Table 1.
Characteristics of included studies
| Author (Year) (Reference) | Country | Study design | Sample size (PSC cases) | Mean age (SD) year | Gender (Male n %) | Disease stage | Mayo risk score / MELD Score | IBD (Yes/No (n (%)) | LT (Yes/No (n (%)) | PROM | Rationale for Assessment | PROM administration |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Gavaler (1991) [66] | USA | Cross- sectional study | 23 (23) | Quiescent group: 34.7 (6.2) Symptomatic group: 39.8 (1.6) | 15 (65%) | Symptomatic UC: Mild: 7 (40%) Moderate: 8 (47%) Severe: 2 (13%) |
NR | Yes (23 (100%)) | Yes (23 (100%)) | Study questionnaire: symptoms of UC | A | Postal & telephone |
| Gross (1999) [26] | USA | Before & after study | 157 (92) | Total sample: 50 (10) | 31 (34%) | NR | MRS: Mean 5.3 | NR | Yes (157 (100%)) | NIDDK-QA, pilot version NIDDKQA | A | Clinic |
| Kim (2000) [28] | USA | Validation study | 96 (17) | 45 (9.3) | 7 (41%) | PSC undergoing LT: 17 (100%) | MRS: mean (SD) = −0.1(1.0) | NR | PSC patients undergoing LT: 17 (100%) | NIDDK-QA, SF- 36 | D | Clinic |
| Bharucha (2000) [67] | USA | Pilot study | 20 (20) | 44 (11) | 12 (60%) | Early stage (1–2): 10 (50%), Late stage (3–4): 10 (50%) |
MRS: mean (SD) = 2.87 (0.95) | Yes (14 (70%)) | No | Grading system fatigue & pruritus | B | Unclear |
| Younossi (2000) [38] | USA | Cross-sectional study | 104 (29) | Total sample:: 55 (12) | Total sample 28 (97%) | NR | NR | NR | No | SF- 36, CLDQ | A | Unclear |
| Younossi (2001) [39] | USA | Cross-sectional study | 353 (45) | Total sample: 54 (11) | Total sample 38 (30%) | Total sample: Child-pugh class: no cirrhosis: 47 (13%) class A: 43 (12%) class B-27 (8%) class C-4 (1%) |
NR | NR | NR | SF-36, CLDQ | A | Clinic |
| Longworth (2003) [45] | England and Wales | Cost effectiveness study | 347 (70) | NR | 48 (69%) | NR | Of 41 patients MELD score median/IQR = 10/6–16 | NR | Yes (45) 64%)) | EuroQol EQ. 5D | C | Postal |
| Bjornsson (2004) [44] | England & Sweden | RCT | 93 (20) | NR | 13 (65%) | Cirrhosis: 5 (1%),Ludwig’s fibrosis score stage 1: 9 (44%), stage 2: 4(21%), stage 3:6(30%) | NR | Yes (16 (80%)) | No | PGWB, FIS, BDI, GSRS, Rome ll modular QA | A | Postal |
| Ter Borg (2004) [36] | Netherlands | RCT | 33 (11) | NR | 10 (91%) | NR | NR | NR | No | VAS, FFSS, MFI | B | NR |
| Ter Borg (2005) [48] | Netherlands | Cross-sectional study | 72 (27) | 45 (NR) | 19 (70%) | Cirrhosis: 15 (56%) | NR | Yes (2 (7%) | NR | VAS, FFSS, SF-36 | A | NR |
| Olsson (2005) [33] | Sweden, Norway, Denmark | RCT | 198 (198) | UDCA: 43.6(12.7) Placebo: 43.1 (11.2) | 139 (70%) | NR | NR | Yes (168 (85%)) | NR | SF- 36 | B | Unclear |
| Gorgun (2005) [21] | USA | Case matched study | 65 (65) | 43.37 (11.2) | 45 (69%) | NR | NR | Yes (65 (100%)) |
No | FPQ, CGQOL | A | |
| Mansour-Ghanaei (2006) [49] | Iran | RCT | 34 (6) | Total sample: 53.97 (11.93) | NR | NR | NR | NR | NR | VAS | B | Unclear |
| Mayo (2007) [50] | USA | RCT | 21 (4) | Total sample: 53.97 (11.93) | Total sample 5 (15%) | NR | aTotal sample MELD mean (range): 11(6–24) | NR | NR | VAS, IDS-SR30 | B | Unclear |
