The Financial Burden of Guideline-recommended Cancer Medications for Metastatic Urothelial Carcinoma.

Bladder cancer is a significant global health concern owing to its prevalence, negative impact on quality of life, and high treatment costs. Treatment for metastatic urothelial carcinoma (mUC) traditionally relies on platinum-based chemotherapy regimens. However, clinical trial results have led to the approval of immune checkpoint inhibitors (ICIs) as viable treatment options. We assessed the escalating costs and economic viability of mUC treatment guidelines in Europe. We used a pragmatic approach that involved: (1) collection of the costs of the recommended medications in the five most populous European countries; (2) conversion of the costs into international dollars to account for differences in purchasing power parity among countries; (3) evaluation of the cost trends over time; and (4) comparison of the medication costs to World Health Organization thresholds. Introduction of ICIs in European guidelines substantially increased the cost of medications for mUC. Intriguingly, important differences across European countries emerged: the annual cost of medications was twofold higher in Italy than in France and the UK. Despite limitations, our study sheds light on the escalating costs and economic challenges of mUC treatment, and highlights the need for assessments of sustainable and cost-effective management approaches. PATIENT SUMMARY: We looked at the costs of treatments for metastatic bladder cancer and found that costs have been rising over time, especially with the introduction of new immune therapies, with notable differences among European countries. While these new treatments improve patient outcomes, they also come with a high price tag, which could strain health care budgets. Our results suggest that cost-effectiveness studies will be essential in determining the best and most sustainable treatment strategies in the future.

A comprehensive assessment of frailty status on surgical, functional and oncologic outcomes in patients treated with partial nephrectomy-A large, retrospective, single-center study.

BACKGROUND: Partial nephrectomy (PN) is a challenging procedure, which can be associated with severe complications. In consequence, the search for accurate and independent indicators of unfavorable surgical outcomes appears warranted. We aimed at evaluating the impact of frailty status on surgical, functional and oncologic outcomes in patients undergoing PN for renal cell carcinoma (RCC). METHODS: A retrospective, single-center study including 1,282 patients treated with PN for clinically localized cT1 RCC was performed. The modified Frailty Index (mFI) was used to assess preoperative frailty. Multivariable logistic, Poisson and linear regression analyses(MVA) tested the effect of frailty on complications, acute kidney injury(AKI), renal function decline after PN. Cumulative incidence and competing-risk analyses investigated survival outcomes. RESULTS: Of 1,282 patients, 220 (17%) were frail. Overall, 982 (76%) vs. 123 (9.6%) vs. 171 (13%) patients underwent open vs. laparoscopic vs. robot-assisted PN. Median follow-up was 66 (IQR: 35-107) months. At MVA, frailty status predicted increased risk of complications [Odds ratio (OR): 1.46, 95%CI 1.17-1.84; P < 0.001]. Moreover, frail patients were at higher risk of postoperative AKI (OR: 1.95, 95%CI 1.13-3.35; P = 0.01). In frail patients, renal function permanently decreased over time (P = 0.01) without any renal function plateau or improvement during the follow-up, which were instead observed in the nonfrail cohort. At competing-risks analyses, frailty status predicted higher risk of other-cause mortality [Hazard ratio (HR): 1.67, 95%CI 1.05-2.66; P = 0.02], but not of cancer-specific mortality (P = 0.3). CONCLUSIONS: Frailty status predicts higher risk of adverse surgical outcomes after PN. Moreover, greater renal function decline was observed in frail patients, compared with nonfrail patients. Finally, the risk of OCM significantly overcomes the risk of dying due to RCC in frail patients.

A Plea for Economically Sustainable Evidence-based Guidelines.

The rising costs of cancer care with the introduction of new agents are a challenge. The impact of these costs differs among countries. We compare costs for metastatic prostate cancer, with prices normalized to international dollars, as an example that highlights the need for cost-effectiveness analyses in trials and treatment guidelines.

The effect of frailty on post-operative outcomes and health care expenditures in patients treated with partial nephrectomy.

