TABLE 4.
PRO Instruments Used to Measure the Top 5 Most Common PRO Concepts in the PBC and PSC Literature
| PRO Concept/Instrument | Frequency, n (%) | |||
|---|---|---|---|---|
| PBC (n = 642) | PSC (n = 177) | Both (n = 107) | Total Concepts (n = 926) | |
| PRURITUS TOTAL | 166 (25.9) | 39 (22.0) | 26 (24.3) | 231 (25.0) |
| No Instrument | 76 (45.8) | 22 (56.4) | 1 (3.8) | 99 (42.9) |
| Itch Assessment With Visual Analog Scale | 33 (19.9) | 6 (15.4) | 18 (69.2) | 57 (24.7) |
| Likert/Numeric Grading Scale NOS | 28 (16.9) | 6 (15.4) | 2 (7.7) | 36 (15.6) |
| 5‐D Itch Scale | 9 (5.4) | 1 (2.6) | 0 (0) | 10 (4.3) |
| PBC‐40 | 8 (4.8)* | 0 (0) | 1 (3.8) | 9 (3.9) |
| Pruritus numeric rating scale (0‐10) | 2 (1.2) | 2 (5.1) | 1 (3.8) | 5 (2.2) |
| Diary NOS | 2 (1.2) | 1 (2.6) | 0 (0) | 3 (1.3) |
| Visual Analog Scale NOS | 2 (1.2) | 1 (2.6) | 0 (0) | 3 (1.3) |
| Itch Diary (Patient‐Reported Symptom Questionnaire) | 1 (0.6)* | 0 (0) | 1 (3.8) | 2 (0.9) |
| Itch Severity Scale | 1 (0.6) | 0 (0) | 1 (3.8) | 2 (0.9) |
| Other ‡ | 4 (2.4) | 0 (0) | 1 (3.8) | 5 (2.0) |
| FATIGUE TOTAL | 130 (20.3) | 28 (15.8) | 14 (13.1) | 172 (18.6) |
| No instrument | 50 (38.5) | 14 (50.0) | 0 (0) | 64 (37.2) |
| Fatigue Impact Scale | 35 (26.9) † | 6 (21.4) | 4 (28.6) | 45 (26.2) |
| Likert/Numeric Grading Scale NOS | 12 (9.2) | 3 (10.7) | 0 (0) | 15 (8.7) |
| PBC‐40 | 13 (10.0)* | 1 (3.6) † | 0 (0) | 14 (8.1) |
| Visual Analog Scale NOS | 4 (3.1) | 4 (14.3) | 6 (42.9) | 14 (8.1) |
| Fisk Fatigue Severity Scale | 5 (3.9) † | 0 (0) | 3 (21.4) | 8 (4.7) |
| Krupp’s Fatigue Severity Scale | 4 (3.1) | 0 (0) | 0 (0) | 4 (2.3) |
| Fatigue Diary | 3 (2.3) | 0 (0) | 0 (0) | 3 (1.7) |
| Modified Fatigue Impact Scale | 2 (1.5) | 0 (0) | 1 (7.1) | 2 (1.2) |
| Other ‡ | 2 (1.4) | 0 (0) | 0 (0) | 3 (1.8) |
| BROAD HRQOL TOTAL | 82 (12.8) | 34 (19.2) | 30 (28.0) | 146 (15.8) |
| SF‐36 | 20 (24.4) | 11 (32.4) | 13 (43.3) | 44 (30.1) |
| PBC‐40 | 35 (42.7)* | 5 (14.7) † | 3 (10.0) | 42 (28.8) |
| Chronic Liver Disease Questionnaire | 5 (6.1)* | 4 (11.8)* | 3 (10.0) | 12 (8.2) |
| PBC‐27 | 4 (4.9)* | 3 (8.8) † | 1 (3.3) | 8 (5.5) |
| Likert/Numeric Grading Scale NOS | 4 (4.9) | 1 (2.9) | 0 (0) | 5 (3.4) |
| Nottingham Health Profile | 3 (3.7) | 0 (0) | 0 (0) | 3 (2.1) |
| NIDDK‐QA | 1 (1.2) | 0 (0) | 2 (6.7) | 3 (2.1) |
| Visual Analog Scale NOS | 3 (3.7) | 0 (0) | 0 (0) | 3 (2.1) |
| 15D | 0 (0) | 1 (2.9) | 1 (3.3) | 2 (1.4) |
| No instrument | 1 (1.2) | 1 (2.9) | 0 (0) | 2 (1.4) |
| Other ‡ | 7 (8.5) | 8 (23.5) | 7 (23.3) | 2219 (15.1) |
| GASTROINTESTINAL AEs/TREATMENT SIDE EFFECTS TOTAL | 41 (6.4) | 10 (5.7) | 8 (7.5) | 59 (6.4) |
| No instrument | 37 (90.2) | 10 (100) | 8 (100) | 55 (93.2) |
| Likert/Numeric Grading Scale NOS | 2 (4.9) | 0 (0) | 0 (0) | 2 (3.4) |
| Diary NOS | 1 (2.4) | 0 (0) | 0 (0) | 1 (1.7) |
| Gastrointestinal Symptom Rating Scale | 1 (2.4) | 0 (0) | 0 (0) | 1 (1.7) |
| PHYSICAL AEs/TREATMENT SIDE EFFECTS TOTAL | 34 (5.3) | 8 (4.5) | 9 (8.4) | 51 (5.5) |
| No instrument | 31 (91.2) | 7 (87.5) | 9 (100) | 47 (92.1) |
| Likert/Numeric Grading Scale NOS | 1 (2.9) | 1 (12.5) | 0 (0) | 2 (3.9) |
| Diary NOS | 1 (2.9) | 0 (0) | 0 (0) | 1 (2.0) |
| Itch Assessment With Visual Analog Scale | 1 (2.9) | 0 (0) | 0 (0) | 1 (2.0) |
Bolded instruments denote instruments that were either developed in the target population or subsequently underwent psychometric testing in the target population.
*
Instrument was developed with input from the target population.
†
Instrument was not developed with input from the target population but later underwent psychometric testing in the target population.
‡
Pooled total of remainder of instruments used to measure the PRO concept (given the low frequency of each instrument being used per PRO concept found in the total population).
Abbreviation: GI, gastrointestinal.