| Van os (2007) [52] | Netherlands | Cross-sectional study | 92(37) | 43.8(12.3) | 24 (65%) | Cirrhosis: 5 (13.5%) | NR) | NR | NR | BDI, SADS | A | Postal |
| Tillman (2009) [37] | Germany | Cross-sectional study | 511(13) | 42 (NR) | NR | NR | NR | NR | NR | SF- 36, FIS, WHOQOL-BREF, HADS | A | In clinic |
| Ananthakrishnan (2010) [47] | USA | Case-control study | 26 (26) | 40.7 (14.8) | 21 (80.8%) | NR | MELD score mean (range) 8 (6–20) | Yes (26(100%)) | No | SIBDQ, HBI, UCAI | A | Outpatient clinic |
| Aberg (2012) [30] | Finland | Cross-sectional study | 401 (56) | 53 (9) | 36 (64%) | NR | NR | NR | Yes (56 (100%)) | 15D, ad hoc questionnaire | A | Postal |
| Benito De Valle (2012) [29] | England & Sweden | Cross-sectional study | 182 (182) | 160 patients no LT: 50 (16) | 112 (70%) | Small duct disease: 17 (11%), Liver cirrhosis: 12 (8%), Decompensated liver disease: 9 (6%) | MRS mean (SD): 0.34 (1.10) | Yes (126 (79%)) | Yes (22 (12%)) | SF-36, CLDQ, FIS, HADS | A | Postal |
| Hagstrom (2012) [68] | Sweden | Cross-sectional study | 96 (96) | 47 (13) | 63 (66%) | Cases child pugh score of 10, significant fibrosis: 26 (27%), non-significant fibrosis: 70 (73%) | NR | Yes (73 (76%)) | Yes (12 (12.5%)) | LDH | A | Interview |
| Gulati (2013) [25] | USA | Cross-sectional study | 40 (24) | Total sample: 11.6 (4.5) | 17 (43%) | Total sample: Cirrhosis 22 (55%) | NR | Total sample: Yes (16 (65%)) | No | A | Unclear | |
| Block (2014) [69] | Norway & Sweden | Case-control study | 48 (48) | NR | 40 (83%) | NR | NR | 48 | Yes (IPAA: 11, IRA: 7) | OS | A | Scheduled follow up visit |
| Gotthardt (2014) [6] | Germany | Cross-sectional study | 113 (113) |
43.6 (14.2) | 81 (71.7%) | NR | MRS n: low/intermediate/ high =48 (42%) / 25 (22%) / 5 (4%) | Yes (71 (63%)) | NR | SF 36, PHQ-9 | A | Postal |
| Hov (2014) [70] | Norway | Case-control study | 240 (240) |
NR | 171 (71%) | NR | NR | Yes (183 (77%)) | Yes (94 (39%)) | Study questionnaire | A | Postal |
| Pavlides (2014) [34] | England | Retrospective case note review | 40 (PSC-IPAA = 21 & PSC-UC = 19) | NR | 31 (78%) | PSC-IPAA had dysplasia: 2 (5%) | NR | Yes (19 (47.5%)) | No | OS, CGQOL, FSFI, IIEF | A | Postal |
| Raszeja-Wyszomirska (2014) [35] | Poland | Cross-sectional study | 102 (102) | 36 (12) | 73 (72%) | Cirrhosis: 30 (29%) | NR | Yes (65 (64%)) | NR | SF 36, PBC-40, PBC-27 | A | Unclear |
| Cheung (2015) [32] | Canada | Cross-sectional study | 162 (99) | 46.1 (15.1) | 50 (51%) | Cirrhosis: 47 (48%), Decompensated liver disease: 16 (16%) | NR | Yes (74) | No | SF-36, PBC-40, PHQ-9, LDQOL, SIBDQ, 10 peered-reviewed QA on emotional and psychosocial | A | Postal or clinic |
| Dyson (2015) [20] | USA | Cross-sectional study | 40 (40) | 51 (13) | 31 (78%) | NR | NR | Yes (24 (60%)) | NR | FIS, ESS, HADs, COMPASS | A | Postal |
| Eaton (2015) [71] | Canada & USA | Case-control study | 1000 (1000) |
NR | 619 (72%) | NR | NR | Yes (741 ((74%)) | Yes (450 ((45%)) | HHQ | A | Postal or clinic |
| Haapamaki (2015) [31] | Finland | Cross-sectional study | 341 (341) |
43.3 (13.7) | 183 (54%) | ERC-score mean (SD): 5.9 (3.4) | NR | Yes (237 (69.5%)) | Yes (9 (2.6%)) | 15D, study questionnaire | A | ERC examination at the HUGH endoscopy unit |
