Patient reported outcome measures of quality of life in Duchenne muscular dystrophy (DMD): a systematic review of content and structural validity using COSMIN

ABSTRACT

Duchenne muscular dystrophy (DMD) is an inherited neuromuscular disorder that predominantly affects boys and men. There is no known cure, so current clinical efforts are focused on improving the health-related quality of life (QoL) of people with DMD. Of the validated methods for assessing QoL, patient-reported outcome measures (PROMs) are the most common. In a field of many options, the choice of which PROM to use should be based predominantly on their validity and reliability for the construct, population, and context of use of interest. In the current report we critically reviewed the content and structural validity of PROMs used to assess QoL in people with DMD, using robust, updated COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guidelines.

In this review, we defined QoL as a multidimensional construct involving physical, psychological, and social components. We conducted a systematic search of the published literature for self-reported, multi-item PROMs assessing at least one aspect of QoL in a sample of at least 75% boys and men with DMD. We further refined these results for studies with evidence of content and structural validity, including development studies. The resulting PROMs and related studies were systematically rated, in terms of methodological quality, and evidence for content and structural validity, as per the latest COSMIN procedures.