Global Assessment Of Disease Activity Research Articles

Addition of constitutional symptoms to the SLEDAI-2K improves overall disease activity assessment: A pilot study.

Constitutional symptoms (fatigue, lymphadenopathy, and weight loss) are not included in the SLE disease activity index-2000 (SLEDAI-2K). In this pilot study, we assessed the concurrent and construct validity of a revised SLEDAI-2K (SLED-R) that included these symptoms with the original SLEDAI-2K (SLED-O), using the physician global assessment of disease activity (PGA) as the reference. Our revised SLED-R substituted the SLED-O's fever descriptor with a constitutional descriptor that included fever, fatigue, lymphadenopathy, and/or weight loss. SLED-O, SLED-R, PGA and patient global assessment (PtGA) scores were collected prospectively. Bland-Altman correlations for repeated measures were calculated and Meng's z-test was used to compare correlations between dependent and overlapping correlation coefficients. Associations between constitutional symptoms and disease activity measures were analyzed using Mann-Whitney U, Kruskal-Wallis, Chi-square tests and repeated measures correlations. 1123 SLED-O, SLED-R, PGA, and 1066 PtGA were collected in 239 subjects. The new descriptor was scored in 45 subjects (18.8%) and 92 instances (8.1%), while the original descriptor, fever, was scored in only 4 subjects (1.7%) and 5 instances (0.4%). Mean SLED-O, PGA and PtGA scores were higher when the constitutional descriptor was scored versus not (p < .001). The correlation between SLED-R and PGA was marginally higher than between SLED-O and PGA (p < .001). Fatigue contributed most to this increase (p = .001) and associated with both higher PGA and PtGA scores (p < .001). Mean SLED-O and PGA scores were higher when ≥1 constitutional symptom(s) were scored versus not (p < .002). Correlations between PGA and PtGA when the new descriptor was scored versus not were similar (p = .860). The frequency of concordance between PGA and PtGA was lower when the new descriptor was scored (55%) versus not (72.5%), with PGA > PtGA when the new descriptor was scored (p < .001). The addition of constitutional symptoms to SLEDAI-2K, particularly fatigue, resulted in a marginal increase in its correlation with PGA, and new constitutional symptoms associated with higher SLED-O and PGA scores. As fatigue is subjective and difficult to attribute to SLE, its validity and inter-rater reliability in scoring remains uncertain. The clinical utility of SLED-R remains unclear, and further studies of its validity and reliability are needed.

BASDAI versus ASDAS in evaluating axial involvement in patients with psoriatic arthritis: a pooled analysis of two phase 3 studies.

In the absence of axial psoriatic arthritis (axPsA)-specific tools, the BASDAI and Ankylosing Spondylitis Disease Activity Score (ASDAS) are used to assess axial symptoms in patients with PsA. Here, we assessed the performance of BASDAI and ASDAS in patients with PsA. Patients with active PsA in DISCOVER-1 and DISCOVER-2 (ClinicalTrials.gov: NCT03162796 and NCT03158285, respectively) with or without axPsA but with available baseline BASDAI information were analysed; those with investigator-identified axial symptoms and imaging-confirmed sacroiliitis comprised the axPsA cohort. Correlations between BASDAI/ASDAS and clinical variables were assessed with Pearson's coefficient (r). Longitudinal effects of enthesitis (Leeds Enthesitis Index [LEI]), swollen joint count and presence versus absence of axPsA on BASDAI/ASDAS (normalized 0-10 scale) were analysed with mixed models for repeated measures. At baseline in the axPsA (n = 312) and non-axPsA (n = 124) cohorts, BASDAI scores showed no or weak correlation with swollen joint count (0.18-0.20), tender joint count (0.12-0.29), LEI (-0.04 to 0.24) and physician global assessment (0.35-0.43); moderate correlation with fatigue (both -0.56); and strong correlation with patient global assessment of disease activity (0.62-0.69) and patient-reported pain (0.66-0.70). Similar correlations were observed for ASDAS. Axial involvement versus non-involvement was associated with higher BASDAI scores and ASDAS (all β ≥ 0.5), without differences between instruments; longitudinal associations between swollen joint count (β ≤ 0.06)/LEI (β ≤ 0.19) and BASDAI/ASDAS were clinically unimportant. BASDAI and ASDAS performed similarly in patients with active PsA and axial involvement, independent of peripheral disease involvement, supporting their performance in assessing axial disease activity. ClinicalTrials.gov, http://clinicaltrials.gov, NCT03162796 and NCT03158285.

