Relationship Between Clinical Outcome Measures and Parent Proxy Reports of Health-Related Quality of Life in Ambulatory Children With Duchenne Muscular Dystrophy
Restricted accessOtherFirst published online September, 2010
Relationship Between Clinical Outcome Measures and Parent Proxy Reports of Health-Related Quality of Life in Ambulatory Children With Duchenne Muscular Dystrophy
In Duchenne muscular dystrophy, data directly linking changes in clinical outcome measures to patient-perceived well-being are lacking. This study evaluated the relationship between clinical outcome measures used in clinical trials of ambulatory Duchenne muscular dystrophy (Vignos functional grade, quantitative knee extension strength, timed functional performance measures, and gait velocity) and 2 health-related quality of life measures—the Pediatric Outcomes Data Collection Instrument and Pediatric Quality of Life Inventory—in 52 ambulatory Duchenne muscular dystrophy subjects and 36 controls. Those with the disease showed significant decrements in parent proxy-reported health-related quality of life measures versus controls across all domains. The Pediatric Outcomes Data Collection Instrument transfers/basic mobility and sports/ physical function and the Pediatric Quality of Life Inventory physical functioning domains had significant associations with age (and hence disease progression) and traditional clinical outcome measures employed in clinical trials of ambulatory boys with Duchenne muscular dystrophy. Selected domains of the Pediatric Outcomes Data Collection Instrument and generic Pediatric Quality of Life Inventory are potential patient-reported outcome measures for clinical trials in ambulatory individuals with the disease.
Cossu G., Sampaolesi M.New therapies for Duchenne muscular dystrophy: challenges, prospects and clinical trials. Trends Mol Med. 2007;13(12):520-526.
2.
NIH Workshop on Translational Research in Muscular Dystrophy; June 25-27, 2007; Silver Spring, MD.
3.
Muscular Dystrophy Association.Clinical Research Coalition Meeting; June 11-12, 2004; Tucson, AZ.
4.
NIH Department of Health and Human Services.Action plan for the muscular dystrophies. Plan developed by the Muscular Dystrophy Coordinating Committee Scientific Working Group; August 16-17, 2005; Bethesda, MD. Approved by the Muscular Dystrophy Coordinating Committee, December 2005.
5.
World Health Organization.Constitution of the World Health Organization: Basic Document . Geneva, Switzerland: World Health Organization;1948.
6.
US Food and Drug Administration.Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Rockville, MD: US Food and Drug Administration; 2009. Fed Reg. 2009;74(235): 65132-65133. Available at: Federal Register Online via GPO Access, wais.access.gpo.gov (DOCID:fr09de09-66).
7.
Mendell JR, Csimma C., McDonald CM, et al. Challenges in drug development for muscle disease: a stakeholders’ meeting. Muscle Nerve. 2007;35(1):8-16.
8.
Acquadro C., Berzon R., Dubois D., et al. Incorporating the patient’s 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. Value Health. 2003;6: 522-531.
9.
Young HK, Lowe A., Fitzgerald DA, et al. Outcome of noninvasive ventilation in children with neuromuscular disease. Neurology. 2007;68:198-201.
10.
Mah JK, Thannhauser JE, Kolski H., et al. Parental stress and quality of life in children with neuromuscular disease. Pediatr Neurol. 2008;39:102-107.
11.
Wong BL, Hynan LS, Iannaccone STA randomized, placebo-controlled trial of creatine in children with spinal muscular atrophy. J Clin Neuromuscul Dis. 2007;8:101-110.
12.
Varni JW, Seid M., Rode CAThe PedsQL: measurement model for the Pediatric Quality of Life Inventory . Med Care. 1999; 37(2):126-139.
13.
Daltroy LH, Liang MH, Fossel AH, Goldberg MJThe POSNA pediatric musculoskeletal functional health questionnaire: report on reliability, validity, and sensitivity to change. Pediatric Outcomes Instrument Development Group. Pediatric Orthopaedic Society of North America. J Pediatr Orthop. 1998;18(5): 561-571.
14.
Vincent KA, Carr AJ, Walburn J., Scott DL, Rose MRConstruction and validation of a quality of life questionnaire for neuromuscular disease (INQoL). Neurology. 2007;68(13):1051-1057.
