Common Functional Ability Score for Young People With Juvenile Idiopathic Arthritis

From Wiley via Jisc Publications RouterHistory: received 2019-12-13, accepted 2020-03-31, pub-electronic 2021-06-04Article version: VoRPublication status: PublishedFunder: Medical Research Council; Id: http://dx.doi.org/10.13039/501100000265; Grant(s): UK Grant number: MR/K501311/1Funder: Versus Arthritis; Id: http://dx.doi.org/10.13039/501100012041; Grant(s): 20380, 20542Funder: NIHR Manchester Biomedical Research Centre; Id: http://dx.doi.org/10.13039/100014653Objective: As young people enter adulthood, the interchangeable use of child and adult outcome measures may inaccurately capture changes over time. This study aimed to use item response theory (IRT) to model a continuous score for functional ability that can be used no matter which questionnaire is completed. Methods: Adolescents (ages 11–17 years) in the UK Childhood Arthritis Prospective Study (CAPS) self‐completed an adolescent Childhood Health Assessment Questionnaire (CHAQ) and a Health Assessment Questionnaire (HAQ). Their parents answered the proxy‐completed CHAQ. Those children with at least 2 simultaneously completed questionnaires at initial presentation or 1 year were included. Psychometric properties of item responses within each questionnaire were tested using Mokken analyses to assess the applicability of IRT modeling. A previously developed IRT model from the Pharmachild‐NL registry from The Netherlands was validated in CAPS participants. Agreement and correlations between IRT‐scaled functional ability scores were tested using intraclass correlations and Wilcoxon’s signed rank tests. Results: In 303 adolescents, the median age at diagnosis was 13 years, and 61% were female. CHAQ scores consistently exceeded HAQ scores. Mokken analyses demonstrated high scalability, monotonicity, and the fact that each questionnaire yielded reliable scores. There was little difference in item response characteristics between adolescents enrolled in CAPS and Pharmachild‐NL (maximum item residual 0.08). Significant differences were no longer evident between IRT‐scaled HAQ and CHAQ scores. Conclusion: IRT modeling allows the direct comparison of function scores regardless of different questionnaires being completed by different people over time. IRT modeling facilitates the ongoing assessment of function as adolescents transfer from pediatric clinics to adult services

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ABSTRACT. Objective. To compare the psychometric functioning of multidimensional disease-specific, multiitem generic, and single-item measures of fatigue in patients with rheumatoid arthritis (RA). Methods. Confirmatory factor analysis (CFA) and longitudinal item response theory (IRT) modeling were used to evaluate the measurement structure and local reliability of the Bristol RA Fatigue Multi-Dimensional Questionnaire (BRAF-MDQ), the Medical Outcomes Study Short Form-36 (SF-36) vitality scale, and the BRAF Numerical Rating Scales (BRAF-NRS) in a sample of 588 patients with RA. Results. A 1-factor CFA model yielded a similar fit to a 5-factor model with subscale-specific dimensions, and the items from the different instruments adequately fit the IRT model, suggesting essential unidimensionality in measurement. The SF-36 vitality scale outperformed the BRAF-MDQ at lower levels of fatigue, but was less precise at moderate to higher levels of fatigue. At these levels of fatigue, the living, cognition, and emotion subscales of the BRAF-MDQ provide additional precision. The BRAF-NRS showed a limited measurement range with its highest precision centered on average levels of fatigue. Conclusion. The different instruments appear to access a common underlying domain of fatigue severity, but differ considerably in their measurement precision along the continuum. The SF-36 vitality scale can be used to measure fatigue severity in samples with relatively mild fatigue. For samples expected to have higher levels of fatigue, the multidimensional BRAF-MDQ appears to be a better choice. The BRAF-NRS are not recommended if precise assessment is required, for instance in longitudina

