Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM

The Upper Limb Clinical Outcome Group

Research output: Contribution to journalArticle

14 Citations (Scopus)

Abstract

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.

Original languageEnglish (US)
Pages (from-to)224-231
Number of pages8
JournalDevelopmental Medicine and Child Neurology
Volume59
Issue number2
DOIs
StatePublished - Feb 1 2017

Fingerprint

Duchenne Muscular Dystrophy
Upper Extremity
Activities of Daily Living
Leisure Activities
Expert Testimony
United States Food and Drug Administration
Self Care
Psychometrics
Disease Progression
Communication
Patient Reported Outcome Measures
Guidelines
Food

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Developmental Neuroscience
  • Clinical Neurology

Cite this

Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy : DMD Upper Limb PROM. / The Upper Limb Clinical Outcome Group.

In: Developmental Medicine and Child Neurology, Vol. 59, No. 2, 01.02.2017, p. 224-231.

Research output: Contribution to journalArticle

@article{3a08f81106d247298e73731bdc18f420,
title = "Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM",
abstract = "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.",
author = "{The Upper Limb Clinical Outcome Group} and K. Klingels and Mayhew, {A. G.} and Mazzone, {E. S.} and T. Duong and V. Decostre and U. Werlauff and E. Vroom and E. Mercuri and Goemans, {N. M.} and M. Eagle and {De Groot}, I. and M. Main and S. Messina and G. Campion and L. Servais and {Van den Hauwe}, M. and E. Es and M. Pane and F. Buccella and J. Kuijer and F. Ceradini and E. Henricson and Henricson, {Erik K} and McDonald, {Craig M}",
year = "2017",
month = "2",
day = "1",
doi = "10.1111/dmcn.13277",
language = "English (US)",
volume = "59",
pages = "224--231",
journal = "Developmental Medicine and Child Neurology",
issn = "0012-1622",
publisher = "Wiley-Blackwell",
number = "2",

}

TY - JOUR

T1 - Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy

T2 - DMD Upper Limb PROM

AU - The Upper Limb Clinical Outcome Group

AU - Klingels, K.

AU - Mayhew, A. G.

AU - Mazzone, E. S.

AU - Duong, T.

AU - Decostre, V.

AU - Werlauff, U.

AU - Vroom, E.

AU - Mercuri, E.

AU - Goemans, N. M.

AU - Eagle, M.

AU - De Groot, I.

AU - Main, M.

AU - Messina, S.

AU - Campion, G.

AU - Servais, L.

AU - Van den Hauwe, M.

AU - Es, E.

AU - Pane, M.

AU - Buccella, F.

AU - Kuijer, J.

AU - Ceradini, F.

AU - Henricson, E.

AU - Henricson, Erik K

AU - McDonald, Craig M

PY - 2017/2/1

Y1 - 2017/2/1

N2 - 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.

AB - 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.

UR - http://www.scopus.com/inward/record.url?scp=84989295908&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84989295908&partnerID=8YFLogxK

U2 - 10.1111/dmcn.13277

DO - 10.1111/dmcn.13277

M3 - Article

C2 - 27671699

AN - SCOPUS:84989295908

VL - 59

SP - 224

EP - 231

JO - Developmental Medicine and Child Neurology

JF - Developmental Medicine and Child Neurology

SN - 0012-1622

IS - 2

ER -