15.
Iannaccone ST, Hynan LS, American Spinal Muscular Atrophy Randomized Trials (AmSMART) Group.Reliability of 4 outcome measures in pediatric spinal muscular atrophy. Arch Neurol. 2003;60(8):1130-1136.
16.
Iannaccone ST, Hynan LS, Morton A., et al. The PedsQL in pediatric patients with spinal muscular atrophy: feasibility, reliability, and validity of the Pediatric Quality of Life Inventory generic core scales and neuromuscular module. Neuromuscul Disord.2009;19(12):805-812.
17.
Davis SE, Hynan LS, Limbers CA, et al. The PedsQL in pediatric patients with Duchenne muscular dystrophy: feasibility, reliability, and validity of the Pediatric Quality of Life Inventory neuromuscular module and generic core scales. J Clin Neuromuscul Dis.2010;11(3):97-109.
18.
Miller D., Nowinski C., Victorson D., et al. The NeuroQOL Project: establishing research priorities through qualitative research and consensus development. Qual Life Res. 2005;14(9):2031.
19.
Nowinski CJ, Victorson DE, Miller D., et al. Clinician attitudes toward quality of life measures for neurological disorders. Neurology. 2006;66(suppl 2):A235.
20.
Cella D., Victorson D., Nowinski C., Peterman A., Miller DMThe NeuroQOL project: using multiple methods to develop a HRQOL measurement platform to be used in clinical research across neurological conditions. Qual Life Res. 2006;A-14:1353.
21.
Miller D., Peterman AH, Victorson D., et al. Evaluating personality and behavior changes following stroke: The development of a disease targeted NeuroQOL scale. Quality of Life Res. 2005; 14(suppl):2031.
22.
Perez L., Huang J., Jansky E., et al. Using focus groups to inform the Neuro-QOL measurement tool: exploring patient-centered, health-related quality of life concepts across neurological conditions. J Neurosci Nurs. 2007;39(6):342-353.
23.
World Health Organization.International classification of functioning, disability and health. Available at: http://www.who.int/classifications/icf/en .Accessed April 27, 2010.
24.
Brooke MH, Griggs RC, Mendell JR, Fenichel GM, Shumate JB, Pellegrino RJClinical trial in Duchenne dystrophy, I: the design of the protocol. Muscle Nerve. 1981;4(3):186-197.
25.
Brooke MH, Fenichel GM, Griggs RC, et al. Clinical investigation of Duchenne muscular dystrophy: interesting results in a trial of prednisone. Arch Neurol. 1987;44(8):812-817.
26.
Mendell JR, Moxley RT, Griggs RC, et al. Randomized, double-blind six-month trial of prednisone in Duchenne’s muscular dystrophy. N Engl J Med. 1989;320(24):1592-1597.
27.
Griggs RC, Moxley RT III, Mendell JR, et al. Prednisone in Duchenne dystrophy: a randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group. Arch Neurol. 1991;48(4):383-388.
28.
Fenichel GM , Florence JM, Pestronk A., et al. Long-term benefit from prednisone therapy in Duchenne muscular dystrophy. Neurology. 1991;41(12):1874-1877.
29.
Fenichel GM , Mendell JR, Moxley RT III, et al. A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy. Arch Neurol. 1991;48(6):575-579.
30.
Escolar DM, Henricson EK, Mayhew J., et al. Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children. Muscle Nerve. 2001;24(6):787-793.
31.
Buyse GM, Goemans N., Henricson E., et al. CINRG pilot trial of oxatomide in steroid-naive Duchenne muscular dystrophy. Eur J Paediatr Neurol. 2007;11(6):337-340.
32.
Escolar DM, Buyse G., Henricson E., et al. CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy. Ann Neurol. 2005;58(1):151-155.
33.
McDonald CM , Henricson EK, Han JJ, et al. The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve.2010;41(4):500-510.
34.
Varni JW, Seid M., Kurtin PSPedsQLTM 4.0: reliability and validity of the Pediatric Quality of Life InventoryTM version 4.0 generic core scales in healthy and patient populations. Med Care. 2001;39(8):800-812.
35.
Varni JW, Seid M., Knight TS, et al. The PedsQL 4.0 generic core scales: sensitivity, responsiveness, and impact on clinical decision-making. J Behav Med. 2002;25(2):175-193.