Fatigue assessment in RA Personal non-commercial use only

ABSTRACT. Objective. To compare the psychometric functioning of multidimensional disease-specific, multiitem generic, and single-item measures of fatigue in patients with rheumatoid arthritis (RA). Methods. Confirmatory factor analysis (CFA) and longitudinal item response theory (IRT) modeling were used to evaluate the measurement structure and local reliability of the Bristol RA Fatigue Multi-Dimensional Questionnaire (BRAF-MDQ), the Medical Outcomes Study Short Form-36 (SF-36) vitality scale, and the BRAF Numerical Rating Scales (BRAF-NRS) in a sample of 588 patients with RA. Results. A 1-factor CFA model yielded a similar fit to a 5-factor model with subscale-specific dimensions, and the items from the different instruments adequately fit the IRT model, suggesting essential unidimensionality in measurement. The SF-36 vitality scale outperformed the BRAF-MDQ at lower levels of fatigue, but was less precise at moderate to higher levels of fatigue. At these levels of fatigue, the living, cognition, and emotion subscales of the BRAF-MDQ provide additional precision. The BRAF-NRS showed a limited measurement range with its highest precision centered on average levels of fatigue. Conclusion. The different instruments appear to access a common underlying domain of fatigue severity, but differ considerably in their measurement precision along the continuum. The SF-36 vitality scale can be used to measure fatigue severity in samples with relatively mild fatigue. For samples expected to have higher levels of fatigue, the multidimensional BRAF-MDQ appears to be a better choice. The BRAF-NRS are not recommended if precise assessment is required, for instance in longitudinal settings. (J Rheumatol First Release Jan 15 2015

Cost-effectiveness of different treat-to-target strategies in rheumatoid arthritis: results from the DREAM registry

Abstract Background Adjusting medication of patients with rheumatoid arthritis (RA) until predefined disease activity targets are met, i.e. Treat-to-Target (T2T), is the currently recommended treatment approach. However, not much is known about long-term cost-effectiveness of different T2T strategies. We model the 5-year costs and effects of a step-up approach (MTX mono - > MTX + csDMARD combination - > Adalimumab - > second anti-TNF) and an initial combination therapy approach (MTX + csDMARD - > MTX + csDMARD higher dose - > anti-TNFs) from the healthcare and societal perspectives, by adapting a previously validated Markov model. Methods We constructed a Markov model in which 3-monthly transitions between DAS28-defined health states of remission (≤2.6), low (2.6  5.1) were simulated. Modelled patients proceeded to subsequent treatments in case of non-remission at each (3-month) cycle start. In case of remission for two consecutive cycles medication was tapered, until medication-free remission was achieved. Transition probabilities for individual treatment steps were estimated using data of Dutch Rheumatology Monitoring registry Remission Induction Cohort I (step-up) and II (initial combination). Expected costs, utility, and ICER after 5 years were compared between the two strategies. To account for parameter uncertainty, probabilistic sensitivity analysis was employed through Gamma, Normal, and Dirichlet distributions. All utilities, costs, and transition probabilities were replaced by fitted distributions. Results Over a 5-year timespan, initial combination therapy was less costly and more effective than step-up therapy. Initial combination therapy accrued €16,226.3 and 3.552 QALY vs €20,183.3 and 3.517 QALYs for step-up therapy. This resulted in a negative ICER, indicating that initial combination therapy was both less costly and more effective in terms of utility gained. This can be explained by higher (±5%) remission percentages in initial combination strategy at all time points. More patients in remission generates less healthcare and productivity loss costs and higher utility. Additionally, higher remission percentages caused less bDMARD use in the initial combination strategy, lowering overall costs. Conclusion Initial combination therapy was found favourable over step-up therapy in the treatment of Rheumatoid Arthritis, when considering cost-effectiveness. Initial combination therapy resulted in more utility at a lower cost over 5 years

Psychometric properties and cross-language equivalence of the revised Bristol Rheumatoid Arthritis Fatigue and the Rheumatoid Arthritis Impact of Disease scales in rheumatoid arthritis

International audienceOBJECTIVE: To assess psychometric properties and cross-language measurement equivalence of six versions of the Bristol Rheumatoid Arthritis Fatigue Scale (BRAF-MDQ) and the Rheumatoid Arthritis Impact of Disease Score (RAID in rheumatoid arthritis (RA).METHODS: Both questionnaires were completed by French (n = 206), German (n = 206), Dutch (n = 317), Spanish (n = 157), Swedish (n = 170) and UK (n = 210) RA patients. The presence of cross-language differential item functioning (DIF) was examined using the generalized partial credit model. The impact of DIF on the item and total scores was examined by comparing DIF unadjusted and DIF adjusted expected item and scale scores. IRT-based methods were used to assess psychometric properties of the instruments.RESULTS: 11 of the 20 BRAF-MDQ (55%) and 4 of the 7 RAID items (57%) exhibited significant DIF in at least one of the six countries. The mean number of items with DIF per country was 2.6 for BRAF-MDQ and 1.1 for RAID. However, the impact of DIF on the total RAID and BRAF-MDQ scores, as well as the BRAF subscales, was found to be negligible at the group level. Only for the BRAF physical subscale was there evidence of minor DIF. Marginal reliabilities of BRAF-MDQ (0.93) and RAID (0.89) were excellent, and precise scores could be obtained across the spectrum of disease impact and fatigue scores measured by these PROMs.CONCLUSION: This study supports the cross-language measurement equivalence of BRAF-MDQ and RAID and provides further support for the psychometric properties of these measures in RA

