited neuromuscular diseases-a world survey.","volume":"1","author":"Emery","year":"1991","journal-title":"Neuromuscul Disord"},{"key":"R2-1-20240808","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1038\/322073a0","article-title":"Analysis of deletions in DNA from patients with Becker and Duchenne muscular dystrophy.","volume":"322","author":"Kunkel","year":"1986","journal-title":"Nature"},{"key":"R3-1-20240808","doi-asserted-by":"crossref","first-page":"719","DOI":"10.1016\/j.nmd.2007.07.001","article-title":"PTC124, nonsense mutations and Duchenne muscular dystrophy.","volume":"17","author":"Wilton","year":"2007","journal-title":"Neuromusc Disord"},{"key":"R4-1-20240808","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1212\/01.WNL.0000148485.00049.B7","article-title":"Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society.","volume":"64","author":"Moxley","year":"2005","journal-title":"Neurology"},{"key":"R5-1-20240808","doi-asserted-by":"crossref","first-page":"1977","DOI":"10.1378\/chest.07-0458","article-title":"American College of Chest Physicians consensus statement on the respiratory and related management of patients with Duchenne muscular dystrophy undergoing anesthesia or sedation.","volume":"132","author":"Birnkrant","year":"2007","journal-title":"Chest"},{"key":"R6-1-20240808","doi-asserted-by":"crossref","first-page":"522","DOI":"10.1046\/j.1524-4733.2003.65309.x","article-title":"Incorporating the patients perspective into drug development and communication: an ad hoc task force report of the Patient-Reported Outcomes (PRO) Harmonization Group meeting at the Food and Drug Administration, February 16, 2001.","volume":"6","author":"Acquadro","year":"2003","journal-title":"Value Health"},{"key":"R7-1-20240808","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1212\/01.wnl.0000251299.54608.13","article-title":"Outcome of noninvasive ventilation in children with neuromuscular disease.","volume":"68","author":"Young","year":"2007","journal-title":"Neurology"},{"key":"R8-1-20240808","doi-asserted-by":"crossref","first-page":"102","DOI":"10.1016\/j.pediatrneurol.2008.04.011","article-title":"Parental stress and quality of life in children with neuromuscular disease.","volume":"39","author":"Mah","year":"2008","journal-title":"Pediatr Neurol"},{"key":"R9-1-20240808","doi-asserted-by":"crossref","first-page":"101","DOI":"10.1097\/CND.0b013e3180315c99","article-title":"A randomized, placebo-controlled trial of creatine in children with spinal muscular atrophy.","volume":"8","author":"Wong","year":"2007","journal-title":"J Clin Neuromus Dis"},{"key":"R12-1-20240808","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1097\/00005650-199902000-00003","article-title":"The PedsQL: measurement model for the Pediatric Quality of Life Inventory.","volume":"37","author":"Varni","year":"1999","journal-title":"Med Care"},{"key":"R13-1-20240808","doi-asserted-by":"crossref","first-page":"S217","DOI":"10.1097\/00005650-198903001-00018","article-title":"Generic and disease-specific measures in assessing health status and quality of life.","volume":"27","author":"Patrick","year":"1989","journal-title":"Med Care"},{"key":"R14-1-20240808","doi-asserted-by":"crossref","first-page":"983","DOI":"10.1093\/jpepsy\/jsn038","article-title":"Evidence-based assessment of health-related quality of life and functional impairment in pediatric psychology.","volume":"33","author":"Palermo","year":"2008","journal-title":"J Pediatr Psychol"},{"key":"R15-1-20240808","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1017\/S001216220600096X","article-title":"The PedsQL in pediatric cerebral palsy: Reliability, validity, and sensitivity of the Generic Core Scales and Cerebral Palsy Module.","volume":"48","author":"Varni","year":"2006","journal-title":"Dev Med Child Neurol"},{"key":"R16-1-20240808","doi-asserted-by":"crossref","first-page":"631","DOI":"10.2337\/diacare.26.3.631","article-title":"The PedsQL in Type 1 and Type 2 diabetes: Reliability and validity of the Pediatric Quality of Life Inventory Generic Core Scales and Type 1 Diabetes