Development and content validation of the Muscular Dystrophy Child Health Index of Life with Disabilities questionnaire for children with Duchenne muscular dystrophy

Abstract

The Muscular Dystrophy Child Health Index of Life with Disabilities (MDCHILD) is a new patient-reported outcome measure for Duchenne muscular dystrophy (DMD). The Priority Framework of Outcomes underpins the content for the MDCHILD. The MDCHILD incorporates the health-related priorities of males with DMD and their parents. The MDCHILD was deemed sensible by children, their parents, and health care professionals.

Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments

“…Many researchers who study quality of life within this perspective emphasize the importance of measuring the individual’s degree of life satisfaction. In other words, they are interested in how pleased an individual feels about particular aspects of his or her life” “Individuals between the ages of 12 and 19 years who have DMD” Research instrument and potentially useful as a clinical measure Doubtful Yes MDCHILD Propp, 2017 [ 100 ] Propp et al 2019 [ 8 ] English (UK) “Health-related priorities for children with DMD (…) defined as concerns, desires, and expectations arising from the lived experience of that condition” Children with DMD (assumed 5–18 years) Cohort studies, clinical trials, and clinical decision-making Doubtful Yes PedsQL 3.0 DMD Uzark et al 2012 [ 62 ] English (US) “Health-related quality of life (QoL), a multidimensional construct that includes physical, psychological, and social functioning, has emerged as an important outcome in pediatric populations with chronic health conditions.” Children with DMD from 2 to 18 years Assumed the same as PedsQL 4.0 GCS Doubtful Yes PedsQL 3.0 MFS Varni et al 2002 [ 101 ] English (US) “designed to measure child and parent perceptions of fatigue in pediatric patients” Assumed the same as PedsQL 4.0 GCS “may be utilized as outcome measures in pediatric cancer clinical trials, research, and clinical practice for HRQOL” Inadequate Yes PedsQL 3.0 NMM Iannaccone et al 2009 [ 102 ] English (US) “HRQOL is a multidimensional construct, consisting at the minimum of physical, psychological (including emotional and cognitive), and social health dimensions delineated by the World Health Organization. HRQOL has emerged as the most appropriate term for quality of life dimensions that represent a patient’s perceptions of the impact of an illness and its treatment on their own functioning and well-being and which are within the scope of healthcare services and medical products.” Children and young people with neuromuscular disorders, in particular spinal muscular atrophy Assumed the same as PedsQL 4.0 GCS Doubtful Yes PedsQL 4.0 GCS Varni et al 1999 [ 103 ] English (US) “The PedsQL measures the patient’s and the parent’s perceptions of the patient’s HRQOL, as def...…”

“…These results speak to the potential harshness of a worst score counts system advocated by COSMIN, which we discuss further below. Further, because the PROM is new, there is a lack of published content validity studies that may improve the quality of evidence for the MDCHILD going forward, such as that contained in a non-peer-reviewed thesis [100], not eligible for inclusion in the current review.…”

“…The most recent PROM developed specifically for use in children and adolescents with DMD was the MDCHILD. Although the PROM is designed to measure “health-related priorities” [ 8 ], much of the content maps onto the CMQM framework [ 9 ] and thus covers QoL. While the MDCHILD had many commendable strengths in PROM design, the overall rating of the PROM development, based on the COSMIN worst score counts system [ 25 ], was rated as doubtful due to lack of details reported in the development papers.…”

“…In order to attempt to measure QoL in people with DMD a number of both generic (such as the EQ-5D [ 6 , 7 ]) and condition-specific (such as the MDCHILD [ 8 ]) patient reported outcome measures (PROMs) are used. However, concerns have been raised about the validity of existing PROMs to comprehensively assess QoL in DMD [ 9 ].…”

Evaluation of the U.S. Adherence Questionnaires VERITAS-PRO and VERITAS-PRN for Use in Patients with Hemophilia in the German Healthcare System

“…Another development of a PRO measure for health-related priorities of children with Duchenne muscular dystrophy (DMD) used the CVR for testing the CV of the "Muscular Dystrophy Child Health Index of Life with Disabilities (MDCHILD)". 40 Multidisciplinary HCP completed an itemby-item analysis of the measure and a 14-item sensibility questionnaire. Applying the CVR, Propp et al could show that the MDCHILD met all the sensibility criteria of children with DMD, their parents, and HCP and was ready for psychometric evaluation.…”