OBJECTIVE: To examine the effect of frailty on short-term post-operative outcomes and total hospital charges (THCs) in patients with non-metastatic renal cell carcinoma, treated with partial nephrectomy (PN). METHODS: Within the National Inpatient Sample (NIS) database we identified 25,545 patients treated with PN from 2000 to 2015. We used the Johns Hopkins Adjusted Clinical Groups (ACG) frailty-defining indicator and we examined the rates of frailty over time, as well as its effect on overall complications, major complications, blood transfusions, non-home-based discharge, length of stay (LOS) and THCs. Time trends and multivariable logistic, Poisson and linear regression models were applied. RESULTS: Overall, 3574 (14.0%) patients were frail, 2677 (10.5%) were older than 75 years and 2888 (11.3%) had Charlson comorbidity index (CCI) ≥ 2. However, the vast majority of frail patients were neither elderly nor comorbid (83%). Rates of frail patients treated with PN increased over time, from 8.3 in 2000 to 18.1% in 2015 (all p < 0.001). Frail patients showed higher rates of overall complications (43.5 vs. 30.3%), major complications (16.6 vs. 9.8%), blood transfusions (11.6 vs 8.3%) and non-home-based discharge (9.9 vs. 5.4%). longer LOS [4 (IQR: 3-6) vs. 4 (IQR: 2-5) days] and higher THCs ($43,906 vs. $38,447 - all p < 0.001). Moreover, frailty status independently predicted overall complications (OR: 1.73), major complications (OR: 1.63), longer LOS (RR: 1.07) and higher THCs (RR: +$7506). Finally, a dose-response on the risk of suboptimal surgical outcomes was shown in patients with multiple risk factors. CONCLUSIONS: One out of seven patients is frail at time of surgery and this rate is on the rise. Moreover, frailty is associated with adverse outcomes after PN. In consequence, preoperative assessment of frailty status should be implemented, to identify patients who may benefit from pre- or postoperative measures aimed at improving surgical outcomes in this patient population.

Assessment of Health-Related Quality of Life in Patients with Advanced Prostate Cancer-Current State and Future Perspectives.

With the therapeutic landscape of advanced prostate cancer rapidly evolving and oncological benefits being shown for a plethora of new agents and indications, health-related quality of life (HRQOL)-associated evidence is still subpar. In the current comprehensive review, we discuss the importance of HRQOL for patients with advanced PC (metastatic hormone-sensitive prostate cancer (mHSPC), metastatic castration-resistant prostate cancer (mCRPC) and non-metastatic castration-resistant prostate cancer (nmCRPC)), and present the most frequently used tools to evaluate HRQOL in recent randomized trials. Furthermore, we discuss the ease of use of these validated questionnaires for clinicians and try to focus on the suggested appropriate use in clinical practice, as well as potential strategies for improvement of HRQOL evaluation in these clinical scenarios of advanced prostate cancer.

Estimated Costs Associated With Radiation Therapy for Positive Surgical Margins During Radical Prostatectomy.