Module.","volume":"26","author":"Varni","year":"2003","journal-title":"Diabetes Care"},{"key":"R17-1-20240808","doi-asserted-by":"crossref","first-page":"2090","DOI":"10.1002\/cncr.10428","article-title":"The PedsQL in pediatric cancer: Reliability and validity of the Pediatric Quality of Life Inventory Generic Core Scales, Multidimensional Fatigue Scale, and Cancer Module.","volume":"94","author":"Varni","year":"2002","journal-title":"Cancer"},{"key":"R18-1-20240808","doi-asserted-by":"crossref","first-page":"297","DOI":"10.1023\/B:JOBM.0000028500.53608.2c","article-title":"The PedsQL in pediatric asthma: Reliability and validity of the Pediatric Quality of Life Inventory Generic Core Scales and Asthma Module.","volume":"27","author":"Varni","year":"2004","journal-title":"J Behav Med"},{"key":"R19-1-20240808","doi-asserted-by":"crossref","first-page":"714","DOI":"10.1002\/art.10095","article-title":"The PedsQL in pediatric rheumatology: Reliability, validity, and responsiveness of the Pediatric Quality of Life Inventory Generic Core Scales and Rheumatology Module.","volume":"46","author":"Varni","year":"2002","journal-title":"Arthritis Rheum"},{"key":"R20-1-20240808","doi-asserted-by":"crossref","first-page":"287","DOI":"10.1002\/pbc.21026","article-title":"The PedsQL Brain Tumor Module: Initial reliability and validity.","volume":"49","author":"Palmer","year":"2007","journal-title":"Pediatr Blood Cancer"},{"key":"R21-1-20240808","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1053\/j.ajkd.2007.09.021","article-title":"Measuring health-related quality of life in children with ESRD: Performance of the Generic and ESRD-Specific Instrument of the Pediatric Quality of Life Inventory (PedsQL ).","volume":"51","author":"Goldstein","year":"2008","journal-title":"Am J Kidney Dis"},{"key":"R22-1-20240808","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1016\/S1058-9813(03)00087-0","article-title":"The Pediatric Quality of Life Inventory in children with heart disease.","volume":"18","author":"Uzark","year":"2003","journal-title":"Prog Pediatr Cardiol"},{"key":"R23-1-20240808","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/1477-7525-5-1","article-title":"How young can children reliably and validly self-report their health-related quality of life? An analysis of 8,591 children across age subgroups with the PedsQL 4.0 Generic Core Scales.","volume":"5","author":"Varni","year":"2007","journal-title":"Health Qual Life Outcome"},{"key":"R24-1-20240808","doi-asserted-by":"crossref","first-page":"800","DOI":"10.1097\/00005650-200108000-00006","article-title":"PedsQL 4.0: Reliability and validity of the Pediatric Quality of Life Inventory Version 4.0 Generic Core Scales in healthy and patient populations.","volume":"39","author":"Varni","year":"2001","journal-title":"Med Care"},{"key":"R25-1-20240808","doi-asserted-by":"crossref","first-page":"843","DOI":"10.1016\/j.pcl.2009.05.016","article-title":"The pediatric quality of life inventory: measuring pediatric health-related quality of life from the perspective of children and their parents.","volume":"56","author":"Varni","year":"2009","journal-title":"Pediatr Clin North Am"},{"key":"R26-1-20240808","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1367\/1539-4409(2003)003<0329:TPAAPP>2.0.CO;2","article-title":"The PedsQL 4.0 as a pediatric population health measure: Feasibility, reliability, and validity.","volume":"3","author":"Varni","year":"2003","journal-title":"Ambul Pediatr"},{"key":"R30-1-20240808","doi-asserted-by":"crossref","first-page":"40","DOI":"10.1097\/00005650-199401000-00004","article-title":"The MOS 36-item short-form health survey (SF-36): III. Tests of data quality, scaling assumptions, and reliability across diverse patient groups.","volume":"32","author":"McHorney","year":"1994","journal-title":"Med Care"},{"key":"R31-1-20240808","doi-asserted-by":"crossref","first-page":"297","DOI":"10.1007\/BF02310555","article-title":"Coefficient alpha and the internal structure of tests.","volume":"16","author":"Cronbach","year":"1951","journal-title":"Psychometrika"},{"key":"R34-1-20240808","doi-asserted-by":"crossref","first-page":"219","DOI":"10.1097\/00004356-200509000-00004","article-title":"Preliminary