OBJECTIVE:To explore patients' perspectives on the impact of appearance changes attributed to glucocorticoid treatment. METHODS:A secondary inductive thematic analysis was conducted of the 'Steroid PRO' semi-structured interviews with patients with rheumatic conditions receiving glucocorticoids in the UK, USA and Australia. RESULTS:Sixty patient interviews were analysed. Patient age was 26-84 years; 39 (65%) were female; and the patients had systemic vasculitis (n = 19), inflammatory arthritis (n = 14), crystal arthropathy (n = 2), connective tissue disorders (n = 16) or other/multiple (n = 9). In addition to participants expressing the need for more information and support, three over-arching themes were identified: (i) societal norms ['I think my main concern, particularly being female, was the weight gain that the steroids had' (female aged 26-30)]; these included real or perceived expectations to which participants felt pressure to conform, which were sustained through interactions with others; (ii) harms to mental health and sense of self ['It makes you feel down. It makes you feel depressed. You don't want to socialize because you're not you' (male aged 61-65)]; glucocorticoids were described as making participants 'not look like' themselves, associated with changes in mood and self-confidence; and (iii) burden of adjustments ['I have a wardrobe right now that goes four different sizes' (female aged 51-55)]; other adjustments related to diet, exercise, work, hobbies, activities of daily living and key life events. CONCLUSION:Patients attribute a variety of impacts on their quality of life to glucocorticoid-related appearance changes. We suggest ways to meet patients' needs for information and support, which can be developed through further work.

Background/Aims Glucocorticoids (GCs) used in the treatment of inflammatory rheumatic conditions can impact on patients in different ways. The objective of this study was to identify socio-demographic and clinical factors associated with health-related quality of life (HRQoL) in patients undertaking GCs for a rheumatic disease. Methods This was a cross-sectional study using online survey methods. Participants taking GCs for a rheumatic disease from Australia, United Kingdom and the United States of America were included. HRQoL was measured using the EQ-5D-5L converted into a linear EQ5D index value. Eight explanatory factors (age, sex, country, educational level, employment status, disease group, self-reported disease state, and dose of GCs) were tested for potential association with HRQoL using univariable and multivariable (hierarchical) analyses. Factors found to have a significant association with EQ5D index at a P < 0.05 level in the multivariable model were considered to be independently associated with HRQoL. Results A total of 945 patients completed the EQ-5D-5L with no missing values: UK n = 742 (79%); USA n = 139 (15%); Australia/New Zealand n = 64 (7%); mean age 57.6 (SD = 13.6); 833 (88%) women. Participants with inflammatory arthritis n = 197 (21%), connective tissue disease and/or vasculitis n = 402 (43%), giant cell arteritis and/or polymyalgia rheumatica n = 346 (37%) were included. Those with self-reported active disease were 726 (77%). Mean (SD) EQ5D index was 0.64 (0.25); there were significant differences between groups in EQ-5D-5L index by age (median split, t=-3.77, p < 0.001); sex (t = 3.40, p < 001); educational level (F[3]=10.51, p < 0.001); employment status (F[6]=37.67, P < 0.001); disease group (T[2]=20.23, p < 0.001); disease state (T=-8.46, p < 0.001); and the dose of GCs(T = 2.95, p < 0.001). Worse HRQoL was associated with older age, being female, low educational attainment, being unemployed, having an inflammatory arthritis or a CTD, disease being active, and taking a higher dose of GC. Of the eight factors that were tested in the multivariable model, five were found to be independently associated with HRQoL. These were: disease state (beta = 0.12, p < 0.001), disease group (beta = 0.06, p < 0.001), employment status (beta = 0.05, p < 0.001), sex (beta = 0.05, p < 0.001), and educational level (beta = 0.04, p < 0.001). This 5-factor model explained 18% of the variance in EQ5D index scores (F[1, 926] = 4.97, P = 0.026). The excluded factors in the model were age, dose, and country. Conclusion In our data, we identified 5 factors independently associated with worse HRQoL in patients being treated with GCs. Over 80% of the variance was not explained by our model likely due to use of a generic HRQoL measure (EQ-5D-5L) as outcome variable, and the small number of explanatory variables tested within this survey. Further research is required to identify potential targets to support interventions.