Importance: Prostate cancer is the most common malignant neoplasm among men and is the one with the highest positive surgical margin (PSM) rate. This high rate is due to the difficulty in balancing the risk of extraprostatic disease and excising periprostatic structures, which ultimately affects patients' quality of life. In the case of a PSM, the appropriateness of adjuvant radiation therapy (aRT) should be discussed. The financial burden of PSMs on health systems has not been investigated. Objective: To estimate the financial costs associated with a PSM during radical prostatectomy on the basis of the odds of undergoing aRT. Design, Setting, and Participants: This cohort study used data on men with prostate cancer from the US National Cancer Database (January 1, 2010, through December 31, 2015). Data were requested in March 2019, accessed in April 2019, and analyzed in August 2019. Exposure: Treatment with radical prostatectomy followed by aRT, if indicated. Main Outcomes and Measures: The attributable risk fraction of PSMs on undergoing aRT was estimated from a logistic regression with aRT administration as the outcome. The analysis was adjusted for patients' socioeconomic and demographic characteristics and tumor characteristics. The aRT cost for the year 2019 was calculated using the Medicare Physician Fee Schedule and the Hospital Outpatient Prospective Payment System. The fraction of this cost attributable to a PSM was estimated according to its attributable risk fraction. Results: In total, 230 175 men were identified (median [interquartile range] age at diagnosis, 62.0 [56.0-67.0] years). Overall, 22.8% of the patients had a PSM. Patients with PSMs were more likely than those without PSMs to be older (median [interquartile range] age, 62.0 [56.0-66.0] years vs 62.0 [57.0-67.0] years) and nonwhite (9320 patients [17.8%] vs 29 872 patients [16.8%]), to have higher comorbidity scores (1604 patients [3.1%] vs 4884 patients [2.7%] with a Charlson-Deyo Comorbidity Index score ≥2) and worse tumor characteristics (category T3 and T4 disease, 26 394 patients [50.3%] vs 36 040 patients [20.3%]), and to have lower socioeconomic indicators (median annual income <$30 000, 5708 patients [10.9%] vs 17 874 patients [10.1%]; proportion of individuals without a high school degree in the area ≥29%, 6925 patients [13.2%] vs 22 648 patients [12.7%]). In addition, PSMs were documented more frequently at nonacademic institutions than academic ones (31 702 patients [60.5%] vs 20 714 patients [39.5%]). A total of 11 585 patients (5.0%) underwent aRT, and 7698 of them (3.3%) had a PSM at the final pathology examination. When controlling for patients' socioeconomic and demographic characteristics and tumor characteristics, men with PSMs were more likely than those with negative margins to undergo aRT, with an odds ratio of 3.79 (95% CI, 3.63-3.96; P < .001). The attributable risk fraction of the presence of a PSM on aRT was 44% (95% CI, 42%-45%). The attributable cost of a PSM was calculated as $17 356 (95% CI, $16 567-$17 751). Assuming 60 000 prostatectomies in 2019 and similar trends of PSM and aRT, the overall health burden attributable to PSMs was calculated to be $52 068 000 (95% CI, $49 701 000-$53 253 000). Conclusions and Relevance: The estimated aRT cost attributable to the presence of a PSM was $17 356, resulting in $52 068 000 in spending on aRT in 2019. Strategies to reduce PSMs could be associated with a reduction in the overall health costs of surgically treated PCa.

Development and initial validation of the MS score for diagnosis of macrophage activation syndrome in systemic juvenile idiopathic arthritis.

OBJECTIVE: To develop and validate a diagnostic score that aids in identifying macrophage activation syndrome (MAS) in patients with systemic juvenile idiopathic arthritis (sJIA). METHODS: The clinical and laboratory features of 362 patients with sJIA-associated MAS and 404 patients with active sJIA without evidence of MAS were collected in a multinational collaborative project. Eighty percent of the study population was used to develop the score and the remaining 20% constituted the validation sample. A Bayesian Model Averaging approach was used to assess the role of each clinical and laboratory variables in the diagnosis of MAS and to obtain the coefficients of selected variables. The final score, named MAS/sJIA (MS) score, resulted from the linear combination of these coefficients multiplied by the values of each variable. The cut-off that best discriminated MAS from active sJIA was calculated by means of receiver operating characteristic (ROC) curve analysis. Score performance was evaluated in both developmental and validation samples. RESULTS: The MS score ranges from -8.4 to 41.8 and comprises seven variables: central nervous system dysfunction, haemorrhagic manifestations, active arthritis, platelet count, fibrinogen, lactate dehydrogenase and ferritin. A cut-off value ≥-2.1 revealed the best performance in discriminating MAS from active sJIA, with a sensitivity of 0.85, a specificity of 0.95 and a kappa value of 0.80. The good performance of the MS score was confirmed in the validation sample. CONCLUSION: The MS score is a powerful and feasible tool that may assist practitioners in making a timely diagnosis of MAS in patients with sJIA.