Drug survival and change of disease activity using a second janus kinase inhibitor in patients with difficult-to-treat rheumatoid arthritis who failed to a janus kinase inhibitor and subsequent biologics.

To assess the drug survival and change of disease activity using a second Janus kinase inhibitor (JAKi) after failure to a JAKi and subsequent biologic disease-modifying anti-rheumatic drugs (bDMARDs) in patients with difficult-to-treat rheumatoid arthritis (RA). This retrospective cohort study included 32 patients with difficult-to-treat RA who failed to a JAKi and subsequently to one or more bDMARDs and then switched to a second JAKi. To assess drug survival, electronic medical records of each patient were reviewed. Data on whether the second JAKi was discontinued, and the reasons for discontinuation were collected. The change of disease activity was assessed by analyzing changes in tender joint count (TJC), swollen joint count (SJC), patient's global assessment of disease activity on a visual-analogue scale (VAS), erythrocyte sedimentation rate (ESR), C-reactive protein (CRP), Disease Activity Score for 28 joints with ESR (DAS28-ESR), and DAS28-CRP from baseline to that at six months from initiation of the second JAKi. Overall, discontinuation of the second JAKi occurred in 20 (62.5%) patients. Primary failure, secondary failure, adverse events, and insurance coverage issues were the reasons for discontinuation in 9 (45.0%), 5 (25.0%), 2 (10.0%), and 4 (20.0%) patients, respectively. The estimated 2-year drug survival rate was 39.3%. In terms of change of disease activity, the second JAKi significantly improved TJC (p < 0.001), SJC (p < 0.001), VAS (p < 0.001), CRP (p = 0.026), DAS28-ESR (p < 0.001), and DAS28-CRP (p < 0.001) at 6-month compared with that at the baseline. Second JAKi could be a therapeutic option in patients with difficult-to-treat RA who have failed to a JAKi and subsequent bDMARDs.

Assessment of Patient-Physician Interactions in Psoriatic Arthritis: National Results of the ASSIST Study.

An overarching principle for the management of psoriatic arthritis (PsA) is a shared decision-making process between physicians and patients. The aim of this study is to assess the patient-physician relationship in a group of patients with PsA, by using the Perceived Efficacy in Patient-Physician Interactions (PEPPI) and CollaboRATE instruments. This is a cross-sectional multicenter study where consecutive patients with PsA were enrolled. For each patient, the main demographic, comorbid conditions, and clinical data were collected, including the assessment of disease activity, function, quality of life, and impact of disease. PEPPI and CollaboRATE questionnaires were used, respectively, to evaluate the patient's perception of the patient-physician relationship and the shared decision-making process. A total of 81 patients with PsA were enrolled at four centers in Italy. Overall, our patients showed a high level of confidence in obtaining needed health care, with relatively high median (IQR) values of PEPPI (20; 16-23), and a good shared decision-making process, with high median (IQR) values of CollaboRATE questionnaire (7; 6-9). PEPPI and CollaboRATE scores showed a statistically significant inverse correlation with different clinical variables such as disease duration, Leeds Enthesitis Index, PsA impact of Disease, Health Assessment Questionnaire, pain, patient's global assessment of disease activity and clinical disease activity for PsA. The presence of comorbidities did not appear to be associated with lower values of PEPPI and CollaboRATE. In this study, few patients with PsA were at risk of suboptimal communication with their physician. This phenomenon appeared to be primarily related to higher disease activity and burden.

Disease activity trajectories from childhood to adulthood in the population-based Nordic juvenile idiopathic arthritis cohort

ObjectivesTo identify long-term disease activity trajectories from childhood to adulthood by using the clinical Juvenile Arthritis Disease Activity Score (cJADAS10) in juvenile idiopathic arthritis (JIA). Second, to evaluate the contribution...

Meaningful score changes for SF-36v2, FACIT-fatigue, and RASIQ in rheumatoid arthritis

