Measuring carer quality of life in Duchenne muscular dystrophy: a systematic review of the reliability and validity of self-report instruments using COSMIN

Abstract

Introduction Duchenne muscular dystrophy is a rare, progressive, life-limiting genetic neuromuscular condition that significantly impacts the quality of life of informal caregivers. Carer quality of life is measured using heterogeneous self-report scales, yet their suitability for Duchenne remains unclear. This review aimed to identify and evaluate the reliability and validity of quality of life instruments in Duchenne carers. Materials and methods Systematic searches were conducted in Embase, MEDLINE, CINAHL, PsycINFO, Cochrane Library and Google Scholar. Full research articles reporting data on multiple-item self-report quality of life instruments in informal Duchenne carers were included. Extracted evidence was qualitatively synthesised and evaluated, including risk of bias, against the Consensus-based Standards for the selection of health Measurement Instruments. Duchenne carer collaborators (N = 17) helped rate the instruments’ content validity. Results Thirty-one articles featuring thirty-two quality of life instruments were included. Content validity was rated as “inconsistent” based on very low quality evidence. For Duchenne carer collaborators, the best instrument was PedsQL Family Impact Module. Only one instrument had evidence for structural validity (rated “unsatisfactory”) and measurement invariance (rated “satisfactory”). Instruments received “satisfactory” ratings for internal consistency and mixed ratings for construct validity and responsiveness. There was no evidence for reliability, measurement error, or criterion validity. Discussion Instruments used to measure Duchenne carer quality of life have limited and often inconsistent supportive psychometric evidence. Further work must investigate instruments’ measurement properties in Duchenne carers and/or the development of new tools. In the interim, we recommend considering the PedsQL Family Impact Module based on Duchenne carer ratings.

The relationship between lower urinary system symptoms and the level of independence and quality of life in children with Duchenne muscular dystrophy

“…Compared to their healthy peers, children with DMD have developmental and motor delays, lower functional levels, poor posture, and gait disorders. These disease-specific symptoms have been observed to affect the health-related quality of life of people with the condition in a variety of ways [22,24]. The addition of bladder and bowel symptoms to these symptoms can further exacerbate the negative impact on QoL.…”

Factors associated with the health-related quality of life among people with Duchenne muscular dystrophy: a study using the Health Utilities Index (HUI)

“…A systematic review and other recent investigations conducted after the data collection period of the present study, highlighted the lack of evidence to support structural and content validity of the HUI and other generic instruments for assessing HRQoL impacts among those with DMD. Additionally, how well existing generic instruments cover the aspects of HRQoL most important from the patient perspective, particularly from pediatric populations,is now emerging [ 11 , 27 – 30 ]. Additional work to validate the content of existing generic measures and confirm that included constructs have relevance to the DMD population, as well as the development of novel DMD-specific measures [ 11 , 12 , 28 ], will help ensure that HRQoL among those with DMD can be reliably and validly measured.…”

Validez de contenido de un instrumento de medición para medir el éxito en la implementación del TPM

“…Por lo tanto, no es posible determinar si la discrepancia observada en cada ítem se debe al azar (Almanasreh et al, 2019). A pesar de su incapacidad para discriminar los efectos de la aleatoriedad en la evaluación de la concordancia en el juicio de los expertos, estos métodos aún se aplican para validar el contenido de los IM (Carlton et al, 2022;Feng et al, 2022;Ma et al, 2023;Reick et al, 2023). Es decir, desde un punto de vista estadístico, los resultados obtenidos al evaluar la validez de contenido de un IM con estos métodos son cuestionables.…”