Phenotypic variability and disparities in treatment and outcomes of childhood arthritis throughout the world: an observational cohort study.

BACKGROUND: To our knowledge, the characteristics and burden of childhood arthritis have never been studied on a worldwide basis. We aimed to investigate, with a cross-sectional study, the prevalence of disease categories, treatment methods, and disease status in patients from across different geographical areas and from countries with diverse wealth status. METHODS: In this multinational, cross-sectional, observational cohort study, we asked international paediatric rheumatologists from specialised centres to enrol children with a diagnosis of juvenile idiopathic arthritis, according to International League of Associations for Rheumatology criteria, who were seen consecutively for a period of 6 months. Each patient underwent retrospective and cross-sectional assessments, including measures of disease activity and damage and questionnaires on the wellbeing and quality of life of the children. We qualitatively compared the collected data across eight geographical areas, and we explored an association between disease activity and damage and a country's gross domestic product (GDP) with a multiple logistic regression analysis. FINDINGS: Between April 4, 2011, and Nov 21, 2016, 9081 patients were enrolled at 130 centres in 49 countries, grouped into eight geographical areas. Systemic arthritis (125 [33·0%] of 379 patients) and enthesitis-related arthritis (113 [29·8%] of 379) were more common in southeast Asia, whereas oligoarthritis was more prevalent in southern Europe (1360 [56·7%] of 2400) and rheumatoid factor-negative polyarthritis was more frequent in North America (165 [31·5%] of 523) than in the other areas. Prevalence of uveitis was highest in northern Europe (161 [19·1%] of 845 patients) and southern Europe (450 [18·8%] of 2400) and lowest in Latin America (54 [6·4%] of 849), Africa and Middle East (71 [5·9%] of 1209), and southeast Asia (19 [5·0%] of 379). Median age at disease onset was lower in southern Europe (3·5 years, IQR 1·9-7·3) than in other regions. Biological, disease-modifying antirheumatic drugs were prescribed more frequently in northern Europe and North America than in other geographical settings. Patients living in countries with lower GDP had greater disease activity and damage than those living in wealthier countries. Damage was associated with referral delay. INTERPRETATION: Our study documents a variability in prevalence of disease phenotypes and disparities in therapeutic choices and outcomes across geographical areas and wealth status of countries. The greater disease burden in lower-resource settings highlights the need for public health efforts aimed at improving equity in access to effective treatments and care for juvenile idiopathic arthritis. FUNDING: IRCCS Istituto Giannina Gaslini.

The European network for care of children with paediatric rheumatic diseases: care across borders.

OBJECTIVES: To provide an overview of the paediatric rheumatology (PR) services in Europe, describe current delivery of care and training, set standards for care, identify unmet needs and inform future specialist service provision. METHODS: An online survey was developed and presented to national coordinating centres of the Paediatric Rheumatology International Trials Organisation (PRINTO) (country survey) and to individual PR centres (centre and disease surveys) as a part of the European Union (EU) Single Hub and Access point for paediatric Rheumatology in Europe project. The survey contained components covering the organization of PR care, composition of teams, education, health care and research facilities and assessment of needs. RESULTS: Response rates were 29/35 (83%) for country surveys and 164/288 (57%) for centre surveys. Across the EU, approximately one paediatric rheumatologist is available per million population. In all EU member states there is good access to specialist care and medications, although biologic drug availability is worse in Eastern European countries. PR education is widely available for physicians but is insufficient for allied health professionals. The ability to participate in clinical trials is generally high. Important gaps were identified, including lack of standardized clinical guidelines/recommendations and insufficient adolescent transition management planning. CONCLUSION: This study provides a comprehensive description of current specialist PR service provision across Europe and did not reveal any major differences between EU member states. Rarity, chronicity and complexity of diseases are major challenges to PR care. Future work should facilitate the development, dissemination and implementation of standards of care, treatment and service recommendations to further improve patient-centred health care across Europe.

