Sanfilippo syndrome (MPS III) is a rare, degenerative condition with no approved therapies. In our prior research we obtained qualitative data from parents about Sanfilippo impact and unmet treatment needs. Here we report a quantitative survey that assessed parents’ perceptions of the relative importance of unmet treatment needs.
We conducted a Best-Worst Scaling (BWS) case 1 experiment in an online survey of parents. Participants completed 12 choice sets to choose symptoms that were most and least important to treat in the context of a non-curative therapy. Log-odds importance weights were estimated using random-parameters logit. Subgroup analysis was conducted based on parent ratings of severity of child’s symptoms: mild/moderate vs. severe.
164 parents responded. Their children with Sanfilippo syndrome (subtypes A-D) ranged in age from 1 to over 40 years old. Symptoms with the highest importance weights were having pain, unsafe behaviors, feeling unwell, not having enough sleep, aggressive/impulsive toward others, trouble getting around, and sharing feelings back and forth. Subgroup analysis by severity (mild/moderate n= 94 and severe n= 70) indicated that parents of severely-progressed children prioritized sharing feelings back and forth, hyperactivity, and child’s worry/anxiety significantly more than parents of mild/moderately progressed children. Parents of mild/moderately progressed children were significantly more likely to prioritize aggressive/impulsive behaviors to others, paying attention, feeling unhappy, following directions, and remembering people and places than parents of severely-progressed children.
Parents’ relative treatment priorities concord with the multi-symptom impacts of Sanfilippo syndrome. The top set of prioritized symptoms span several domains: health, behavioral, motor, and communication. Pain and safety, two areas important to child and family quality of life, were most prioritized in the combined sample and also in progression-based subgroups.
