Background
Methods
Search strategy
Inclusion criteria
Selection of studies
Data extraction
Content comparison of included PROMs
Quality assessment
Evidence synthesis
Results
Study selection
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 |
Characteristics of PROMs
PROM | Construct | Therapeutic area | Domains | Total No. of items | Scoring method | Recall period | Administration | Completion time | Data collectiona | Cost & permissionb |
---|---|---|---|---|---|---|---|---|---|---|
15 D © | HRQOL | Generic | Mobility,Vision,Hearing, Breathing, Sleeping, Eating, Speech, Elimination, Usual Activities, Mental function,Discomfort, symptoms, Depression, Distress, Vitality, Sexual Activity | 15 | 1 to 5 levels | Present health status | Self-administered | 5–10 min | PP | A, B |
5-D Itch | Pruritus | Severity of symptoms | Duration, Degree, Direction, Disability, Distribution | 5 | 0–5 (0 being least problematic and 5 most problematic) | Last 2 weeks | Self-administered | < 5 min | PP | Unknown |
BDI | Psychological functioning (incl. coping) | Psychology/ Behaviour | Cognitive-affective, Somatic | 21 | Higher score = greater depression | Last 2 weeks including today | Self-administered/ Interviewer-administered | 5–10 min | E, PP | B,D |
CGQOL | HRQOL | Disease specific (IBD) | Unknown | 3 | 0–1.0 (1 being the best) | Unknown | Unknown | Unknown | PP | Unknown |
CLDQ | HRQOL | Digestive System Diseases | Abdominal symptoms, Fatigue, Systemic symptoms, Activity, Emotional function, Worry | 29 | Higher score = better QoL | Last two weeks | Self-administered | 10 min | E, PP | B,D |
COMPASS | Autonomic nervous system diseases | Signs and symptoms | Orthostatic intolerance, vasomotor, secretomotor, gastrointestinal, bladder and pupilometer | 31 | Higher score = higher autonomic symptom severity | In past year/ past 5 years | Self-administered | No information | PP | No information |
EQ -5D | HRQOL | Generic | Mobility, Self-care, Usual activities, Pain/discomfort, Anxiety/depression | 5 + VAS (20 cm) | Higher score = better QoL | Today | Interviewer-administeredProxy-ratedSelf-administered | A few minutes | E, PP, IVR, T | B,D |
ESS | Sleep disorder | Signs and symptoms | Sleep | 8 | Higher score = higher sleepiness | Over recent times | Self-administered | 2–3 min | E, PP | A,B |
FFSS | HRQOL | Signs & symptoms | Fatigue | 9 | High score = higher fatigue | Past two weeks | Self-administered | < 5 min | E, PP | B,D |
FIS | Symptoms of fatigue | Pathological Conditions, Signs and Symptoms | Cognitive functioning, Physical functioning, Psychosocial functioning | 40 | Lower score = less fatigue | Past four weeks | Self -administered | 10 min | PP | A,B |
FSFI | Signs and symptoms | Female Urogenital Diseases & Pregnancy | Desire, Arousal, Lubrication, Orgasm, Global satisfaction, Pain | 19 | Higher score = better functioning | During the past 4 weeks | Self-administered | Information not found | E, PP | C |
Grading system for fatigue & pruritus | Fatigue and Pruritus | Severity of symptoms | Unknown | Unknown | Pruritus, grades 0 -no, 1-mild, 2- sleep interference,3- substantial sleep disturbance Fatigue, grade 0- no; 1- present, but no interference with activity; 2-extra rest required & activity limited 3- patient unable to work a full day. | Unknown | Unknown | Unknown | Unknown | Unknown |
GSRS | Signs and symptoms | Signs & symptoms, Digestive system diseases | Abdominal pain syndrome, Reflux syndrome, Indigestion syndrome, Diarrhoea syndrome, Constipation syndrome | 15 | Lower score-better QoL | Last week | Self-administered | 10 min | PP | B,D |
HADS | Signs and symptoms | Nervous System Diseases Mental Disorders | Anxiety, Depression | 14 | Lower score = better QoL | In the past week | Self-administered | 2–5 min | E, PP | C |
HHHQ | Diet | Dietary habits | Patient demographics, Education, Medical surgical history and environmental exposure including dietary habits | 370 questions | Unknown | Unknown | Self-report | Unknown | Unknown | Unknown |