Background/Aims To explore patients' perspectives on the impact of appearance changes attributed to glucocorticoid treatment. Methods We conducted a secondary analysis of semi-structured interviews with patients with rheumatic conditions receiving glucocorticoids in the UK, USA, and Australia. Interview data were analysed inductively to develop preliminary individual and overarching themes. Results 60 patient interviews were analysed. Patients ranged from 26-84 years, and 39 (65%) were female. Patients had diagnoses of systemic vasculitis (n=19), inflammatory arthritis (n=14), crystal arthropathy (n=2), connective tissue disorders (n=16), or other/multiple (n=9). Interviews contained rich data on how appearance changes impacted health-related quality of life. Three preliminary overarching themes were identified. i) Societal influences: participants reported pressure to conform to cultural and societal ideals: "I think with the weight gain it was more of an emotional response. [] There's a part of you that's thin is better. So, I think that impacted me the most, just because of that aspect of what others thought of me..." (Female [Mixed/Multiple ethnicity] aged 27, granulomatosis with polyangiitis [Australia]) and "...within a week my dad had seen me and said, "Gosh you've put on a lot of weight". Obviously, that's not what I want to hear [] but I think it was just pure shock." (White female aged 40, eosinophilic granulomatosis with polyangiitis [UK])".ii)?Impact on mental health and sense of self: glucocorticoids were often described as making participants 'not look like' themselves, associated with changes in mood, self-confidence, and reluctance to socialise: "It makes you feel down. It makes you feel depressed. You don't want to socialise because you're not you." (White male aged 65, inflammatory myositis [Australia]) and "Yeah, that's it and it will hopefully look nice, which is a really vain thing to think about isn't it, but it does really affect [] how you feel and kind of your state of mind and things..." (White female aged 39, Takayasu arteritis [UK]). iii) Burden of adjustments included dieting, purchasing additional clothing sizes, working from home, discontinuing hobbies, and postponing key life events: "I have a wardrobe right now that goes four different sizes as my weight goes up and down and up and down." (White female aged 55, systemic lupus erythematosus [USA]) and "Well, with the high dose of prednisone since I gained weight, I get really tired if I try to do like exercise or something. And I used to be very sporty, so that just completely changed." (Hispanic female aged 29, systemic lupus erythematosus [USA]). Conclusion Patients attribute a variety of impacts on their health-related quality of life to glucocorticoid-related appearance changes and report a burden of adjustments above management of their chronic rheumatic condition. Further work is required to establish ways to mitigate these through improved information provision and support. Disclosure S.J. Lax: Grants/research support; post funded by National Institute for Health and Care Research Advanced Fellowship [grant number NIHR300863]. E. Dures: Grants/research support; recipient of grants from Above & Beyond Bristol Hospitals Charity, UWE Bristol, Vifor Pharma, National Institute for Health and Care Research (Research for Patient Benefit) and Sanofi. S. Bridgewater: None. C. Silverthorne: None. V. Lowndes: None. P. Richards: None. J. Dawson: None. C. Hill: Grants/research support; recipient of an Australian Rheumatology Association Project Grant, administered by Arthritis Australia. S. Goodman: None. S.L. Mackie: None. M.E. Ndosi: None. F.A. Pearce: Grants/research support; recipient of a National Institute for Health and Care Research Advanced Fellowship [grant number NIHR300863] and a grant from Vifor pharma. J. Robson: Consultancies; consultancy and speaking fees from Vifor Pharma. Grants/research support; recipient of grants from Above & Beyond Bristol Hospitals Charity, UWE Bristol, Vifor Pharma, National Institute for Health and Care Research (Research for Patient Benefit) and Sanofi.

Objectives GCA is systemic vasculitis manifesting as cranial, ocular or large vessel vasculitis. A prior qualitative study developed 40 candidate items to assess the impact of GCA on health-related quality of life (HRQoL). This study aimed to determine final scale structure and measurement properties of the GCA patient reported outcome (GCA-PRO) measure. Methods Cross-sectional study included UK patients with clinician-confirmed GCA. They completed 40 candidate items for the GCA-PRO at times 1 and 2 (3 days apart), EQ-5D-5L, ICECAP-A, CAT-PROM5 and self-report of disease activity. Rasch and exploratory factor analyses informed item reduction and established structural validity, reliability and unidimensionality of the final GCA-PRO. Evidence of validity was also established with hypothesis testing (GCA-PRO vs other PRO scores, and between participants with 'active disease' vs those 'in remission') and test-retest reliability. Results The study population consisted of 428 patients: mean (s.d.) age 74.2 (7.2), 285 (67%) female; 327 (76%) cranial GCA, 114 (26.6%) large vessel vasculitis and 142 (33.2%) ocular involvement. Rasch analysis eliminated 10 candidate GCA items and informed restructuring of response categories into four-point Likert scales. Factor analysis confirmed four domains: acute symptoms (eight items), activities of daily living (seven items), psychological (seven items) and participation (eight items). The overall scale had adequate Rasch model fit (chi(2) = 25.219, degrees of freedom = 24, P = 0.394). Convergent validity with EQ5D-5L, ICECAP-A and Cat-PROM5 was confirmed through hypothesis testing. Internal consistency and test-retest reliability were excellent. Conclusion The final GCA-PRO is a 30-item, four-domain scale with robust evidence of validity and reliability in measuring HRQoL in people with GCA.

