Appendix 9Health-related quality-of-life systematic review: data extraction forms
Reference
Study characteristics
Research question
What are the stated objectives of the study?
To evaluate the effectiveness of multidisciplinary foot-care, and to evaluate the methodological considerations of a trial of multidisciplinary care in JIA.
Describe the type of study and study design.
Exploratory randomised controlled trial
Was the sample from (1) the general population; (2) patients with the disease of interest; (3) individuals with knowledge of the disease; (4) other?
Are inclusion/exclusion criteria clearly described? Do these exclude any individuals that may be relevant (e.g. > 80 years)?
The sample was drawn from patients with the disease of interest (i.e. children and adolescents with a definitive diagnosis of JIA and inflammatory joint disease affecting the foot/ankle).
The inclusion/exclusion criteria were clearly stated; however, might exclude a proportion of individuals with the disease of interest but whose disease has not affected the foot/ankle.
Patients were included if they satisfied at least one of the following: (1) previously documented arthritis in the foot including small joints derived from medical case notes; (2) previously documented foot arthritis in one or more large joints derived from medical case notes; or (3) current widespread polyarthritis involving large and small foot joints derived from clinical examination by a consultant paediatric rheumatologist. Patients with an unconfirmed diagnosis of JIA, and/or only upper limb, jaw, or neck involvement were excluded.
What are the characteristics of the baseline cohort for the evaluation?
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| Age, years, mean (SD) |
| Intervention arm | 10.1(4.22) |
| Control arm | 10.0(3.39) |
| Male/female, n |
| Intervention arm | 7/14 |
| Control arm | 6/17 |
| Race (if appropriate) | NR |
| Disease subtypes, n (%) |
| Intervention arm |
| Persistent oligoarthritis | 7 (33) |
| EO | 4 (19) |
| Polyarthritis RF–ve | 6 (29) |
| Polyarthritis RF+ve | 0 (0) |
| Psoriatic arthritis | 2 (10) |
| ERA | 2 (10) |
| Undifferentiated | 0 (0) |
| Control arm |
| Persistent oligoarthritis | 4 (17) |
| EO | 5 (22) |
| Polyarthritis RF–ve | 10 (43) |
| Polyarthritis RF+ve | 2 (9) |
| Psoriatic arthritis | 1 (4) |
| ERA | 0 (0) |
| Undifferentiated | 1 (4) |
| Sample size, n |
| Intervention arm | 21 |
| Control arm | 23 |
| Pharmacological management, n (%) |
| Intervention arm |
| Analgesics | 2 (9) |
| NSAIDs | 18 (86) |
| Methotrexate | 7 (33) |
| Etanercept | 1 (5) |
| Sulphasalazine | 0 (0) |
| Rituximab | 5 (24) |
| Control arm |
| Analgesics | 3 (13) |
| NSAIDs | 16 (70) |
| Methotrexate | 5 (22) |
| Etanercept | 0 (0) |
| Sulphasalazine | 1 (4) |
| Rituximab | 5 (22) |
| Combination methotrexate and etanercept | – |
| QoL instrument | EQ-5D-Y (patients) and EQ-5D-3L (parents/guardians) questionnaires |
| Utility values (Y/N) | Y |
| Treatment effect, if reported | Both the treatment groups appeared to improve by one point on the JAFI impairment scale between baseline and 12 months follow up, however, the differences between groups for change scores did not reach statistical significance |
Country/setting
What is the country and setting for the evaluation?
Royal Hospital for Sick Children, Glasgow, UK.
Data sources
Effectiveness
Were the QoL data derived from: a single (observational) study, a review/synthesis or combination of previous studies, expert opinion?
This single exploratory RCT.
Results
Summarise the results.
There were no significant differences between treatment groups for secondary outcomes at final follow-up.
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| Intervention arm | Control arm |
|---|
| Baseline |
|---|
| Self EQ-5D utility index, mean (SD) | 0.57 (0.31) | 0.58 (0.35) |
| Self EQ-5D utility index, median (IQR) | 0.62 (0.52–0.76) | 0.66 (0.52–0.75) |
| Proxy EQ-5D utility index, mean (SD) | 0.69 (0.29) | 0.60 (0.33) |
| Proxy EQ-5D utility index, median (IQR) | 0.69 (0.58–1) | 0.62 (0.55–0.82) |
| Change at 12 months |
|---|
| Self EQ-5D utility index, median (IQR) | 0 (–0.1 to 0.01) | 0 (–0.04 to 0.04) |
| Proxy EQ-5D utility index, median (IQR) | 0 (0–0.11) | 0 (0–0.1) |
Were the methods for deriving these data adequately described (give sources if using data from other published studies)? (Was a valid preference based instrument used to describe health states, such as EQ-5D? Was the valuation of health states from the UK general population?)