| Kalaitzakis (2015) [27] | England and Sweden | Cross-sectional study | 163 (163) |
No LT: 50 (16) | No LT 122 (75%) |
No LT Small-duct disease: 15 (10%), Diver cirrhosis: 11 (8%), Decompensated liver disease: 8 (6%) | No LT MRS: mean (SD) = 0.11(1.42) | No LT Yes (116 (71%)) | Yes (19 (12%)) | SF 36, SF-6D, CLDQ, study questionnaire | A, C | Unclear |
| Raszeja-Wyszomirska (2015) [41] | Poland | Cross-sectional study | 33 (33) | 35.3 (13.38) | 11 (33%) | Cirrhosis: 6 (18%) | NR | Yes (22 (67%) | NR | SF 36, PBC-40, PBC-27 | A | NR |
| Carbone (2017) [46] | Italy | Longitudinal study | 227 (64) | 50(11) | 39 (66%) | NR | NR | NR | NR | EQ-5D | A | Clinic |
| Kempinska (2017) [40] | Poland | Cohort study | 275 (275) | Median 55, range 28–90 | 182 (66%) | NR | NR | NR | NR | SF 36, PBC-40, PBC-27 | A | NR |
| Kittanamongkolchai (2017) [51] | USA | Before and after study | 13 (5) | 46.4 (13.2) | 1 (20%) | NR | NR | NR | NR | Pruritus numerical rating scale | B | Physician administered |
| Tabibian (2017) [42] | USA | Pilot study | 16 (16) | 40 (NR) | 13 (81%) | All patients had stage 1–3 PSC | NR | 13 (81%) | NR | FFSS, 5-D itch scale, CLDQ, SF-36 | B | NR |
| Younossi (2017) [43] | USA | Validation study | 102 (102) | 44 (13) | 33 (32%) | Cirrhosis: 37 (39%) | NR | 67 (68%) | NR | PSC PRO, SF-36, CLDQ, PBC-40, 5-D Itch | D | ePRO website |
15D 15-dimensional health-related quality of life measure, 5-D Itch Five dimensions Itch, BDI Beck Depression Inventory, CGQOL Cleveland global quality of life questionnaire, CLDQ Chronic liver disease questionnaire, COMPASS Composite Autonomic Symptom Scale, ESS Epworth Sleepiness Scale, EQ. 5D EuroQol EQ. 5D, FFSS Fisk Fatigue Severity Scale, FIS Fatigue Impact Scale, FSFI Female Sexual Satisfaction Index, GSRS Gastrointestinal Symptom Rating Scale, HADS Hospital anxiety and depression scale, HBI Harvey-Bradshaw Index, HHQ Health Habits and History Questionnaires, IBD Inflammatory Bowel Disease, IDS-SR30 30-item Inventory of Depressive Symptomatology-self report, IIEF International index of erectile function, LDH Lifetime drinking history, LDQOL Liver Disease Quality of Life Questionnaire, LT Liver Transplant, MELD Model For End-Stage Liver Disease, MFI Multidimensional Fatigue Inventory, MRS Mayo Risk Score, NIDDK-QA National institute of diabetes and digestive and kidney disease liver transplant questionnaire, NR Not Reported, OS Oresland Scale, PBC-40 Primary Biliary Cirrhosis, PF Pouch Function Questionnaire, PGWB Psychological general well-being index, PHQ-9 Patient Health Questionnaire, PSC PRO Primary Sclerosing Cholangitis patient-reported outcome, RCT Randomised Controlled Trial, SADS Schedule for Affective Disorders and Schizophrenia, SD Standard Deviation, SF-36 Short form 36, SIBDQ Short Inflammatory Bowel Disease Questionnaire, UC Ulcerative Colitis, UCAI UC Activity Index, VAS Visual Analogue Scale, WHOQOL-BREF World Health Organization Quality of Life assessment instrument
aRationale for assessment: A; Burden (HRQOL /symptom) of disease, B: Effectiveness of treatment, C: Cost Effectiveness/Health Utilities, D:Validation of a Patient Reported Outcome Measure, (PROM)