Correction to: The Italian version of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR).

The family name of author Francesco La Torre was incorrect in the published article. The correct family name should read as La Torre F.

Cross-cultural adaptation and psychometric evaluation of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR) in 54 languages across 52 countries: review of the general methodology.

The aim of this project was to cross-culturally adapt and validate the Juvenile Arthritis Multidimensional Assessment Report (JAMAR) questionnaire in 54 languages across 52 different countries that are members of the Paediatric Rheumatology International Trials Organisation (PRINTO). This effort was part of a wider project named Epidemiology and Outcome of Children with Arthritis (EPOCA) to obtain information on the frequency of juvenile idiopathic arthritis (JIA) categories in different geographic areas, the therapeutic approaches adopted, and the disease status of children with JIA currently followed worldwide. A total of 13,843 subjects were enrolled from the 49 countries that took part both in the cross-cultural adaptation phase and in the related validation and data collection: Algeria, Argentina, Belgium, Brazil, Bulgaria, Canada, Chile, Colombia, Croatia, Czech Republic, Denmark, Ecuador, Egypt, Estonia, Finland, France, Georgia, Germany, Greece, Hungary, India, Islamic Republic of Iran, Israel, Italy, Latvia, Libya, Lithuania, Mexico, Netherlands, Norway, Oman, Paraguay, Poland, Portugal, Romania, Russian Federation, Saudi Arabia, Serbia, Slovakia, Slovenia, South Africa, Spain, Sweden, Switzerland, Thailand, Turkey, Ukraine, United Kingdom and United States of America. 9021 patients had JIA (10.7% systemic arthritis, 41.9% oligoarthritis, 23.5% RF negative polyarthritis, 4.2% RF positive polyarthritis, 3.4% psoriatic arthritis, 10.6% enthesitis-related arthritis and 5.7% undifferentiated arthritis) while 4822 were healthy children. This introductory paper describes the overall methodology; results pertaining to each country are fully described in the accompanying manuscripts. In conclusion, the JAMAR translations were found to have satisfactory psychometric properties and it is thus a reliable and valid tool for the multidimensional assessment of children with JIA.

The Italian version of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR).

The Juvenile Arthritis Multidimensional Assessment Report (JAMAR) is a new parent/patient reported outcome measure that enables a thorough assessment of the disease status in children with juvenile idiopathic arthritis (JIA). We report the results of the cross-cultural adaptation and validation of the parent and patient versions of the JAMAR in the Italian language.The reading comprehension of the questionnaire was tested in 10 JIA parents and patients. Each participating centre was asked to collect demographic, clinical data and the JAMAR in 100 consecutive JIA patients or all consecutive patients seen in a 6-month period and to administer the JAMAR to 100 healthy children and their parents.The statistical validation phase explored descriptive statistics and the psychometric issues of the JAMAR: the 3 Likert assumptions, floor/ceiling effects, internal consistency, Cronbach's alpha, interscale correlations, test-retest reliability, and construct validity (convergent and discriminant validity).A total of 1296 JIA patients (7.2% systemic, 59.5% oligoarticular, 21.4% RF negative polyarthritis, 11.9% other categories) and 100 healthy children, were enrolled in 18 centres. The JAMAR components discriminated well healthy subjects from JIA patients except for the Health Related Quality of Life (HRQoL) Psychosocial Health (PsH) subscales. All JAMAR components revealed good psychometric performances.In conclusion, the Italian version of the JAMAR is a valid tool for the assessment of children with JIA and is suitable for use both in routine clinical practice and clinical research.

Whole-body MRI in the assessment of disease activity in juvenile dermatomyositis.