BackgroundInterpretation thresholds for patient-reported outcome (PRO) scores are of crucial importance, particularly when interpreting treatment benefit. This study was designed to determine the within-patient meaningful improvement (WPMI) thresholds for the Short-Form 36 Health Survey version 2 (SF-36v2), the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue), and the novel Rheumatoid Arthritis Symptoms and Impact Questionnaire (RASIQ) among patients with rheumatoid arthritis (RA).MethodsIn this post-hoc analysis, anchor-based and supportive distribution-based methods were used to derive WPMI based on blinded data from all treatment arms in two Phase 2 RA trials with otilimab. Patient’s Global Assessment of Disease Activity (PtGA) was the general anchor for all SF-36v2 scales. SF-36 Patient’s Global Impression of Status (PGIS), PtGA, and VT03 (an SF-36v2 item) were used as anchors for FACIT-Fatigue. SF-36 PGIS, PtGA, and Patient’s Assessment of Arthritis Pain (PAIN) were anchors for RASIQ. Mean change was calculated for the anchor category associated with minimal meaningful improvement from baseline to Week 24 for SF-36v2 and FACIT-Fatigue, and to Week 12 for RASIQ. Sensitivity and specificity were used to evaluate the accuracy of estimated WPMI values.ResultsFor the SF-36v2 physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health domains, anchor-based estimates of WPMI based on 0–100 scores were 24.5, 24.5, 25.4, 13.6, 21.5, 20.5, 16.9, and 14.3, respectively. Anchor-based WPMI estimates were 9.7 for the Physical Component Summary score and 7.6 for the Mental Component Summary score (using norm-based T-score metric). For FACIT-Fatigue (range 0–52), WPMI estimates ranged from 9.7 to 11.3 points. For RASIQ (range 0–100), anchor-based WPMI was determined as a change between -32.7 and -21.7 points for the Joint Pain scale, -26.7 to -23.7 for the Joint Stiffness scale, and -21.1 to -17.4 for the Impact scale.ConclusionsThis study derived WPMI thresholds for SF-36v2, FACIT-Fatigue, and RASIQ among patients with RA, using multiple anchors. Derivation of WPMI thresholds for these PRO instruments will enable their broader use in evaluating and interpreting treatment benefit in future RA studies.

Profiling of plasma extracellular vesicles identifies proteins that strongly associate with patient's global assessment of disease activity in rheumatoid arthritis.

Rheumatoid arthritis (RA) is an autoimmune disease characterized by chronic synovial inflammation and cartilage/bone damage. Intercellular messengers such as IL-1 and TNF play a crucial role in the pathophysiology of RA but have limited diagnostic and prognostic values. Therefore, we assessed whether the protein content of the recently discovered extracellular vesicles (EVs), which have gained attention in the pathogenesis of RA, correlates with disease activity parameters in RA patients. We identified and quantified proteins in plasma-derived EVs (pEVs), isolated by size exclusion chromatography from 17 RA patients by mass spectrophotometry (MS). Quantified protein levels were correlated with laboratory and clinical parameters and the patient's own global assessment of their disease activity (PGA-VAS). In a second MS run, the pEV proteins of nine other RA patients were quantified and compared to those from nine healthy controls (HC). No differences were observed in the concentration, size, and protein content of pEVs from RA patients. Proteomics revealed >95% overlapping proteins in RA-pEVs, compared to HC-pEVs (data are available via ProteomeXchange with identifier PXD046058). Remarkably, in both runs, the level of far more RA-pEV proteins correlated positively to PGA-VAS than to either clinical or laboratory parameters. Interestingly, all observed PGA-VAS positively correlated RA-pEV proteins were associated with the actin-cytoskeleton linker proteins, ezrin, and moesin. Our observation suggests that PGA-VAS (loss of vitality) may have a different underlying pathological mechanism in RA, possibly related to enhanced muscle actin-cytoskeleton activity. Furthermore, our study contributes to the growing awareness and evidence that pEVs contain valuable biomarkers for diseases, with added value for RA patients.

Relationships of Work Productivity and Activity Impairment With Patient-Reported Outcomes in Ankylosing Spondylitis: Results From Two Trials.

We examined the relationships of work productivity and activity impairment with key patient-reported outcomes (PROs) assessing pain, disease activity, and health-related quality of life (HRQoL) in patients with ankylosing spondylitis (AS). This post hoc analysis pooled available data from baseline to end of the double-blind phase of phase 2 and 3 placebo-controlled tofacitinib trials in patients with active AS. A repeated-measures longitudinal model assessed the relationships (linear or nonlinear) between Work Productivity and Activity Impairment questionnaire in Spondyloarthritis (WPAI:SpA) domains (absenteeism, activity impairment, presenteeism, and productivity loss) as outcomes and key PROs (total back pain, nocturnal spinal pain, Patient Global Assessment of Disease Activity, AS Quality of Life, EuroQol 5-Dimension 3-Level [EQ-5D-3L], and EQ-5D Visual Analog Scale [EQ-5D-VAS]) as predictors. Data from 330 to 475 patients were available, depending on the analysis. Relationships between WPAI:SpA domains and PROs were approximately linear. The worst PRO scores were associated with a decline in patients' work capacity (measured by activity impairment, presenteeism, and productivity loss [>65%]); the best scores were associated with improvements in WPAI:SpA domains (8%-23%). Incremental PRO improvements were associated with improvement of activity impairment, presenteeism, and productivity loss. Relationships between absenteeism and PROs were the weakest, owing to absenteeism being low in the study population. Evidence of linear relationships between work productivity and activity impairment with patient-reported pain, disease activity, and HRQoL was observed. Interventions to control pain and disease activity and improve HRQoL are therefore likely to improve work productivity and reduce activity impairment in patients with AS.