IDS-SRS 30 | Signs and symptoms | Psychiatry/Psychology/Behaviour | Vegetative features, Cognitive changes, Mood disturbance, Endogenous symptoms, Anxiety symptoms | 30(28 initial version) | Higher score = higher severity | Past 7 days | Clinical-rated, interviewer-administered, self-administered | 10–15 min | E, IVR, PP | C |
IIEF | HRQOL | Erectile Dysfunction | Erectile function, Orgasmic function, Sexual desire, Intercourse satisfaction, Overall satisfaction | 15 | Higher score = better QoL. Scores by dimension | Past 4 weeks | Self-administered | 15 min | PP | B,D |
LDH | Alcohol consumption patterns | Intake assessment | Consumption levels (quantity), frequency of use, variability in consumption, types of beverages, drinking pattern, solitary versus social drinking, time of the day alcohol consumption | Unclear | Scored by hand or calculator | Unknown | Unknown | 20 min | Unknown | Cost nominal (copyright) |
LDQOL 1.0 | HRQOL | Digestive System Diseases | - Generic core SF-36v2 - Disease-targeted scales: Liver disease-related symptoms, Effects of liver disease, Concentration/Memory, Health distress, Sleep, Loneliness, Hopelessness, Stigma of liver disease, Sexual functioning/problems | 72 | Higher score = Better HRQOL. | The past 4 weeks; Presently (for few items) | Self-administered | 18 (+/− 9) min | PP | D |
MFI | Signs and symptoms | Pathological conditions, signs and symptoms | General fatigue, Physical fatigue, reduced activity, Reduced motivation, Mental fatigue | 20 | Lower score = better QoL | Lately | Self-administered | 5 min | PP | B |
NIDDK-QA | HRQOL | Patients undergoing Liver transplant | Liver disease symptoms, physical functioning, health satisfaction & overall well-being (OWB) | 47 | Higher scores indicate better QOL | Unknown | Unknown | Unknown | Unknown | Unknown |
OS | Functional outcome | IPAA or IRA | Bowel movements, urgency, evacuation difficulties, soiling or seepage, perianal/stomal soreness, protective pad, dietary restrictions and social handicap | Unclear | best 0, worst 15 | Unknown | Unknown | Unknown | Unknown | Unknown |
PBC-27 | HRQOL | Disease specific | Symptoms, Dryness,Itch, Fatigue, Cognitive, Emotional and Social | 40 | Higher scores = greater symptoms impact & poorerHRQOL. | Last four weeks | Self-completion | < 5 min | PP | Unknown |
PBC-40 | HRQOL | Disease specific | Other Symptoms domain, Itch, Fatigue, Cognitive, Social and Emotional | 27 | Higher scores = greater symptoms impact & poorerHRQOL. | Last four weeks | Self-completion | 5 min | PP | Free access |
PedsQL 4.0 | HRQOL | Generic | Physical functioning, Emotional functioning, Social functioning,school functioning | 21 to 23 | Higher score = better QoL | Standard version: past one month. Acute version: past 7 days | Interviewer-administered Proxy-rated Self-administered | 5 min | PP | A,B |
PGWB | HRQOL | Generic | Anxiety, Depression mood, Positive well-being, Self-control, General health, Vitality | 22 | Higher score = better QoL | Standard version = past month/ acute version = last week/ last four weeks | Self-administered/Interviewer-administered | 15 min | PP | |
PHQ-9 | Depression | Severity of depression | Nine questions on symptoms | 10 | Depression severity:1–4: None; 5–9: Mild; 10–14: Moderate, 15–19: Moderately severe, 20 to 27: Severe | over past 2 weeks | Self-completion | 2 to 5 min | PP | Unknown |
Pruritus numerical rating scale | Pruritus | Severity of symptoms | Unknown | Unknown | Numerical rating scale 0–10 (0 for having no symptoms and 10 for having the worst imaginable pruritus) | Unknown | Unknown | Unknown | Unknown | Unknown |
PSC PRO | HRQOL | Disease specific | PSC symptoms, Physical function, Activities of Daily Living, Work Productivity, Role Function, Emotional Impact, Social/Leisure Impact, Q uality of Life, Total Impact of Symptoms | 42 | Module 1: 0–10 scale; Module 2 has 7 four item domains: 1–5 scale, summed within dmains and domain mean summed to give overall impact score | Module 1–24 h recall | Self-administered | 7–15 min | E, PP | Unknown |
