Table 6 Patient Reported Outcomes mean scores by disease type
PRO | Number of Patients Completed | Mean Score | Independent t-test between groups | PRO ref. range | ||
|---|---|---|---|---|---|---|
GD 1 | nGD | GD 1 | nGD patients | |||
CHU9D* | 3 | 10 | 0.93 (SE = .016) | 0.81 (SE = 0.025) | t(11) = 2.43; p = .033 | 0.33–1 (higher score = perfect health) |
PedsQL MFS* | 3 | 10 |  |  |  | 0–100 (higher score = less fatigue) |
General Fatigue | 69.44 (SE 2.78) | 56.25 (SE 6.37) | t(11) = 1.09; p = .298 | |||
Sleep Fatigue | 56.99 (SE7.35) | 58.08 (SE 8.32) | t(11) = −.07; p = .945 | |||
Cognitive Fatigue | 69.44 (SE 15.47) | 57.08 (SE 4.89) | t(11) = 1.04; p = .321 | |||
Self-esteem: Rosenberg Self-worth* | 2 | 10 | 14.5 (SE = 0.5) | 16.5 (SE = 0.5) | t(10) = −1.70; p = .119 | 0–30 (< 15 = Low self-esteem) |
Perceived Stress Scale* | 2 | 10 | 6.5 (SE 4.5) | 22.7 (SE 6.17) | t(10) = − 3.38; p = .007 | 0–40 (higher score = greater perceived stress) |
REM Sleep Behaviour Disorder Questionnaire | 5 | 11 | 1.4 (SE .872) | 5.64 (SE .877) | t(14) = −2.94; p = .011 | > 5 = problematic sleep |
Pittsburgh Sleep Quality Index | 5 | 10 | 3.8 (SE 2.17) | 8.8 (SE 1.76) | t(13) = −1.90; p = .079 | > 5 = problematic sleep |
Sleep Disturbance Scale for Children | 0 | 3 | NA | 72.67 | NA | Total T-Score > 70 pathological* (Subdomains scored identically) |