Background: Glucocorticoids (GCs) play a pivotal role in the management of inflammatory rheumatic conditions but they can adversely impact on patients in different ways. Conversely, there are certain patient characteristics that may be correlated with a poorer health-related quality of life (HRQoL). Objectives: The objective of this study was to identify socio-demographic and clinical factors associated with HRQoL in patients undertaking GCs for a rheumatic disease. Methods: This was a cross-sectional study using online survey methods. Participants taking GCs for a rheumatic disease from Australia/New Zealand (AU/NZ), United Kingdom (UK) and the United States of America (USA) were included. HRQoL was measured using the generic EuroQoL (EQ-5D-5L) converted into a linear EQ5D index value. Eight explanatory factors (age, sex, country, educational level, employment status, disease group, self-reported disease state, and dose of GCs) were tested for potential association with HRQoL using univariable and multivariable (hierarchical) analyses. Factors found to have a significant association with EQ5D index at a p<0.05 level in the multivariable model were considered independently associated with HRQoL. Results: A total of 945 patients completed the EQ-5D-5L with no missing values: UK n=742 (79%), USA n=139 (15%), AU/NZ n=64 (7%); mean age was 57.6 (SD=13.6); 833 (88%) were women. Participants with self-reported active disease were 726 (77%). Those with inflammatory arthritis were 197 (21%), connective tissue disease and/or vasculitis were 402 (43%), giant cell arteritis and/or polymyalgia rheumatica were 346 (36%).Mean (SD) EQ5D index was 0.64 (0.25); There were significant differences between groups in age (median split, t=-3.77, p<0.001); sex (t=3.40, p<0.01); educational level (F[3]=10.51, p<0.001); employment status (F[6]=37.67, p<0.001); disease group (T[2]=20.23, p<0.001); disease state (T=-8.46, p<0.001); and the dose of GCs (T=2.95, p<0.001). Worse HRQoL was associated with older age, being female, low educational attainment, being unemployed, having an inflammatory arthritis or a CTD, disease being active, and taking a higher dose of GCs. Five factors were identified as being independently associated with worse HRQoL: disease state (β=0.12, p<0.001), disease group (β=0.06, p<0.001), employment status (β =0.05, p<0.001), sex (β =0.05, p<0.001), and educational level (β =0.04, p<0.001). Conclusion: We identified 5 factors which were independently associated with worse HRQoL in patients being treated with GCs. Over 80% of the variance was not explained by our model likely due to use of a generic HRQoL measure (EQ-5D-5L) as outcome, and the small number of explanatory factors tested within this survey. Further research is required to identify potential targets to support interventions. REFERENCES: NIL. Acknowledgements: NIL. Disclosure of Interests: None declared.Figure 1EQ-5D-5L proportions reported by dimensions dichotomised into no problem or any problems. Disease activity was patient‘s self-report of active or inactive disease. Table 1EQ-5D-5L frequencies in the 3 disease categories, reported by dimensions dichotomised into no problem or any problems.IA (n=197) (%)CTD/vasculitis(n=402) (%)GCA/PMR(n=346) (%)Chi-Square (p-value)MobilityNo problem18 (9.2)109 (27.1)101 (29.2)30.987 (<0.001)Any problems179 (90.9)293 (72.9)245 (70.8)Self-careNo problem57 (28.9)220 (54.7)232 (67.1)73.604 (<0.001)Any problems140 (71.1)182 (45.3)114 (32.9)ActivityNo problem17 (8.7)59 (14.7)65 (18.8)10.234 (0.006)Any problems180 (91.4)343 (85.3)281 (81.2)Pain/DiscomfortNo problem6 (3.1)48 (11.9)27 (7.8)13.833 (<0.001)Any problems191 (97.0)353 (87.8)319 (92.2)Anxiety/DepressionNo problem54 (27.4)89 (22.1)120 (34.7)14.587 (<0.001)Any problems143 (72.6)313 (77.9)226 (65.3)

The Glucocorticoid Toxicity Index (GTI) is a composite instrument designed to capture change in glucocorticoid-related morbidity over time. 1 Stone JH McDowell PJ Jayne DRW et al. The Glucocorticoid Toxicity Index: measuring change in glucocorticoid toxicity over time. Semin Arthritis Rheum. 2022; 55152010 Crossref PubMed Scopus (12) Google Scholar It was developed through consensus methods and multicriteria decision making among 19 medical specialists, with relative domain weights decided via clinician consensus. 2 Miloslavsky EM Naden RP Bijlsma JW et al. Development of a Glucocorticoid Toxicity Index (GTI) using multicriteria decision analysis. Ann Rheum Dis. 2017; 76: 543-546 Crossref PubMed Scopus (113) Google Scholar The GTI has now been used in more than 45 studies, including 12 phase 3 clinical trials. 1 Stone JH McDowell PJ Jayne DRW et al. The Glucocorticoid Toxicity Index: measuring change in glucocorticoid toxicity over time. Semin Arthritis Rheum. 2022; 55152010 Crossref PubMed Scopus (12) Google Scholar Glucocorticoid Toxicity Index scores by domain in patients with antineutrophil cytoplasmic antibody-associated vasculitis treated with avacopan versus standard prednisone taper: post-hoc analysis of data from the ADVOCATE trialReplacing a standard prednisone taper with avacopan in patients with ANCA-associated vasculitis reduced glucocorticoid toxicity in multiple GTI domains. For individual patients, glucocorticoid toxicity was often nuanced, improving in some domains while worsening in others. These findings emphasise the value of a composite measure of glucocorticoid toxicity that quantifies cumulative worsening and aggregate change directly. Full-Text PDF

