Table 3 Impacts reported by caregivers prior to the pabinafusp alfa trial
Impact domains | Pre-trial, specific sub-domain concepts (if applicable) |
|---|---|
Hospitalizations (n = 7) | •Hospitalizations (n = 7) |
Cognition/development (n = 5) | •Communication issues (n = 3) •Lack of independence (n = 2) •Necessity for feeding support, a decrease in task proficiency, issues with comprehension, and lapses in word recall (n = 1, each) |
Physical limitations (n = 5) | •Stopping running or playing due to heart condition (n = 2) •Difficulty walking (n = 2) •Bedsores, needing a wheelchair, difficulty sitting on the floor or kneeling, inability to use chopsticks, precautions needed against falls (n = 1, each) |
Bathroom issues (n = 3) | •Inability to use bathroom alone/needing help (n = 2) •Diapers needed (n = 2) •Inability to express the need to use the bathroom, large urine volumes, issues with toilet training (n = 1 each) |
Schooling (n = 3) & social (n = 3) | •Hyperactivity leading to an inability to remain alone, struggles with social interaction, frequent school absenteeism, and the necessity for caregivers to accompany children during school activities, as well as reported difficulties in the educational setting (n = 1, each) |
Seizures (n = 1) | •Seizures (n = 1) |