Table 2 Symptoms reported by caregivers prior to the pabinafusp alfa trial
Symptom domains | Specific sub-domain concepts (if applicable) |
|---|---|
Developmental/neurological involvement (n = 6) | •General observable cognitive development issues (n = 3) •Diagnosed brain function/cognitive disorders (n = 2) •Specific behavioral and developmental concerns (n = 1, each) |
Motor issues (n = 3) | •Walking (n = 2) •Falling, grip problems, and movement difficulties (n = 1, each) |
Joint/tissue involvement (n = 3) | •Joint deformation/stiffness (n = 3) •Connective tissue disease (n = 1) |
General illness/infections (n = 3) | •Ear infections (n = 2) •Generally prone to illness (n = 1) |
Hernias (n = 3) | •Hernias (n = 3) |
Others (less frequently reported) | •Difficulty swallowing, heart valve regurgitation, difficulty breathing, seizures (n = 1 each) |