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Table 1 Characteristics of included studies

From: Patient-reported outcome measures used in patients with primary sclerosing cholangitis: a systematic review

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
  1. 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
  2. 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)