Abstract Background/Aims Systemic glucocorticoids are key in the management of life- and organ-threatening rheumatic diseases, but have wide-ranging adverse effects of concern to patients and clinicians. We aimed to validate a Patient Reported Outcome (PRO) measure (the Steroid PRO) for assessing the impact of glucocorticoids on health-related quality of life (HRQoL) in patients receiving glucocorticoids for rheumatic disease. Methods This was a cross-sectional study of adults from the UK, USA, Australia and New Zealand, taking glucocorticoids for a rheumatic disease. An online survey was conducted at two time points: Time 1: (i) demographics - age, gender, country, ethnicity, educational level; (ii) clinical information - diagnosis, glucocorticoid dose; (iii) 40 candidate items for the Steroid-PRO, developed in a previous qualitative study; (iv) EQ5D-5L. Time 2 (optional, completed 3-5 days later): (i) the Steroid PRO candidate items; (ii) a condition change question. The survey was distributed via social media and patient organisations. After descriptive analysis, iterative testing with Rasch measurement model and exploratory factor analysis (EFA) informed item reduction and established structural validity, reliability and unidimensionality of the final Steroid-PRO. Item reduction was based on clinical importance, lack of fit to the Rasch model, and redundancy. Evidence of discriminative validity of the Steroid-PRO was established by comparing its scores for participants receiving lower dose glucocorticoid (up to 10 mg) versus higher dose (>10 mg). Intraclass correlation (ICC) between Time 1 and Time 2 was calculated for patients who reported ‘no change’ compared with 3-5 days ago. Results A total of 946 patients returned complete responses at Time 1 (Time 2: 447 responses). They were from UK, n = 743 (78.5%); USA, n = 139 (14.7%); Australia, n = 59 (6.2%). Their mean age was 57.6 (SD = 13.6); 833 (88%) were women. They were treated for inflammatory arthritis (n = 194), connective tissue disease and/or vasculitis (n = 398), and giant cell arteritis and/or polymyalgia rheumatica (n = 341). A total of 25 items were removed due to floor effects and lack of fit to the Rasch model. The remaining 15 items showed a satisfactory fit to the model. EFA suggested a scale structure with four domains: Participation (4 items), Appearance (3 items), Psychological (5 items), and Treatment concerns (3 items). This 4-domain structure was supported by the Rasch model, confirming construct validity; χ2=47.82 (DF = 36), p = 0.899; and reliability (Person Separation Index, PSI=0.757). Patients on a lower glucocorticoid dose had lower Steroid-PRO domain scores than those receiving higher dose (discriminant validity). ICC between Time 1 and Time 2 ranged from 0.892 to 0.942 (95%CI 0.868 to 0.953) suggesting excellent (test-retest) reliability. Conclusion The final Steroid-PRO is a 15-item, valid and reliable 4-domain scale measuring the impact of glucocorticoid therapy on HRQoL of people with rheumatic diseases. Disclosure S. Bridgewater: Grants/research support; S.B. has received funding for research from Vifor Pharma. M. Ndosi: Grants/research support; M.N. has received funding for research from Vifor Pharma. J. Dawson: None. P. Richards: None. C. Silverthorne: None. E. Dures: Grants/research support; E.D. has received funding for research from Vifor Pharma. S. Goodman: Consultancies; S.G. reports consultancy for UCB. Grants/research support; S.G. has received research support from Novartis. Other; S.G. has given talks for NYU, Case Western, ACR Rheumatology Courses. C. Hill: None. S.L. Mackie: Consultancies; S.L.M. has provided consultancy on behalf of her institution for Roche/Chugai, Sanofi, AbbVie, AstraZeneca. Grants/research support; S.L.M. has been an investigator on clinical trials for Sanofi and GSK, as a research grant co-applicant, S.L.M. has received research funding (partial salary support) from Vifor, paid to her institution. Other; S.L.M. has given talks on behalf of her institution for Pfizer, Vifor and UCB. In all cases fees were paid to her institution and no personal fees were received, S.L.M. was supported by Roche to attend EULAR2019 and by Pfizer to attend ACR2021 virtually. J.C. Robson: Consultancies; J.C.R. reports: Consultancy for Vifor Pharma. Grants/research support; Funding for research from Vifor Pharma. Other; Speaking fees from Vifor Pharma.