36.
Varni JW, Burwinkle TM, Seid M., Skarr D.The PedsQL 4.0 as a pediatric population health measure: feasibility, reliability, and validity. Ambul Pediatr. 2003;3(6):329-341.
37.
Varni JW, Limbers CA, Burwinkle TMHow 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 PedsQLTM 4.0 generic core scales. Health Qual Life Outcomes . 2007;3;5:1.
38.
Varni JW, Limbers CAThe Pediatric Quality of Life Inventory: measuring pediatric health-related quality of life from the perspective of children and their parents. Pediatr Clin North Am.2009;56(4):843-863.
39.
Varni JW, Limbers CAThe PedsQL 4.0 generic core scales young adult version: feasibility, reliability and validity in a university student population. J Health Psychol.2009;14(4):611-622.
40.
Hunsaker FG , Cioffi DA, Amadio PC, et al. The American Academy of Orthopaedic Surgeons outcomes instruments: normative values from the general population . J Bone Joint Surg Am. 2002;84(2):208-215.
41.
Haynes RJ, Sullivan E.The Pediatric Orthopaedic Society of North America pediatric orthopaedic functional health questionnaire: an analysis of normals. J Pediatr Orthop . 2001;21(5):619-621.
42.
Pencharz J. , Young NL, Owen JL, Wright JGComparison of three outcomes instruments in children. J Pediatr Orthop. 2001;21(4): 425-432.
43.
Barnes D., Linton JL, Sullivan E., et al. Pediatric Outcomes Data Collection Instrument scores in ambulatory children with cerebral palsy: an analysis by age groups and severity level . J Pediatr Orthop. 2008;28(1):97-102.
44.
Archibald KC , Vignos PJ Jr.A study of contractures in muscular dystrophy . Arch Phys Med Rehabil. 1959;40(4):150-157.
45.
Vignos PJ Jr , Archibald KCMaintenance of ambulation in childhood muscular dystrophy. J Chronic Dis. 1960;12:273-290.
46.
McDonald CM , Abresch RT, Carter GT, et al. Profiles of neuromuscular diseases: Duchenne muscular dystrophy. Am J Phys Med Rehabil. 1995;74(5 suppl):S70-S92.
47.
Mayhew JE, Florence JM, Mayhew TP, et al. Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy. Muscle Nerve. 2007;35(1):36-42.
48.
Wagner KR, Fleckenstein JL, Amato AA, et al. A phase I/II trial of MYO-029 in adult subjects with muscular dystrophy. Ann Neurol. 2008;63(5):561-571.
49.
Leibowitz D. , Dubowitz V.Intellect and behavior in Duchenne muscular dystrophy. Dev Med Child Neurol. 1981;23(5):577-590.
50.
Sutherland DH, Olshen R., Cooper L., et al. The pathomechanics of gait in Duchenne muscular dystrophy. Dev Med Child Neurol. 1981;23(1):3-22.
51.
D’Angelo MG, Berti M., Piccinini L., et al. Gait pattern in Duchenne muscular dystrophy. Gait Posture. 2009;29(1):36-41.
52.
Palisano RJ , Hanna SE, Rosenbaum PL, et al. Validation of a model of gross motor function for children with cerebral palsy. Phys Ther. 2000;80(10):974-985.
53.
Abresch RT, McDonald DA, Widman LM, et al. Impact of spinal cord dysfunction and obesity on the health-related quality of life of children and adolescents. J Spinal Cord Med. 2007;30(suppl 1): S112-S118.
National Institute of Neurological Disorders and Stroke. Neuro-QOL Web site . Available at: http://www.NeuroQOL.org/default.aspx . Accessed April 27, 2010.
56.
Cella D., and PROMIS Cooperative Group.The Patient-Reported Outcomes Measurement Information System: progress of an NIH roadmap cooperative group during its first two years. Med Care. 2007;45:S3-S11.
57.
Calhoun CL, Haley SM, Riley A., Vogel LC, McDonald CM, Mulcahey MJDevelopment of items designed to evaluate activity performance and participation in children and adolescents with spinal cord injury. Int J Pediatr. 2009; 2009:854904.