validation of the Chinese version of the Pediatric Quality of Life Inventory.","volume":"28","author":"Chan","year":"2005","journal-title":"Int J Rehabil Res"},{"key":"R35-1-20240808","doi-asserted-by":"crossref","first-page":"3","DOI":"10.2466\/pr0.1966.19.1.3","article-title":"The intraclass correlation coefficient as a measure of reliability.","volume":"19","author":"Bartko","year":"1966","journal-title":"Psychol Rep"},{"key":"R36-1-20240808","doi-asserted-by":"crossref","first-page":"1041","DOI":"10.1023\/A:1016647407161","article-title":"Perception of quality of life by patients, partners and treating physicians.","volume":"9","author":"Wilson","year":"2001","journal-title":"Qual Life Res"},{"key":"R39-1-20240808","first-page":"139","article-title":"Factor analysis, causal indicators and quality of life.","volume":"6","author":"Fayers","year":"1997","journal-title":"Qual Life Res"},{"key":"R40-1-20240808","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1037\/1082-989X.1.1.30","article-title":"Forming inferences about some Intraclass Correlation Coefficients.","volume":"1","author":"McGraw","year":"1996","journal-title":"Psychol Methods"},{"key":"R41-1-20240808","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/1477-7525-4-58","article-title":"Factors influencing agreement between child self-report and parent proxy-reports on the Pediatric Quality of Life Inventory 4.0 (PedsQL) Generic Core Scales.","volume":"4","author":"Cremeens","year":"2006","journal-title":"Health Qual Life Outcomes"},{"key":"R43-1-20240808","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/BF02289400","article-title":"Coefficient alpha and the reliability of composite measurements.","volume":"32","author":"Novick","year":"1967","journal-title":"Psychometrika"},{"key":"R44-1-20240808","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1037\/0033-2909.101.2.213","article-title":"Child\/adolescent behavioral and emotional problems: implications of cross-informant correlations for situational specificity.","volume":"101","author":"Achenbach","year":"1987","journal-title":"Psychol Bull"},{"key":"R45-1-20240808","doi-asserted-by":"crossref","first-page":"743","DOI":"10.1016\/0895-4356(92)90052-O","article-title":"The role of health care providers and significant others in evaluating the quality of life of patients with chronic disease: a review.","volume":"45","author":"Sprangers","year":"1992","journal-title":"J Clin Epidemiol"},{"key":"R46-1-20240808","doi-asserted-by":"crossref","first-page":"347","DOI":"10.1023\/A:1012253723272","article-title":"Can parents rate their childs health-related quality of life?: Results from a systematic review.","volume":"10","author":"Eiser","year":"2001","journal-title":"Qual Life Res"},{"key":"R47-1-20240808","doi-asserted-by":"crossref","first-page":"895","DOI":"10.1007\/s11136-008-9350-5","article-title":"Parent-child agreement across child health-related quality of life instruments: a review of the literature.","volume":"17","author":"Upton","year":"2008","journal-title":"Qual Life Res"},{"key":"R48-1-20240808","doi-asserted-by":"crossref","first-page":"717","DOI":"10.1023\/B:QURE.0000021686.47079.0d","article-title":"Statistical considerations for use of composite health-related quality of life scores in randomized trials.","volume":"13","author":"Vickers","year":"2004","journal-title":"Qual Life Res"}],"container-title":["Journal of Clinical Neuromuscular Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.ovid.com\/00131402-201003000-00001","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,6]],"date-time":"2026-08-06T18:16:17Z","timestamp":1786040177000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.ovid.com\/00131402-201003000-00001"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2010,3]]},"references-count":38,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2010]]}},"URL":"https:\/\/doi.org\/10.1097\/cnd.0b013e3181c5053b","relation":{},"ISSN":["1522-0443"],"issn-type":[{"value":"1522-0443","type":"print"}],"subject":[],"published":{"date-parts":[[2010,3]]}}}