Caregiver-reported meaningful change in functional domains for individuals with developmental and epileptic encephalopathy: A convergent mixed-methods design.

Aim: To investigate how caregivers of children with developmental and epileptic encephalopathy and severe developmental impairments describe meaningful change for functional domains and why it is important.

Method: This was a convergent mixed-methods design study. A survey was completed by 267 parents of children aged 12 months or older. For prioritized functional domains (communication, gross motor, fine motor, eating), parents reported the smallest improvement that would be important and explained why. Data were analyzed using directed content analysis and meaningful change codes were mapped to impairment levels.

Results: The median age of the children was 8 years 8 months (interquartile range 4 years 2 months-14 years 6 months) and 149 (55.8%) were female. Content analysis yielded 86 meaningful change codes. Common codes described capacity to communicate preferences and emotions, gain sitting and walking skills, grasp objects for play, eat foods without choking, or using utensils. Some codes were reported for each impairment level (e.g. communicating needs/wants/likes for expressive communication); others were specific to an impairment level (e.g. gaining head control if unable to walk). Meaningful change was anticipated to affect health, independence and safety, care regimens, and quality of life of affected individual and families.

Interpretation: The meaningful change codes indicate critical components within domains for evaluations in clinical trials.