Table 2 Potential Parent Reported Outcome Measures - For Systemic & Lung Disease

Lack of energy / decreased physical activity/being sluggish

Behaviors like sitting on the couch and playing with iPad, or just sitting quietly

Fatigue - reporting tiredness after minor exertion

Disturbed sleep at night

Not wanting to get out of bed

Grumpy behavior: being annoyed without reason, with siblings and parents

Less smiling and laughing behavior

Reduced exploratory behavior / reduced sense of curiosity

Decreased interest in play

Lack of appetite

Cough

Labored breathing

Breathlessness with every day-life activities (i.e., climbing stairs)

Decreased ability to run around

Increased respiratory rate

O2 requirement (for patients who use oxygen supplementation)