Rome ll modular questionnaire | Symptoms | Functional bowel disorder | Esophageal symptoms, Gastroduodenal symptoms, Bowel symptoms, Abdominal pain symptoms, Biliary symptoms and Anorectal symptoms | Unknown | Unknown | Unknown | Unknown | Unknown | Unknown | Unknown |
SADS | Signs and symptoms | Depression | Depressive mood and ideation, Endogenous (ie. Melancholic, vital or vegetative) features, Depressive syndrome, Suicidal ideation and behaviour | 30 | Unknown | Past week only | Unknown | Unknown | Unknown | Unknown |
SF-36 | HRQOL | Generic | Physical Functioning, Role-Physical, Bodily Pain, General Health,Vitality, Social Functioning, Role-Emotional,Mental Health | 36 | 0 to 100, higher score = better health status | Standard version 4 weeks / Acute version 1 week | Self-administered/Interviewer-administered | 5–10 min | E, C, IVR, T, PP | B |
SF-6D | Utilities & Health states | Generic- preference based measure | Physical functioning, role limitation, social functioning, pain, mental health, vitality | Unknown | 0.296-most severe problems 1.0-no problems | Unknown | Unknown | Unknown | Unknown | Unknown |
SIBDQ | HRQOL | Digestive System Diseases | Bowel symptoms, systematic symptoms, Emotional function, Social function | 10 | 1 to 7, higher score = better QOL | Last two weeks | Self-administered/Interviewer-administered | 5 min | E, PP | D |
VAS | Fatigue and Pruritus | Severity of symptoms | Fatigue, Energy, Pruritus | Pruritus: 10 cm line | Pruritus 0 -no pruritus / 10- worst pruritus imaginable | Right now | Self-administered | Vas: Fatigue < 2 min | PP | Free access |
WHOQOL-BREF | HRQOL | Generic | Physical, Psychological, social relationship, Environment, + 2 overall QOL & general health status | 26 | Higher score = better QoL | Last 2 weeks | Interviewer-administered, self-administered | 5 min self-administration, 15–20 min interviewer-administration | PP | D |
Content comparison of included PROMs
Quality assessment
PROM (Author, Year) | Total sample size | PSC sample size | Domains | Test retest reliability (Pearson Correlation) | Internal consistency (Cronbach’s Alpha) |
---|---|---|---|---|---|
NIDDK-QA (Kim, 2000) | 96 | 17 | Liver symptoms men women | 0.94 | Men = 0.94, women =0.87 |
Physical function | 0.99 | 0.88 | |||
Health satisfaction | 0.82 | NR | |||
Overall well being | 0.83 | 0.91 | |||
Time interval of 2 weeks | |||||
NIDDK-QA (Gross, 1999) | 157 | 92 | Symptoms | NR | 0.81 & 0.85 |
Functioning | NR | 0.82 & 0.88 | |||
Index of General Affect (IGA) | NR | 0.91 & 0.93 | |||
PSC PROM (Younossi, 2017) | 102 | Test retest n = 53 Internal consistency n = 155 | PSC Symptoms | 0.84 | 0.89 |
Physical Function | 0.83 | 0.91 | |||
Activities of Daily Living | 0.85 | 0.86 | |||
Work Productivity | 0.7 | 0.93 | |||
Role Function | 0.83 | 0.91 | |||
Emotional Impact | 0.82 | 0.91 | |||
Social/Leisure Impact | 0.8 | 0.93 | |||
Quality of Life | 0.79 | 0.94 | |||
Total Impact of Symptoms | 0.88 |
Internal consistency
Reliability
Validity
Evidence synthesis
Author (Year) | PROM | Internal consistency | Test-retest reliability | Measurement error | Content validity | Structural validity | Hypothesis testing | Criterion validity | Cross structural validity |
---|---|---|---|---|---|---|---|---|---|
Discriminant validity | Concurrent validity | ||||||||
Kim (2000) | NIDDK-QA | Poor | Poor | NR | NR | NR | Poor | Poor | NR |
Gross (1999) | NIDDK-QA | Poor | NR | NR | NR | NR | NR | NR | NR |
Younossi, (2017) | PSC PROM | Fair | Fair | NR | Excellent | Fair | NR | NR | NR |
PROM | Internal consistency | Test-retest reliability | Measurement error | Content validity | Structural validity | Hypothesis testing | Criterion validity | Responsiveness |
---|---|---|---|---|---|---|---|---|
Discriminant validity | Concurrent validity | |||||||
NIDDK-QA | ? | ? | NR | NR | NR | ? | ? | NR |
PSC PROM | + | + | NR | + | + | NR | NR | NR |