[en] AIM: To develop a patient-reported outcome measure (PROM) assessing upper limb function related to activities of daily living (ADL) that cannot be observed in a clinical setting, specifically for patients with Duchenne muscular dystrophy (DMD) across a wide age range, applicable in the different stages of the disease. METHOD: The developmental process was based on US Food and Drug Administration guidelines. This included item generation from a systematic review of existing tools and expert opinion on task difficulty and relevance, involving individuals with DMD. Cultural aspects affecting ADL were taken into consideration to make this tool applicable to the broad DMD community. Items were selected in relation to a conceptual framework reflecting disease progression covering the full range of upper limb function across different ADL domains. RESULTS: After pilot testing and iterative Rasch analyses, redundant or clinically irrelevant items were removed. The final questionnaire consists of 32 items covering four domains of ADL (food, self-care, household and environment, leisure and communication). Test-retest reliability was excellent. INTERPRETATION: A DMD-specific upper limb PROM was developed on the basis of clinical relevance and psychometric robustness. Its main purpose is to document the patient self-reported natural history of DMD and assess the efficacy of interventions.
Disciplines :
Pediatrics Neurology
Author, co-author :
Klingels, K.
Mayhew, A. G.
Mazzone, E. S.
Duong, T.
Decostre, V.
Werlauff, U.
Vroom, E.
Mercuri, E.
Goemans, N. M.
Upper Limb Clinical Outcome Group
Other collaborator :
Servais, Laurent ; Université de Liège - ULiège > Département des sciences cliniques > Neuropédiatrie
Language :
English
Title :
Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM.
Mazzone E, Martinelli D, Berardinelli A, et al. North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy. Neuromuscul Disord 2010; 20: 712–16.
Mayhew A, Cano S, Scott E, Eagle M, Bushby K, Muntoni F; North Star Clinical Network for Paediatric Neuromuscular Disease. Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy. Dev Med Child Neurol 2011; 53: 535–42.
McDonald CM, Henricson EK, Han JJ, et al. The 6-minute walk test in Duchenne/Becker muscular dystrophy: longitudinal observations. Muscle Nerve 2010; 42: 966–74.
Seferian AM, Moraux A, Annoussamy M, et al. Upper limb strength and function changes during a one-year follow-up in non-ambulant patients with Duchenne muscular dystrophy: an observational multicenter trial. PLoS ONE 2015; 10: e0113999.
Janssen MM, Bergsma A, Geurts AC, de Groot IJ. Patterns of decline in upper limb function of boys and men with DMD: an international survey. J Neurol 2014; 261: 1269–88.
Mercuri E, McDonald C, Mayhew A, et al. International workshop on assessment of upper limb function in Duchenne Muscular Dystrophy: Rome, 15–16 February 2012. Neuromuscul Disord 2012; 22: 1025–28.
Mayhew A, Mazzone ES, Eagle M, et al. Development of the performance of the upper limb module for Duchenne muscular dystrophy. Dev Med Child Neurol 2013; 55: 1038–45.
Pane M, Mazzone ES, Fanelli L, et al. Reliability of the performance of upper limb assessment in Duchenne muscular dystrophy. Neuromuscul Disord 2014; 24: 201–06.
US Food ande 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. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf (accessed 9 August 2016).
Steffensen B, Hyde S, Lyager S, Mattsson E. Validity of the EK scale: a functional assessment of non-ambulatory individuals with Duchenne muscular dystrophy or spinal muscular atrophy. Physiother Res Int 2001; 63: 119–34.
Vandervelde L, Van den Bergh PY, Goemans N, Thonnard JL. ACTIVLIM: a Rasch-built measure of activity limitations in children and adults with neuromuscular disorders. Neuromuscul Disord 2007; 17: 459–69.
Vandervelde L, Van den Bergh PY, Penta M, Thonnard JL. Validation of the ABILHAND questionnaire to measure manual ability in children and adults with neuromuscular disorders. J Neurol Neurosurg Psychiatry 2010; 81: 506–12.
Lue YJ, Su CY, Yang RC, et al. Development and validation of a muscular dystrophy-specific functional rating scale. Clin Rehabil 2006; 20: 804–17.
Landfeldt E, Mayhew A, Eagle M, et al. Development and psychometric analysis of the Duchenne muscular dystrophy Functional Ability Self-Assessment Tool (DMDSAT). Neuromuscul Disord 2015; 25: 937–44.
Mazzone ES, Vasco G, Palermo C, et al. A critical review of functional assessment tools for upper limbs in Duchenne muscular dystrophy. Dev Med Child Neurol 2012; 54: 879–85.
Andrich D. Rasch Models for Measurement. Beverly Hills, CA: Sage Publications, 1988.
Tennant A, McKenna SP, Hagell P. Application of Rasch analysis in the development and application of quality of life instruments. Value Health 2004; 7: S22–26.
Van de Winkel A, Feys H, Lincoln N, De Weerdt W. Assessment of arm function in stroke patients: Rivermead Motor Assessment arm section revised with Rasch analysis. Clin Rehabil 2004; 21: 471–79.
Brooke MH, Griggs RC, Mendell JR, et al. Clinical trial in Duchenne dystrophy. I. The design of the protocol. Muscle Nerve 1981; 4: 186–97.
Chambers CT, Johnston C. Developmental differences in children's use of rating scales. J Pediatr Psychol 2002; 27: 27–36.
Tennant A, Badley EM, Sullivan M. Investigating the proxy effect and the saliency principle in household based postal questionnaires. J Epidemiol Community Health 1991; 45: 312–16.