MRI ankle in juvenile idiopathic arthritis and its comparison with clinical disease activity scores

Objectives: Juvenile idiopathic arthritis (JIA) is a common cause of morbidity and consequent disability among youth up to 16 years of age. Our study aims to image the ankle joints of JIA patients and correlate the findings with the clinical disease activity scores. Juvenile Arthritis Disease Activity Score (JADAS-27) is used by pediatricians to assess disease activity in JIA patients. There are four factors that determine this score – Global assessment of disease activity by the physician as well as patient/parent, joint count with active disease, and erythrocyte sedimentation rate (ESR). However, these clinical scores are dependent on the clinician’s experience and observation and, therefore, subjective. Material and Methods: Thirty patients of either sex with a clinical diagnosis of JIA as per the International League of Association for Rheumatology definition with clinically involved ankle joints were included in the study. Chronic arthritis due to other causes, such as infection, trauma, and so on, was excluded. All clinically diagnosed cases of JIA were subjected to magnetic resonance imaging (MRI) evaluation on Siemens Skyra 3 Tesla High-Resolution MR system using a dedicated coil for the ankle joint. Six sub-joints for each patient are evaluated, namely - The tibiotalar joint, subtalar joint, calcaneocuboid joint, calcaneonavicular joint, naviculocuneiform joint, and tarsometatarsal joint. Image analysis and scoring shall be done for each patient at each joint as per the Juvenile Arthritis MRI SCORE, consisting of the following parameters-synovial hypertrophy score, cartilage lesion score, bone erosion score, and bone marrow change score. Each of these scores is calculated individually based on the percentage of involved bone volume. All individual scores were summed to get the total MRI Score. The data collected were then statistically analyzed. Results: A positive correlation was sought between the JADAS-27 score and the total MRI score with the P-value being 0.03. A positive correlation was also found between the total MRI score and the ESR value of the patient (P - 0.02). Conclusion: MRI findings can be a potential marker of disease activity in JIA patients and these findings could also predict the prognosis accurately in these patients. The findings are objective and comparable to the aforementioned clinical scores.

How Russian patients with rheumatoid arthritis assess their condition: initial data from the OPTIMA (Patient Assessment of Severity, Outcomes and Medical Care in Arthritis) pilot study