Objectives Glucocorticoids used in the treatment of inflammatory rheumatic conditions can impact on health-related quality of life. An underpinning qualitative study developed a long-list of candidate items for a treatment-specific patient-reported outcome (PRO) measure. The objective of this paper is to determine scale structure and psychometric properties of the Steroid PRO. Methods A cross-sectional survey of adults from the UK, USA, Australia and New Zealand, taking glucocorticoids for a rheumatic disease. Initial survey collected demographics, clinical information, 40 Steroid PRO candidate items and EuroQol-5 Dimensions- 5 levels (EQ-5D-5L). Follow-up, 3–5 days later, collected Steroid PRO candidate items and a condition-change (‘transition’) question. Analysis included Rasch measurement model, exploratory factor analysis (EFA), and hypothesis testing for discriminative validity, convergence validity and test–retest reliability. Results Total responses 946: UK n=743 (79%); USA n=139 (15%); Australia/New Zealand n=64 (7%); mean age 57.6 (SD=13.6); 833 (88%) women. Participants with inflammatory arthritis n=197 (21%), connective tissue disease and/or vasculitis n=402 (42%), giant cell arteritis and/or polymyalgia rheumatica n=347 (37%). Twenty-five items were removed due to lack of fit to Rasch model. Of the remaining items, EFA suggested four subscales: Social impact (4 items); Impact on appearance (3 items); Psychological impact (5 items); Treatment concerns (3 items). Rasch modelling supported a four-subscale structure and total score, confirming construct validity and reliability. Hypothesis testing confirmed discriminant and convergence validity. Intraclass correlation coefficient (total score) was 0.809 demonstrating excellent (test–retest) reliability. Conclusions The Steroid PRO is a 15-item, valid and reliable scale for measuring the impact of glucocorticoid therapy in people with rheumatic diseases.

Aims:The aim of this study was to capture 12-month outcomes from a representative multicentre cohort of patients undergoing total ankle arthroplasty (TAA), describe the pattern of patient-reported outcome measures (PROMs) at 12 months, and identify predictors of these outcome measures. Methods:Patients listed for a primary TAA at 19 NHS hospitals between February 2016 and October 2017 were eligible. PROMs data were collected preoperatively and at six and 12 months including: Manchester-Oxford Foot and Ankle Questionnaire (MOXFQ (foot and ankle)) and the EuroQol five-dimension five-level questionnaire (EQ-5D-5L). Radiological pre- and postoperative data included Kellgren-Lawrence score and implant position measurement. This was supplemented by data from the National Joint Registry through record linkage to determine: American Society of Anesthesiologists (ASA) grade at index procedure; indication for surgery, index ankle previous fracture; tibial hind foot alignment; additional surgery at the time of TAA; and implant type. Multivariate regression models assessed outcomes, and the relationship between MOXFQ and EQ-5D-5L outcomes, with patient characteristics. Results:Data from 238 patients were analyzed. There were significant improvements in MOXFQ and EQ-5D-5L among people who underwent TAA at six- and 12-month assessments compared with preoperative scores (p < 0.001). Most improvement occurred between preoperative and six months, with little further improvement at 12 months. A greater improvement in MOXFQ outcome postoperatively was associated with older age and more advanced radiological signs of ankle osteoarthritis at baseline. Conclusion:TAA significantly benefits patients with end-stage ankle disease. The lack of substantial further overall change between six and 12 months suggests that capturing PROMs at six months is sufficient to assess the success of the procedure. Older patients and those with advanced radiological disease had the greater gains. These outcome predictors can be used to counsel younger patients and those with earlier ankle disease on the expectations of TAA.

Abstract Background/Aims Giant cell arteritis (GCA) presents in people over the age of 50 with cranial, ocular, and large vessel vasculitis. This study aims to validate a disease-specific patient-reported outcome measure (PROM) for patients with GCA, to capture the impact of GCA and its treatment on health-related quality of life. Methods This cross-sectional study included UK patients with clinician-confirmed GCA; diagnosed within the last three years or flaring within the last year. Patients completed the 40 candidate PROM items, the EQ5D-5L, CAT-PROM5 and self-report of disease activity. Rasch and factor analysis were used to determine internal validity and factor structure. Item reductions were based on clinical importance, Rasch model fit, and redundancy. Tests of validity included comparison of the GCA-PROM (i) in participants with ‘active disease’ versus patients ‘in remission’ (known groups validity) and (ii) with EQ5D-5L and CAT-PROM5 scores (convergent validity). Results The survey included 428 patients, mean age (SD) of 74.2 (7.2), 285 (67%) female. 327 (76%) cranial GCA, 114 (26.6%) large vessel vasculitis and 142 (33.2%) ocular involvement. Positive diagnostic tests included 167 (39%) temporal artery biopsy, 177 (41.4%) temporal artery ultrasound, and 51 (11.9%) Positron Emission Tomography and Computed Tomography (PET-CT). Active disease was reported in 197 (46%). After the initial analysis (40 items), ten items were deleted, and two response categories collapsed to ensure overall fit to the Rasch model. This resulted in a final PROM comprising a 30-item scale with a 4-response category structure. Factor analysis confirmed four factors (domains): Acute symptoms (8 items), Activities of daily living (7 items), Psychological (7 items) and Participation (8 items), all of which individually fitted the Rasch model (X2 = 25.219, DF = 24, p = 0.394 including reliability [Person Separation Index, PSI=0.828]), (construct validity). Each domain correlated, at least moderately, with EQ5D-5L and CAT-PROM5 scores (Spearman’s Correlation Coefficients 0.44 to 0.78) (convergent validity). The new GCA-PRO discriminated between patients with active disease and remission (known groups validity) (Table 1). Conclusion The 30-item GCA-PROM demonstrates internal and external validity in measuring health-related quality of life in people with GCA. Disclosure M. Ndosi: None. C. Almeida: None. J. Dawson: None. E. Dures: None. R. Greenwood: None. C. Guly: None. S. Mackie: Honoraria; Sept 2021: Roche/Chugai payment to my institution for delivering a talk on giant cell arteritis at an educational day. Grants/research support; MRC Confidence in Concept scheme, 2021 Grant to develop early diagnosis methods for giant cell arteritis, 2018: MRC Proximity to Discovery: Protein biomarkers for polymyalgia rheumatica and giant cell arteritis (PI: Morgan, University of Leeds), 2019: Roche: giant cell arteritis tocilizumab registry (PI: Morgan, University of Leeds). Other; Site Sub-Investigator on clinical trial in giant cell arteritis for Roche, Site Principal Investigator on clinical trial in giant cell arteritis for GSK, Site Principal Investigator and UK Chief Investigator on clinical trials (giant cell arteritis and polymyalgia rheumatica) for Sanofi. A. Bromhead: None. S. Stern: None. J.C. Robson: None.

