Prioritizing Parental Worry Associated with Duchenne Muscular Dystrophy Using Best-Worst Scaling.

Area of Science:

• Pediatric rare diseases
• Clinical psychology
• Patient-reported outcomes

Background:

• Duchenne muscular dystrophy (DMD) is a severe pediatric disorder impacting families.
• Understanding parental concerns is crucial for effective clinical support.
• Existing assessment methods may not fully capture parental priorities.

Purpose of the Study:

• To identify and prioritize parental worries related to Duchenne muscular dystrophy.
• To compare these priorities between parents of ambulatory and non-ambulatory children.
• To demonstrate the utility of Best-Worst Scaling (BWS) in assessing parental concerns.

Main Methods:

• A community-engaged approach identified 16 short-term worries in DMD.
• Best-Worst Scaling (BWS) was employed with 119 parents to rank worries.
• Independent-sample t-tests analyzed differences based on child's ambulatory status.

Main Results:

• The highest parental priorities were weakness progression and consistent care access.
• Parents of ambulatory children prioritized avoiding missed treatments and parental adequacy more than those with non-ambulatory children.
• Parents of non-ambulatory children were more concerned about their child feeling like a burden.

Conclusions:

• Caregiver interventions must address the emotional burden of DMD, irrespective of disease stage.
• BWS provides a clinically relevant and accessible method for prioritizing patient and caregiver concerns.
• This approach offers an alternative to traditional rating scales for assessing disease impact.