Dynamic monitoring of the patient's condition is a fundamental element of the modern strategy for the treatment of rheumatoid arthritis (RA), which aims to achieve remission or low inflammatory activity of the disease. A simple and informative indicator that can be used to assess treatment outcomes from a patient's perspective is the Patient Acceptable State Status (PASS).Objective: to determine the severity of patient-reported outcomes (PROs) and their relationship to the PASS indicator in patients with RA in reallife clinical practice.Material and methods. A one-time survey of 945 patients with RA was conducted in paper and electronic form from January to June 2023. The survey included the determination of PROs (pain, fatigue, anxiety, depression, activities of daily living, global assessment of health – GAH, – global assessment of disease activity – GADA) and PASS. The majority of patients were women – 87.8%, mean age – 46.3±13.2 years, median disease duration – 6 [3; 14] years. 80.4% of patients received synthetic disease modifying antirheumatic drugs, 23.9% – biologic disease modifying antirheumatic drugs (bDMARDs) and Janus kinase inhibitors (JAKi), 36.5% – glucocorticoids (GC), 82.8% – nonsteroidal anti-inflammatory drugs (NSAIDs).Results and discussion. According to the survey, the severity of joint pain (according to a numerical rating scale, NRS 0–10) was on average 5.3±2.6, fatigue – 6.2±2.6, GAH – 5.4±2.4, GADA – 5.4±2.7, median anxiety – 5 [3; 8], depression – 5 [2; 7], limitation of daily activity – 5 [3; 7]. Satisfaction with their health condition (PASS+) reported 54.8% of patients, dissatisfaction (PASS-) was associated with the presence of moderate/severe pain (NRS ≥4; odds ratio, OR 2.665; 95% confidence interval, CI 2.072–3.429; p&lt;0.001), fatigue (OR 2.497; 95% CI 1.818–3.430; p&gt;&lt;0.001), anxiety (OR 1.683; 95% CI 1.395–2.029; p&gt;&lt;0.001) and depression (OR 1.537; 95% CI 1.308–1.805; p&gt;&lt;0.001). PASS- was statistically significant more common in patients taking NSAIDs and GCs and significantly less common in patients receiving bDMARDs and JAKi. Conclusion. PASS is associated with the main PROs (pain, fatigue, anxiety and depression) and can be used as one of the indicators for evaluating the effectiveness of RA treatment. Keywords: rheumatoid arthritis; Patient Acceptable State Status; patient-reported outcomes; pain; fatigue; depression; anxiety&gt; ˂ 0.001), fatigue (OR 2.497; 95% CI 1.818–3.430; p&lt;0.001), anxiety (OR 1.683; 95% CI 1.395–2.029; p&gt;&lt;0.001) and depression (OR 1.537; 95% CI 1.308–1.805; p&gt;&lt;0.001). PASS- was statistically significant more common in patients taking NSAIDs and GCs and significantly less common in patients receiving bDMARDs and JAKi. Conclusion. PASS is associated with the main PROs (pain, fatigue, anxiety and depression) and can be used as one of the indicators for evaluating the effectiveness of RA treatment. Keywords: rheumatoid arthritis; Patient Acceptable State Status; patient-reported outcomes; pain; fatigue; depression; anxiety&gt; ˂ 0.001), anxiety (OR 1.683; 95% CI 1.395–2.029; p&lt;0.001) and depression (OR 1.537; 95% CI 1.308–1.805; p&gt;&lt;0.001). PASS- was statistically significant more common in patients taking NSAIDs and GCs and significantly less common in patients receiving bDMARDs and JAKi. Conclusion. PASS is associated with the main PROs (pain, fatigue, anxiety and depression) and can be used as one of the indicators for evaluating the effectiveness of RA treatment. Keywords: rheumatoid arthritis; Patient Acceptable State Status; patient-reported outcomes; pain; fatigue; depression; anxiety&gt; ˂ 0.001). PASS- was statistically significant more common in patients taking NSAIDs and GCs and significantly less common in patients receiving bDMARDs and JAKi.Conclusion. PASS is associated with the main PROs (pain, fatigue, anxiety and depression) and can be used as one of the indicators for evaluating the effectiveness of RA treatment.

Organ damage is a major determinant of work productivity impairment in Behçet’s syndrome: a post hoc analysis of the BODI validation study

Abstract Objectives To evaluate the prevalence, magnitude and potential determinants of work productivity impairment in patients with Behçet’s syndrome (BS), focusing on the role of irreversible organ damage. Methods A post hoc analysis of the BS Overall Damage Index (BODI) prospective validation study was performed. Demographics and clinical features were recorded in all patients. The Work Productivity and Activity Impairment: General Health (WPAI:GH) questionnaire was administered to assess the work limitation and the BODI to measure organ damage. The independent effect of BS features on WPAI:GH outcomes was evaluated by regression analysis. Results Of 148 patients, 34.5% were unemployed, with age [odds ratio (OR) 1.035] and BODI score (OR 1.313 for a 1-unit increase) as the only factors significantly (P &amp;lt; 0.05) associated with the unemployment state. Overall work impairment was reported in ≈64.2% of the employed patients. Indeed, 22.7% reported missing work hours due to their health (absenteeism), with a mean time loss of 34.4%, whereas 60.2% declared reduced performance at work because of their health (presenteeism), with a mean productivity impairment of 45.4%. Ocular damage was associated with absenteeism (β = 0.225); female sex (β = 0.260), physician global assessment of disease activity (β = 0.502) and an increased BODI score (β = 0.166 for 1-point increase) with presenteeism; and fibromyalgia (β = 0.246), Physician Global Assessment (β = 0.469) and musculoskeletal damage (β = 0.325) with overall work impairment. Conclusions Disease activity and organ damage accrual remarkably affect work productivity in BS patients. Achieving remission and preventing damage accrual are crucial and complementary objectives.

The contributions of clinical disease activity, functional disability, and illness intrusiveness to depressive symptoms in pediatric inflammatory bowel disease.