Background Giant cell arteritis (GCA) is caused by inflammation of the blood vessels of the head and neck; patients can present with cranial, ocular or large vessel vasculitis involvement. Treatment is with glucocorticoids, steroid sparing agents and biologics to control inflammation and protect sight. Objectives The aim of this study was to produce a validated disease specific PROM for patients with GCA, to capture the impact of GCA and its treatment on health-related quality of life. Methods Patients with clinician- confirmed GCA from the UK, either diagnosed in the last three years or with a flare in the last year, were included in the survey. A longlist of 40 candidate questionnaire items, each with a 5-point Likert scale, had previously been developed, based on a qualitative study with patients from the UK and Australia [1]. In this cross-sectional survey, patients completed the 40-item draft GCA-PROM alongside EQ5D-5L, CAT-PRO5 and self-report of GCA disease activity. Rasch and factor analysis were used in an iterative manner to determine the underlying construct validity of the new PROM. Items were fitted to the Rasch model to determine its construct validity, reliability, unidimensionality and statistical sufficiency of the total score from the scale. Factor analysis was used to establishing factor structure. Item reduction decisions were be based on clinical importance, lack of fit to the Rasch model, and redundancy detected during principal component analysis. External validity was tested by comparing the scores of the newly validated GCA-PROM (i) in participants who self-identify as having ‘active disease’ versus patients ‘in remission’ (known groups validity) (ii) with scores derived from EQ5D-5L and CAT-PRO5 (convergent validity). Results The survey included 428 patients; 327 (76%) cranial GCA, 114 (26.6%) large vessel vasculitis and 142 (33.2%) ocular involvement. 285 (67%) of participants were female with a mean age (SD) of 74.2 (7.2). 167 (39%) temporal artery biopsies and 177 (41.4%) temporal artery ultrasounds, and 51 (11.9%) Positron Emission Tomography and Computed Tomography (PET-CT)s were reported as positive. 108 (25%) received second-line immunosuppressants, and 34 (7.9%) anti-IL6 therapy. Active disease was reported in 197 (46%). Four factors (domains) were identified after deletion of 10 redundant items: Acute symptoms (8 items), Activities of daily living (7 items), Psychological (7 items) and Participation (8 items). The four domains were analysed as ‘super-items’ and shown to fit the Rasch model. The overall scale had an adequate fit to the Rasch model: X 2 = 25.219, DF=24, p=0.394 including reliability PSI=0.828. The raw-to-linear transformation scale was calibrated to enable parametric analyses if desired. Each domain was shown to have known-groups validity (p<0.001 patients reporting active versus inactive disease) and correlation with EQ5D-5L and CAT-PRO5 (Rs) ranging between 0.4.42 and 0.778. Conclusion The GCA-PROM is a new patient reported outcome measure for patients with GCA which demonstrates good internal and external validity. References [1]Robson JC, Almeida C, Dawson J, Bromhead A, Dures E, Guly C, Hoon E, Mackie S, Ndosi M, Pauling J, Hill C. Patient perceptions of health-related quality of life in giant cell arteritis; international development of a disease-specific Patient-Reported Outcome Measure. Rheumatology (Oxford). 2021:keab076. http://dx.doi.org/10.1093/rheumatology/keab076 Disclosure of Interests Joanna Robson Speakers bureau: Vifor Pharma EULAR 2021 Symposium, Consultant of: Vifor Pharma Advisory board 2021, Grant/research support from: Vifor Pharma Steroid PRO grant, Celia Almeida: None declared, Jill Dawson: None declared, Emma Dures: None declared, Rosemary Greenwood: None declared, Catherine Guly: None declared, Sarah Mackie Speakers bureau: Roche/Chugai Educational talk on GCA., Consultant of: Roche/Chugai Sanofi Abbvie (2021-) AstraZeneca (2021-), Grant/research support from: Vifor Pharma Steroid PRO 2020 Vifor Pharma GTI Validation 2020 Roche GCA Tocilizumab Registry 2019 , Alison Bromhead: None declared, Steve Stern: None declared, Mwidimi Ndosi: None declared

Objectives Glucocorticoids (GCs) ('steroids') are used to treat rheumatic diseases but adverse effects are common. We aimed to explore the impact of GC therapy on health-related quality of life (HRQoL), to inform the development of a treatment-specific patient-reported outcome measure (PROM) for use in clinical trials and practice. Methods Semi-structured qualitative interviews were conducted with patients from the UK, USA and Australia, treated for a rheumatic condition with GCs in the last 2 years. Purposive sampling was used to select participants with a range of demographic and disease features. An initial conceptual framework informed interview prompts and cues. Interviews elicited GC-related physical and psychological symptoms and salient aspects of HRQoL in relation to GC therapy. Interview data were analysed inductively to