A valid preference-based instrument was used: EQ-5D-Y and EQ-5D-3L.
Are the levels of missing data reported? How are they dealt with?
For missing data identified at the end of the study, a sensitivity analysis was performed in order to identify the most appropriate method to address this problem (LOCF, mean value imputation, maximum value imputation, minimum value imputation and random value imputation). LOCF was found to be the most conservative method while being less labour intensive; therefore, it was subsequently used to impute all missing data at final follow-up.
Mapping
If a model was used, describe the type of model (e.g. regression) or other conversion algorithm.
Conclusions/implications
Give a brief summary of the author’s conclusions from their analysis.
Integrated multidisciplinary foot care did not result in a significant reduction in disease-related foot impairments and disability.
What are the implications of the study for the model?
In both arms, a proportion of participants received etanercept, so the utility values reported cannot be used in the model for baseline HRQoL with standard of care.
Reference
Prince et al. (2011),124 Prince et al. (2010)137
Study characteristics
Research question
What are the stated objectives of the study?
To evaluate changes in HRQoL in patients with refractory JIA who are being treated with etanercept.
Describe the type of study and study design.
Was the sample from: (1) the general population; (2) patients with the disease of interest; (3) individuals with knowledge of the disease; (4) other?
Are inclusion/exclusion criteria clearly described? Do these exclude any individuals that may be relevant (e.g. > 80 years)?
JIA patients younger than 18 years treated with etanercept.
What are the characteristics of the baseline cohort for the evaluation?
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| Study | Prince et al. (2010)137 | Prince et al. (2011)124 |
|---|
| Age | 11.9 years (IQR 8.1–14.9) | 11.6 years (IQR 7.9–14.9) |
| Sex, n (%) |
Male
Female | 20 (38)
33 (62) | 20 (41)
33 (59) |
| Ethnicity (if appropriate) | | |
| Indication/disease, n (%) | Systemic 14 (26)
Polyarticular RF+ve 5 (9)
Polyarticular RF–ve 18 (34)
EO 11 (21)
ERA 2 (4)
Juvenile PA 3 (6) | Systemic 11 (22)
Polyarticular RF+ve 4 (8)
Polyarticular RF–ve 18 (37)
EO 11 (22)
ERA 2 (4)
Juvenile PA 3 (6) |
| Other characteristics (sample size) | Sample size 53
Median disease duration JIA (years) at start of etanercept 3.0 | Sample size 49
Median disease duration JIA (years) at start of etanercept 3.6 |
| Quality-of-life instrument | HUI3 | HUI3 |
| Utility values (Y/N) | Yes | Yes |
| Treatment effect, if reported | Significant improvements were shown after 3 months and these continued at least up to 27 months | Significant improvements were shown after 3 months and these continued at least up to 27 months |
Country/setting
What is the country and setting for the evaluation?
Data sources
Effectiveness
Were the QoL data derived from: a single (observational) study, a review/synthesis or combination of previous studies, expert opinion?
Single prospective study.
Results
Summarise the results.
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| Prince et al. (2010)137 | Baseline | 3 months | 15 months | 27 months |
|---|
| HUI3 mean (SE) | 0.53 (0.04) | 0.69 (0.05) | 0.74 (0.06) | 0.78 (0.07) |
Were the methods for deriving these data adequately described (give sources if using data from other published studies)? (Was a valid preference based instrument used to describe health states, such as EQ-5D? Was the valuation of health states from the UK general population?)
Are the levels of missing data reported? How are they dealt with?
Mapping
If a model was used, describe the type of model (e.g. regression) or other conversion algorithm.
Conclusions/implications
Give a brief summary of the author’s conclusions from their analysis.
This study shows that the HRQoL of patients with refractory JIA can be substantially improved by the use of etanercept.
What are the implications of the study for the model?
This is a potential source of HRQoL for the SHTAC economic model.