OBJECTIVE: To compare whole-body MRI (WB-MRI) with clinical examination in the assessment of disease activity in juvenile dermatomyositis (JDM). METHODS: WB-MR images were obtained from 41 JDM patients and 41 controls using a 1.5 T MRI scanner and short τ inversion recovery sequences. 18 patients had follow-up WB-MRI. Muscle, subcutaneous tissue and myofascial signal abnormalities were scored in 36 muscular groups and on proximal and distal extremities. WB-MRI and clinical assessments were performed concurrently and results compared. Validation procedures included analysis of feasibility, reliability, construct validity, discriminative ability and responsiveness. RESULTS: WB-MRI revealed distal legs (26/41 patients) and forearm (19/41 patients) muscle inflammation undetected during clinical examination and allowed an accurate assessment of subcutaneous (23/41 patients) and myofascial involvement (13/41 patients). 27 patients showed a patchy distribution of muscle inflammation while in seven the abnormal hyperintense areas tended to be homogeneously distributed. The inter-reader agreement for muscular, subcutaneous and myofascial WB-MRI scores was excellent. Correlations between WB-MRI muscle score and disease activity measures were excellent (Manual Muscle Test: rs=-0.84, Childhood Myositis Assessment Scale: rs=-0.81). WB-MRI score was higher in JDM active patients when compared with the control group (pB<0.0001) and the inactive patients (pB=0.004), and showed an excellent responsiveness (standardised response mean=1.65). Follow-up WB-MRI showed resolution of inflammation in nine patients whereas clinical criteria for remission were satisfied in five. CONCLUSIONS: WB-MRI provides additional information to clinical evaluation and represents a promising tool to estimate total inflammatory burden, tailor treatment and monitor its efficacy.

Agreement among musculoskeletal pediatric specialists in the assessment of radiographic joint damage in juvenile idiopathic arthritis.

OBJECTIVE: To evaluate agreement among musculoskeletal pediatric specialists in assessing radiographic joint damage in juvenile idiopathic arthritis (JIA). METHODS: Two pediatric rheumatologists, 2 pediatric radiologists, and 2 pediatric orthopedic surgeons evaluated independently 60 radiographs of both wrists and hands of children with polyarticular-course JIA. Films were scored using an adapted and simplified version of the Larsen score, ranging from 0-5. Study radiographs were selected from 568 films used in a previous study aimed to validate an adapted pediatric version of the Sharp/van der Heijde (SHS) score. To enable comparison of specialists' scores with the adapted SHS score, the 60 radiographs were divided into 6 classes of severity of damage based on quintiles of the adapted SHS score. Agreement was evaluated in terms of absolute agreement and through weighted kappa statistics. RESULTS: The pediatric radiologists tended to assign lower scores and to provide more frequently scores of 0 than did the other specialists. Weighted kappa for the 3 pairs of specialists ranged from 0.67-0.69, indicating substantial agreement. Absolute agreement ranged from 51.3-55.7%, depending on the pair of specialists examined. Both absolute and weighted kappa concordance between specialists' scores and the adapted SHS score were poorer for the pediatric radiologist than for the other specialists. CONCLUSION: We observed fair agreement in the assessment of radiographic damage among pediatric specialists involved in the care of children with JIA. The radiologists tended to be more reserved than the rheumatologists and orthopedic surgeons in labeling radiographs as damaged or in considering changes as important.

Performance of Birmingham Vasculitis Activity Score and disease extent index in childhood vasculitides.