develop initial individual themes and domains. Candidate questionnaire items were developed and refined. Results Sixty semi-structured qualitative interviews were conducted (UK n = 34, USA n = 10, Australia n = 16). The mean age was 58 years; 39/60 were female; and 18 rheumatic diseases were represented. Some 126 individual themes were identified and organized into six domains: physical symptoms; psychological symptoms; psychological impact of steroids; impact of steroids on participation; impact of steroids on relationships; and benefits of steroids. Candidate questionnaire items were tested and refined by piloting with patient research partners, iterative rounds of cognitive interviews and linguistic translatability assessment, informing a draft questionnaire. Conclusion We describe an international qualitative study to develop candidate items for a treatment-specific PROM for patients with rheumatic diseases. A future survey will enable the validation of a final version of the PROM.

Background Glucocorticoids (GCs) are a key treatment for inflammatory rheumatic diseases, but they cause a wide range of adverse side-effects which are of concern both to patients and clinicians. Objectives The objective of this study was to explore the impact of GC therapy on health-related quality of life (HRQoL) during treatment for rheumatic diseases, as a basis for development of a Patient-Reported Outcome Measure (PROM) to be used in clinical trials and practice. Methods Patients from the UK, USA and Australia who were treated with GCs in the last two years for a rheumatic condition were invited to take part in semi-structured qualitative interviews. Purposive sampling was used to include participants with a range of demographic and disease features. A steering committee of patient research partners, clinicians and methodologists devised an initial conceptual framework, which informed interview prompts and cues. Interviews were carried out by experienced qualitative researchers who encouraged participants to tell their stories and talk about the effects, both adverse and beneficial, of their experiences and perceptions of treatment with GCs, to identify salient physical and psychological symptoms and aspects of HRQoL. The interview data were organised using NVivo, and inductive analysis identified initial themes and domains. Candidate questionnaire items were developed and refined using cognitive interviewing, linguistic assessment, and input from patient research partners. Results Sixty semi-structured qualitative interviews were conducted (UK n=34, USA n=10, Australia n=16). Mean participant age was 58 years; 39 (66.1%) were female. Purposive sampling of participants provided a broad range of demographic features, GC dosages and inflammatory rheumatic conditions, with 27% having connective tissue disease, 25% inflammatory arthritis, 30% systemic vasculitis and 16% other rheumatic conditions. Initial domains were developed to identify key themes relating to treatment using GCs and their impact on HRQoL; see Figure 1. Figure 1. Steroid PRO Initial Themes A long-list of 134 initial candidate questionnaire items was developed from the individual themes. These items were reviewed by a qualitative working group of patient research partners, researchers and clinicians to reduce duplication and ambiguity of items. The resulting 62 items were tested and refined by piloting with patient research partners, iterative rounds of cognitive interviews with patients with a range of rheumatic conditions from the UK, USA and Australia, and a linguistic translatability assessment, to define a draft questionnaire of 40 items. Conclusion This international qualitative study underpins the development of candidate items for a treatment-specific PROM for patients with rheumatic diseases. The draft questionnaire is currently being tested in an online large-scale survey to determine the final scale structure and measurement properties using Rasch analysis, factor analysis, test-retest, comparison with EQ5D, and known groups analysis. Disclosure of Interests Susan Bridgewater Grant/research support from: Vifor Pharma, Michael A Shepherd Grant/research support from: Vifor Pharma, Jill Dawson: None declared, Pamela Richards: None declared, Christine Silverthorne: None declared, Mwidimi Ndosi: None declared, Celia Almeida: None declared, Rachel J Black: None declared, Jonathan T.L. Cheah: None declared, Emma Dures: None declared, Nilasha Ghosh: None declared, Elizabeth A Hoon: None declared, Suellen Lyne: None declared, Iris Navarro-Millan Consultant of: Honorarium on Swedish Orpham Biovitrum (SOBI) advisory board 2021, Diyu Pearce-Fisher: None declared, Carlee Ruediger: None declared, Joanna Tieu: None declared, Kevin Yip: None declared, Sarah Mackie: None declared, Susan Goodman: None declared, Catherine Hill: None declared, Joanna Robson Speakers bureau: EULAR Symposium 2021 for Vifor Pharma, Consultant of: Honorarium for Vifor Pharma advisory board 2021, Grant/research support from: Vifor Pharma 2020-2022