Development and validation of a patient-reported gout attack intensity score for use in gout clinical studies

OBJECTIVE Inflammation-related symptoms such as pain, swelling and tenderness of the affected joint are frequently assessed using 5-point diary rating scales in gout clinical trials. Combining these into a single gout attack symptom intensity score may be a useful summary measure for these data, which is potentially more responsive to change compared with the individual components. The objective of this study was to develop a patient-reported gout flare intensity score, the Gout Attack Intensity Score (GAIS), for use in clinical studies, that includes components for gout-related pain, swelling and tenderness. METHODS Data from a randomized controlled trial comparing anakinra to standard of care for the treatment of acute gout attacks were used for this study. A 7-day flare diary was completed by patients, including questions relating to intensity of pain, swelling and tenderness (5-point rating scales). Scalability of these items was assessed using Mokken Scale Analysis, and reliability using greatest lower bound reliability coefficients. Known-groups validity was evaluated, as well as the responsiveness to change and the presence of floor and ceiling effects. RESULTS Scalability of the single items was supported, and GAIS scores were reliable (greatest lower bound textgreater0.80). GAIS scores demonstrated responsiveness to change with high effect sizes (textgreater0.8), and discriminated better between responders and non-responders compared with its single-item components. No floor and ceiling effects were found. CONCLUSION The GAIS seems to be a reliable and responsive instrument for assessing patient-reported gout attack intensity that may be used in gout clinical studies

Long-term clinical, functional, and cost outcomes for early rheumatoid arthritis patients who did or did not achieve early remission in a real-world treat-to-target strategy

OBJECTIVE: To retrospectively compare the long-term clinical, functional, and cost outcomes for early RA patients (symptoms < 1 year) who did or did not achieve early remission in a treat-to-target strategy. METHOD: Five-year data of 471 patients included in the DREAM remission induction cohort were used. Patients were treated according to a pre-specified 28-joint Disease Activity Score (DAS28) remission driven step-up treatment strategy starting with methotrexate, addition of sulfasalazine, and exchange of sulfasalazine for biological medication in case of failure. Two- and 3-year healthcare costs were available for selected subsamples of patients only. RESULTS: DAS28 remission was achieved in 27.7%, 38.2%, and 51.6% of patients at 2, 3, and 6 months, respectively. Achieving DAS28 remission at 2, 3, or 6 months was consistently associated with significantly lower DAS28 and Health Assessment Questionnaire-Disability scores at 1, 3, and 5 years of follow-up (all P values < 0.02). Patients in remission at 2, 3, or 6 months also had significantly lower medication costs per patient over the first 2 and 3 years of treatment, mainly due to lower biologic use, but differences in total healthcare resource costs (hospital admissions plus consultations) were less pronounced. Mean total medication and total healthcare resource costs at 3 years were €1131 and €1757 for patients in remission at 6 months vs. €7533 (P < 0.01) and €2202 (P = 0.09) for those not in remission. CONCLUSION: Achieving early remission was associated with beneficial clinical outcomes for early RA patients and lower costs in the long term. Key Points • Previous studies in rheumatoid arthritis patients have demonstrated that early good response is associated with sustained remission and better long-term clinical outcomes. • This study extents these findings by examining the long-term benefits of achieving early remission on clinical, patient-reported, and economic outcomes in a real-world cohort of patients with very early rheumatoid arthritis treated according to treat-to-target principles. • The findings of this study clearly demonstrate that aiming for early remission in rheumatoid arthritis patients is beneficial in the long-term in terms of better clinical and functional outcomes and lower healthcare costs

A systematic literature review of patient-reported outcome measures used in gout: an evaluation of their content and measurement properties

