Table 6 Changes reported by caregivers after the start of the pabinafusp alfa trial
Symptom domains | Specific sub-domain concepts (if applicable) |
|---|---|
Developmental/neurological | Improved (n = 5): speaking/language skills (n = 4, two probed), concentration (n = 3, one probed), facial expressions (n = 2), self-control (n = 2, probed), eye contact, proactivity, mental clarity, quicker understanding of concepts (n = 1 each) Worsened (n = 1): gradual decline in cognition and concentration (n = 1), self-control (n = 1, probed) |
Motor functioning/mobility | Improved: ability to walk (n = 3, one probed) Worsened (n = 2): difficulty moving around (n = 2), balance issues, movement ability regressed but variable (n = 1 each, same participant) |
Musculoskeletal/somatic/ organ involvement | Improved (n = 3): tongue size (n = 2), putting on additional muscle, previously enlarged organs decreasing in size, no progression in organ, heart condition, stools (n = 1 each) Worsened (n = 1): back issues, chest catching/locking up (n = 1 each, same participant) |
Joint/hand function | Improved (n = 3): flexibility (n = 2, one probed), less stiffness/softer muscles, joints improved with rehab/massage (n = 1 each) Worsened: hand/wrist stiffness (n = 1) |
Fatigue | Improved: fatigue (n = 1) Worsened: fatigue (n = 2) |
Others | Improved: ear infections/illness frequency (n = 2), sleeping patterns (n = 2, 1 probed) Worsened: difficulty swallowing, seizures (n = 1 each, same participant) |