OBJECTIVES: To evaluate the performance of the Birmingham Vasculitis Activity Score (BVAS) v3 and the Disease Extent Index (DEI) for the assessment of disease activity in 4 primary childhood (c-) systemic vasculitides. METHODS: Patients fulfilling the EULAR/PRINTO/PRES (Ankara) c-vasculitis classification criteria for Henoch-Schönlein purpura (HSP), childhood (c) polyarteritis nodosa (c-PAN), c-Wegener's granulomatosis (c-WG) and c-Takayasu arteritis (c-TA) with disease duration at the time of diagnosis ≤3 months were extracted from the PRINTO database. The performance of the BVAS and DEI were examined by assessing convergent validity, the pattern of disease involvement, and responsiveness. We also evaluated alternative unweighted scoring methods for both tools. RESULTS: The analysis set included 796 patients with 669 HSP, 80 c-PAN, 25 c-WG and 22 c-TA. The median age at diagnosis was 6.9 years (6.6-12) and median delay in making the diagnosis from the onset of signs/symptoms was 0.01 (0.003-0.027) years. A strong correlation was found between the BVAS and DEI (rs=0.78) while correlation with the physician global assessment was moderate (rs=0.48) with BVAS and poor with DEI (rs=0.25). Both the BVAS and DEI sub-scores and total scores were able to descrive the disease involvement in the 4 childhood vasculitides. Responsiveness was large (>1.5) for both tools. The performance characteristics of the BVAS and DEI with the unweighted methods were comparable. CONCLUSIONS: This study demonstrates that both the BVAS and DEI are valid tools for the assessment of the level of disease activity in a large cohort of childhood acute and chronic vasculitides.

Development and initial validation of composite parent- and child-centered disease assessment indices for juvenile idiopathic arthritis.

OBJECTIVE: To develop and validate a parent-centered and a child-centered composite disease assessment index for juvenile idiopathic arthritis (JIA): the Juvenile Arthritis Parent Assessment Index (JAPAI) and the Juvenile Arthritis Child Assessment Index (JACAI), respectively. METHODS: The JAPAI and the JACAI include 4 measures: parent/child rating of overall well-being, pain, physical function, and health-related quality of life (HRQOL). Validation analyses were conducted on nearly 5,000 patients and included assessment of construct validity, discriminant validity, responsiveness to change, and reliability. Besides the 4-item version, a 3-item version of both indices, which did not include HRQOL, was tested. RESULTS: The JAPAI and the JACAI demonstrated good construct validity, yielding high correlations with the Juvenile Arthritis Disease Activity Score and moderate correlations with physician global rating and joint counts. Correlations obtained for the JAPAI and the JACAI and for the 4-item and the 3-item versions were comparable. Factorial analysis by principal component analysis showed that both indices are monodimensional. Both the JAPAI and JACAI discriminated well between different disease states and courses and between different levels of American College of Rheumatology Pediatric criteria in a clinical trial, and revealed fair responsiveness to clinical change. Internal consistency was satisfactory, with a Cronbach's alpha of >0.80 in all but 1 of the patient samples tested. CONCLUSION: The JAPAI and the JACAI were found to be valid instruments for assessment of disease status in JIA and suitable surrogates of physicians' evaluations. Both indices are potentially applicable in clinical practice, observational studies, and therapeutic trials.

A new approach to clinical care of juvenile idiopathic arthritis: the Juvenile Arthritis Multidimensional Assessment Report.

OBJECTIVE: To develop and test a new multidimensional questionnaire for assessment of children with juvenile idiopathic arthritis (JIA) in standard clinical care. METHODS: The Juvenile Arthritis Multidimensional Assessment Report (JAMAR) includes 15 parent or patient-centered measures or items that assess well-being, pain, functional status, health-related quality of life, morning stiffness, disease activity, disease status and course, joint disease, extraarticular symptoms, side effects of medications, therapeutic compliance, and satisfaction with illness outcome. The JAMAR is proposed for use as both a proxy-report and a patient self-report, with the suggested age range of 7-18 years for use as a self-report. From March 2007 to September 2009, the questionnaire was completed by the parents of 618 children with JIA in 1814 visits and by 332 children in 749 visits. RESULTS: The JAMAR was found to be feasible and to possess face and content validity. All parents and children reported that the questionnaire was simple and easy to understand. Completion and scoring appeared to be quick, requiring < 15 minutes. There were very few missing data. Parents' proxy-reported and children's self-reported data were remarkably concordant. The JAMAR provided thorough information for the study patients about recent medical history and current health status. It performed similarly across different children's ages and characterized the level of disease activity and disability well. CONCLUSION: The development of the JAMAR introduces a new approach in pediatric rheumatology practice. This new questionnaire may help enhance the quality of care of children with JIA.