Abstract Background Gout is a common, monosodium urate crystal-driven inflammatory arthritis. Besides its clinical manifestations, patients often also suffer from pain, physical impairment, emotional distress and work productivity loss, as a result of the disease. Patient-reported outcome measures (PROMs) are commonly used to assess these consequences of the disease. However, current instrument endorsements for measuring such outcomes in acute and chronic gout clinical settings are based on limited psychometric evidence. The objective of this systematic literature review was to identify currently available PROMs for gout, and to critically evaluate their content and psychometric properties, in order to evaluate the current status regarding PROMs for use in gout patients. Methods Systematic literature searches were performed in the PubMed and EMBASE databases. The methodological quality of included papers was appraised using the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist, and evaluation of measurement properties (reliability, responsiveness, construct validity, floor and ceiling effects) was done in accordance with published quality criteria. Item content was appraised by linking health concepts to the International Classification of Functioning Disability and Health (ICF) framework. Results In total, 13 PROMs were identified, of which three were targeted specifically at gout patients. The majority of the PROMs were rated positively for content validity. For most instruments, limited evidence was available for construct validity and reliability. Instruments to assess pain scored well on responsiveness and floor and ceiling effects, but not much is known about their reliability in gout. Conclusions The physical functioning subscale of the SF-36v2 (Short Form-36 item version 2) is the only PROM that had sufficient supporting evidence for all its psychometric properties. Many of the commonly used PROMs in gout are currently not yet well supported and more studies on their measurement properties are needed among both acute and chronic gout populations

Measuring Disease Exacerbation and Flares in Rheumatoid Arthritis: Comparison of Commonly Used Disease Activity Indices and Individual Measures

To evaluate and compare the utility of commonly used outcome measures for assessing disease exacerbation or flare in patients with rheumatoid arthritis (RA). Data from the Dutch Potential Optimalisation of (Expediency) and Effectiveness of Tumor necrosis factor-α blockers (POET) study, in which 462 patients discontinued their tumor necrosis factor-α inhibitor, were used. The ability of different measures to discriminate between those with and without physician-reported flare or medication escalation at the 3-month visit (T2) was evaluated by calculating effect size (ES) statistics. Responsiveness to increased disease activity was compared between measures by standardizing change scores (SCS) from baseline to the 3-month visit. Finally, the incremental validity of individual outcome measures beyond the Simplified Disease Activity Score was evaluated using logistic regression analysis. The SCS were greater for disease activity indices than for any of the individual measures. The 28-joint Disease Activity Score, Clinical Disease Activity Index, and Simplified Disease Activity Index performed similarly. Pain and physician's (PGA) and patient's global assessment (PtGA) of disease activity were the most responsive individual measures. Similar results were obtained for discriminative ability, with greatest ES for disease activity indices followed by pain, PGA, and PtGA. Pain was the only measure to demonstrate incremental validity beyond SDAI in predicting 3-month flare status. These results support the use of composite disease activity indices, patient-reported pain and disease activity, and physician-reported disease activity for measuring disease exacerbation or identifying flares of RA. Physical function, acute-phase response, and the auxiliary measures fatigue, participation, and emotional well-being performed poorl

Calibration of the PROMIS Physical Function Item Bank in Dutch Patients with Rheumatoid Arthritis

Contains fulltext : 127702.pdf (publisher's version ) (Open Access)OBJECTIVE: To calibrate the Dutch-Flemish version of the PROMIS physical function (PF) item bank in patients with rheumatoid arthritis (RA) and to evaluate cross-cultural measurement equivalence with US general population and RA data. METHODS: Data were collected from RA patients enrolled in the Dutch DREAM registry. An incomplete longitudinal anchored design was used where patients completed all 121 items of the item bank over the course of three waves of data collection. Item responses were fit to a generalized partial credit model adapted for longitudinal data and the item parameters were examined for differential item functioning (DIF) across country, age, and sex. RESULTS: In total, 690 patients participated in the study at time point 1 (T2, N = 489; T3, N = 311). The item bank could be successfully fitted to a generalized partial credit model, with the number of misfitting items falling within acceptable limits. Seven items demonstrated DIF for sex, while 5 items showed DIF for age in the Dutch RA sample. Twenty-five (20%) items were flagged for cross-cultural DIF compared to the US general population. However, the impact of observed DIF on total physical function estimates was negligible. DISCUSSION: The results of this study showed that the PROMIS PF item bank adequately fit a unidimensional IRT model which provides support for applications that require invariant estimates of physical function, such as computer adaptive testing and targeted short forms. More studies are needed to further investigate the cross-cultural applicability of the US-based PROMIS calibration and standardized metric