Clinical disease activity associated with inflammatory bowel disease (IBD)can place physical limitations on youths' activities of daily living. In turn, functional limitations potentially contribute to youths' heightened experience of IBD-induced intrusions on a wide range of routine and valued activities (i.e., illness intrusiveness), which can increase their risk for depressive symptoms. The present study examined the contributions of clinical disease activity, functional disability, and illness intrusiveness to depressive symptoms in youth with IBD. Youth (N = 180) completed the Functional Disability Inventory (FDI),Illness Intrusiveness Scale-Child (IIS-C),and Children's Depression Inventory-2 (CDI-2).Physicians completed the Physicians Global Assessment of disease activity (PGA). Results revealed a mediating effect for functional disability in the association between disease activity and depressive symptoms (PGA → FDI → CDI-2);illness intrusiveness mediated the association between functional disability and depressive symptoms (i.e., FDI → IIS-C → CDI-2).Serial mediation revealed that clinical disease activity conferred an indirect effect on youth depressive symptoms through the sequential effects of functional disability and illness intrusiveness (i.e., PGA → FDI → IIS-C → CDI-2). Taken together, these findings indicate that youth who encounter more physical limitations as a function of clinical disease activity are more likely to experience an amplified sense of IBD-related intrusions on their ability to participate in meaningful activities. In turn, heightened illness intrusiveness increases the likelihood of depressive symptoms. Clinical interventions that help youth maintain adequate functional ability in the face of IBD disease activity and encourage involvement in positively valued activities could decrease the negative impact of IBD on youths' emotional adjustment.

The role of upadacitinib for the treatment of axial spondyloarthritis.

Janus kinase inhibitors were recently approved for treatment of axial spondyloarthritis following clinical trials demonstrating benefit for symptom control. Upadacitinib treatment resulted in Assessment of SpondyloArthritis International Society 40 response improvement (defined as at least 40% improvement and an absolute improvement in global assessment of disease activity, patient assessment of back pain and other indices) in 45-52% of trial participants with axial spondyloarthritis. We review the data for efficacy and safety of upadacitinib in this patient population.

Protocol for a multicentre randomised controlled trial examining the effects of temporarily pausing Bruton tyrosine kinase inhibitor therapy to coincide with SARS-CoV-2 vaccination and its impact on immune responses in patients with chronic lymphocytic leukaemia

IntroductionPeople who are immunocompromised have a poor biological response to vaccinations. This study aims to determine in patients with chronic lymphocytic leukaemia (CLL) if a 3-week pause in Bruton tyrosine...

Plasma pentraxin 3 in idiopathic inflammatory myopathies: a possible new biomarker of disease activity.

Pentraxin-3 (PTX3) is a component of humoral innate immunity with essential functions both in promotion and resolution of inflammation. We aimed to study the PTX3 in the plasma and in the muscle of patients with idiopathic inflammatory myopathies (IIM) and whether PTX3 may correlate with disease activity. Plasma PTX3 levels were assessed in 20 patients with IIMs, 10 dermatomyositis (DM), and 10 polymyositis (PM), compared to 10 patients with rheumatoid arthritis (RA) and 10 healthy donors (HDs) aged, sex, and body mass index matched. Disease activity in IIMs was assessed by Myositis Disease Activity Assessment Visual Analog Scale (MYOACT), while disease activity score on 28 joints (DAS28) was used for RA patients. Muscle histopathology and immunohistochemical (IHC) analyses were also performed. Mean plasma PTX3 levels were significantly higher in IIM patients than HDs (518 ± 260 pg/ml vs. 275 ± 114 pg/ml, P = 0.009). Linear regression analysis adjusted for age, sex, and disease duration showed a direct correlation between PTX3 and CPK levels (β: 0.590), MYOACT (β: 0.759), and physician global assessment of disease activity (β: 0.832) in IIMs. No association between PTX3 levels and DAS28 was found in RA. Global PTX3 pixel fraction was higher in IIM than HDs muscle, but a lower PTX3 expression was found in perifascicular areas of DM and in myofibers with sarcolemmal staining for membrane attack complement. PTX3 plasma levels were increased in IIMs and correlated with disease activity suggesting a possible role as biomarker of disease activity. PTX3 showed a different distribution in DM or PM muscle.

