TABLE 5.
Number of Times a PRO Concept Is Mentioned in Unpublished and Ongoing Studies Listed in US and EU Clinical Trials Registries for PBC and PSC
| PRO Concepts | Frequency, n (%) | ||
|---|---|---|---|
| PBC (n = 50) | PSC (n = 34) | Total (n = 88) | |
| Functioning/quality of life | |||
| Broad HRQOL | 14 (28.0) | 9 (26.5) | 24 (27.3) |
| Cognitive functioning | 1 (2.0) | 0 (0.0) | 1 (1.1) |
| Physical functioning | 1 (2.0) | 1 (2.9) | 2 (2.3) |
| Symptoms | |||
| Anxiety | 1 (2.0) | 0 (0.0) | 1 (1.1) |
| Autonomic dysfunction | 1 (2.0) | 0 (0.0) | 1 (1.1) |
| Depression | 1 (2.0) | 0 (0.0) | 1 (1.1) |
| Fatigue | 5 (10.0) | 4 (11.8) | 10 (11.4) |
| Multiple gastrointestinal symptoms | 0 (0.0) | 1 (2.9) | 1 (1.1) |
| Poor sleep quality | 2 (4.0) | 0 (0.0) | 2 (2.3) |
| Pruritus/itch | 19 (38.0) | 8 (23.5) | 29 (33.0) |
| Sleep disturbance | 0 (0.0) | 1 (2.9) | 1 (1.1) |
| Sleepiness/daytime somnolence | 1 (2.0) | 0 (0.0) | 1 (1.1) |
| Weakness | 1 (2.0) | 0 (0.0) | 1 (1.1) |
| Treatment AEs/side effects | |||
| Gastrointestinal AEs | 2 (4.0) | 4 (11.8) | 6 (6.8) |
| Physical AEs | 1 (2.0) | 4 (11.8) | 5 (5.7) |
| Other | |||
| Health perception | 0 (0.0) | 2 (5.9) | 2 (2.3) |