Abstract Background/Aims Giant cell arteritis (GCA) presents in people over 50, with headaches, visual involvement and large vessel vasculitis. A 30-item GCA-specific patient reported outcome measure (GCA PRO) was developed and tested in a clinical setting to pilot its feasibility and acceptability to patients and clinicians as a communication tool. Methods Patients seen in rheumatology or ophthalmology departments in Bristol and Leeds completed the GCA PRO prior to their consultation. Items referred to patients’ health-related quality of life over the past 7 days. Clinicians were given a copy of patients’ responses and a summary sheet highlighting overall scores which they referred to during the consultation. After the appointment, patients and clinicians completed short answer questionnaire (SAQ) plus free-text feedback forms reporting on the experience of use of the GCA PRO within the consultation. Results The GCA PRO was piloted during 16 clinic appointments. 16 patients, mean age (SD) of 74.7 (7.0), 11 (68.8%) female, 7 (43.8%) active disease and 5 (31.3%) with ocular involvement took part. Seven clinicians participated - 5 rheumatologists and 2 ophthalmologists. 87% percent of patients agreed that the GCA PRO had helped them to explain their condition; clinicians agreed that the GCA PRO had helped them to understand the patient’s condition 88% of the time (Table 1). Clinicians noted that “it was easier for the patient to convey his anxiety and feelings towards the disease and treatment” and that the GCA PRO “indirectly helped via prompting discussion of patient’s anxieties and worries”. However, they were more equivocal on its impact on decision making: “management plan was informed by symptoms, bloods and stage of illness”. Patients commented that “it helped us to plan how to manage my GCA based on my answers”, and that “the questions seemed relevant and it was helpful to me to be able to think about them before the appointment”. Conclusion The GCA-PRO was found to be an acceptable tool for use in clinic by patients and clinicians, especially in terms of explaining and understanding the patient’s condition. Disclosure C. Almeida: None. C. Guly: None. S. Mackie: Honoraria; Sept 2021: Roche/Chugai payment to my institution for delivering a talk on giant cell arteritis at an educational day. Grants/research support; MRC Confidence in Concept scheme, 2021 to develop early diagnosis methods for giant cell arteritis, 2018: MRC Proximity to Discovery: Protein biomarkers for polymyalgia rheumatica and giant cell arteritis. (PI: Morgan, University of Leeds), 2019: Roche: giant cell arteritis tocilizumab registry (PI: Morgan, University of Leeds). Other; Site Sub-Investigator on clinical trial in giant cell arteritis for Roche, Site Principal Investigator on clinical trial in giant cell arteritis for GSK, Site Principal Investigator and UK Chief Investigator on clinical trials (giant cell arteritis and polymyalgia rheumatica) for Sanofi. A. Bromhead: None. S. Stern: None. E. Dures: None. J. Dawson: None. M. Ndosi: None. R. Greenwood: None. J.C. Robson: None.

Aims The aim of this study was to develop and evaluate machine-learning-based computerized adaptive tests (CATs) for the Oxford Hip Score (OHS), Oxford Knee Score (OKS), Oxford Shoulder Score (OSS), and the Oxford Elbow Score (OES) and its subscales. Methods We developed CAT algorithms for the OHS, OKS, OSS, overall OES, and each of the OES subscales, using responses to the full-length questionnaires and a machine-learning technique called regression tree learning. The algorithms were evaluated through a series of simulation studies, in which they aimed to predict respondents’ full-length questionnaire scores from only a selection of their item responses. In each case, the total number of items used by the CAT algorithm was recorded and CAT scores were compared to full-length questionnaire scores by mean, SD, score distribution plots, Pearson’s correlation coefficient, intraclass correlation (ICC), and the Bland-Altman method. Differences between CAT scores and full-length questionnaire scores were contextualized through comparison to the instruments’ minimal clinically important difference (MCID). Results The CAT algorithms accurately estimated 12-item questionnaire scores from between four and nine items. Scores followed a very similar distribution between CAT and full-length assessments, with the mean score difference ranging from 0.03 to 0.26 out of 48 points. Pearson’s correlation coefficient and ICC were 0.98 for each 12-item scale and 0.95 or higher for the OES subscales. In over 95% of cases, a patient’s CAT score was within five points of the full-length questionnaire score for each 12-item questionnaire. Conclusion Oxford Hip Score, Oxford Knee Score, Oxford Shoulder Score, and Oxford Elbow Score (including separate subscale scores) CATs all markedly reduce the burden of items to be completed without sacrificing score accuracy. Cite this article: Bone Jt Open 2022;3(10):786–794.