Clinical features, treatment, and outcomes of celiac-associated arthritis: a retrospective cohort study

BackgroundAlthough arthritis is recognized as an extra-intestinal manifestation of celiac disease, little is known about the clinical course and outcomes of pediatric celiac-associated arthritis. This study describes clinical characteristics, treatments, and outcomes of children with celiac-associated arthritis.MethodsThis was a retrospective cohort study of children with celiac disease seen in pediatric rheumatology clinic between 2004 and 2021 for joint complaints. Data was abstracted from electronic health records. Patient demographics and clinical manifestations were evaluated using standard descriptive statistics. Physician- and patient-reported outcomes were evaluated at the index visit, 6-month follow-up, and last recorded visit, and were compared using Wilcoxon signed-rank tests.ResultsTwenty-nine patients with celiac disease were evaluated for joint complaints, and 13 were diagnosed with arthritis. Their mean age was 8.9 years (SD 5.9), and 61.5% were female. Celiac disease diagnosis was made before arthritis diagnosis in only 2 cases (15.4%). Initial testing that led to the celiac disease diagnosis was obtained by the rheumatologist in 6 cases (46.2%). Only 8 patients (61.5%) had concomitant GI symptoms, and of these, 3 patients had BMI z-scores <-1.64 and 1 had impaired linear growth. Arthritis presentation was most often oligoarticular (76.9%) and asymmetric (84.6%). Most cases required systemic therapy (n = 11, 84.6%) with DMARDs, biologics, or both. Of the 10 patients who required systemic therapy and reported compliance with the gluten-free diet, 3 (30%) were able to stop systemic medications. Two of 3 patients who cleared celiac serologies came off systemic medications. Statistically significant improvement was noted in the number of joints involved (p = 0.02) and physician global assessment of disease activity (p = 0.03) between the index and final visit.ConclusionsRheumatologists play an important role in the identification of celiac disease, as arthritis was the presenting symptom in most cases and was not always associated with GI symptoms or poor growth. The arthritis was most often oligoarticular and asymmetric. Most children required systemic therapy. The gluten-free diet may not be sufficient to manage arthritis, but antibody clearance may be an indicator of higher likelihood of disease control off medications. Outcomes are promising with a combination of diet and medical therapy.

Safety of Measles-Mumps-Rubella booster vaccination in patients with juvenile idiopathic arthritis: A long-term follow-up study.

To study short and long-term disease activity and vaccine-related adverse events in a cohort of JIA patients who received the live attenuated measles-mumps-rubella (MMR) booster vaccine while being treated with immunosuppressive and immunomodulatory therapies. A retrospective study was performed in the UMC Utrecht, clinical and therapeutic data were collected from electronic medical records for two visits before and two visits after the MMR booster vaccine of JIA patients. Drug therapy was collected and adverse events related to the vaccine were requested from the patients during clinical visits or by short phone interviews. Associations between MMR booster vaccination and the active joint count, physician global assessment of disease activity, patient-reported visual analogue scale (VAS) for well-being and clinical Juvenile Arthritis Disease Activity Score (cJADAS) were analyzed using multivariable linear mixed effects analyses. A total of 186 JIA patients were included in the study. At the time of vaccination, 51% of the patients used csDMARD and 28% used bDMARD therapy. Overall, adjusted disease activity scores after MMR booster vaccination were not significantly different compared to pre-vaccination. Mild adverse events related to the MMR booster were reported for 7% of the patients. No serious adverse events were reported. MMR booster vaccination was safe and did not worsen disease activity during long-term follow-up in a large cohort of JIA patients being treated with both csDMARDs and biological DMARDs.

Efficacy and safety of baricitinib in Japanese patients with autoinflammatory type I interferonopathies (NNS/CANDLE, SAVI, And AGS)

BackgroundThis study evaluated the efficacy and safety of baricitinib (Janus kinase-1/2 inhibitor), in adult and pediatric Japanese patients with Nakajo-Nishimura syndrome/chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (NNS/CANDLE), stimulator of interferon genes-associated vasculopathy with onset during infancy (SAVI), or Aicardi-Goutières syndrome (AGS).MethodsA Phase 2/3, multicenter, open-label study (NCT04517253) was conducted across 52 weeks. Primary efficacy endpoint assessed the change in mean daily diary score (DDS) from baseline to the end of primary treatment period. Other efficacy endpoints included change in mean DDS to the end of maintenance period, daily corticosteroid use, Physician’s Global Assessment of Disease Activity (PGA) scores, and daily symptom-specific score (DSSS) from baseline to primary and maintenance treatment periods. All treatment-emergent adverse events (TEAEs) that occurred postdosing were recorded.ResultsOverall, 9 patients (5 with NNS, 3 with SAVI, and 1 with AGS) were enrolled; 55.6% were females, mean age was 26 years, and mean corticosteroid use/weight was 0.2 mg/kg. At the end of primary treatment period, mean DDS decreased from baseline in patients with NNS/CANDLE (0.22) and SAVI (0.21) and increased in the patient with AGS (0.07). At the end of maintenance treatment period, mean DDS decreased from baseline in patients with NNS/CANDLE (0.18) and SAVI (0.27) and increased in the patient with AGS (0.04). Mean percent corticosteroid use decreased by 18.4% in 3 out of 5 patients with NNS/CANDLE and 62.9% in 1 out of 3 patients with SAVI. Mean PGA score decreased from baseline in patients with NNS/CANDLE (1.60), SAVI (1.33), and AGS (1.0), and mean DSSS improved from baseline. All patients reported ≥ 1 TEAE. Frequently reported AEs included BK polyomavirus detection (3; 33.3%), increased blood creatine phosphokinase (2; 22.2%), anemia (2; 22.2%), and upper respiratory tract infection (2; 22.2%). Three (33.3%) patients reported serious adverse events, 1 of which was related to study drug. One patient with SAVI died due to intracranial hemorrhage, which was not related to study drug.ConclusionBaricitinib may offer a potential therapeutic option for patients with NNS/CANDLE, SAVI, and AGS, with a positive benefit/risk profile in a vulnerable patient population with multiple comorbidities.Trial registrationNLM clinicaltrials.gov, NCT04517253. Registered 18 August 2020.

Evaluating key performance indicators of the process of care in juvenile idiopathic arthritis

ObjectiveTo determine whether and how often the information to measure a set of key performance indicators (KPIs) in juvenile idiopathic arthritis (JIA) is found in data collected routinely in a Pediatric Rheumatology Clinic.MethodsA retrospective electronic chart review and administrative data analysis was conducted for a cohort of 140 patients with JIA at a tertiary Pediatric Rheumatology Clinic between 2016–2020. The set of KPIs include measuring patient outcomes (joint assessment, physician’s global assessment of disease activity, assessment of functional ability, composite disease activity measurement), access to care (waiting time between referral and first visit, visit with the rheumatologist within the first year of diagnosis, annual follow-up visits with the rheumatologist), and safety (tuberculosis screening, and laboratory monitoring). Documentation was assessed as a binary variable indicating whether the required information was ever found. Documentation frequency for each KPI was assessed with counts and percentages of the number of times the required information was documented for each clinic visit. Compliance with the safety KPI definitions was assessed using administrative databases.ResultsData for each KPI were found at least once in the cohort and documentation varied in frequency and consistency. Access to care and safety KPIs were documented more frequently than patient outcome KPIs. A joint assessment was documented at every visit for 95% of patients, 46% for an assessment of pain, and none for a physician’s global assessment of disease activity, an assessment of functional ability, or a composite disease activity measurement.ConclusionAlthough feasible to measure, there is an opportunity for improving the consistency of documentation. Having an active system of monitoring KPIs and tools to simplify measurement is a key step in the process toward improved patient care outcomes. Streamlining the collection of KPI data can increase the likelihood of compliance. Next steps should involve replicating this study in various centres.

Factors Associated With Maintenance of Remission Following Change From Combination Therapy to Monotherapy in Patients With Rheumatoid Arthritis.

Some patients with rheumatoid arthritis (RA) who persist in remission may decide to stop their therapy. We evaluated baseline characteristics associated with remaining in remission or low disease activity (LDA) following medication withdrawal. The Study of Etanercept and Methotrexate in Combination or as Monotherapy in Subjects With Rheumatoid Arthritis (SEAM-RA) was a phase III, multicenter, randomized withdrawal, double-blind, controlled study in patients with RA on methotrexate (MTX) + etanercept (ETN). If remission (Simplified Disease Activity Index [SDAI] ≤ 3.3) was sustained through a 24-week run-in period, patients then entered a 48-week double-blind period and were randomized 2:2:1 to receive MTX monotherapy, ETN monotherapy, or continue combination therapy. Multivariate logistic regression analysis was performed to identify baseline factors associated with remission or LDA at the end of both periods. Of 371 patients enrolled, 253 entered the double-blind period. After adjusting for other factors, covariates associated with achieving SDAI remission at the end of the run-in period included younger age, longer duration of MTX treatment, and less severe clinical disease variables. Covariates associated with maintaining remission/LDA at the end of the 48-week double-blind period included lower patient global assessment of disease activity (PtGA), lower C-reactive protein, rheumatoid factor (RF) negativity, longer RA duration in the MTX arm, shorter duration of ETN treatment, and lower magnesium. These findings indicate patients with overall lower disease activity are more likely to remain in SDAI remission/LDA after switching from combination therapy to monotherapy. RF-negative status and lower PtGA scores were strongly associated with increased likelihood of remaining in remission/LDA with MTX or ETN monotherapy. (SEAM-RA; ClinicalTrials.gov: NCT02373813).