Inactive Disease Status Research Articles

Clinical outcomes of tocilizumab therapy in polyarticular and systemic juvenile idiopathic arthritis: a single-center analysis (2018-2022)

AbstractThis single-center retrospective study aimed to evaluate the safety and efficacy of Tocilizumab (TOC) in children with polyarticular (pJIA) and systemic juvenile idiopathic arthritis (sJIA) who exhibited inadequate responses to disease-modifying antirheumatic drugs (DMARDs) and biological modifiers (bDMARDs). Conducted at the Department of Pediatric Rheumatology, National Institute of Geriatrics, Rheumatology, and Rehabilitation in Warsaw, Poland, between 2018 and 2022, the study enrolled 29 patients diagnosed with JIA based on International League of Associations for Rheumatology (ILAR) criteria. The cohort comprised 13 sJIA and 16 pJIA patients, aged 2-18 years, receiving TOC treatment for 24 months. Safety and efficacy assessments included analysis of medical documentation, laboratory tests (CRP, ESR, WBC), and Juvenile Disease Activity Score (JADAS) 71 at baseline, 3, 6, 12, and 24 months post-treatment initiation. Significant reductions in CRP and ESR levels were observed within three months, with sustained improvement in JADAS71 scores over the 24-month treatment period. A substantial majority, 73.07% of patients, achieved inactive disease status or low disease activity, highlighting T0C’s effectiveness. Adverse effects were manageable, predominantly involving mild to moderate infections, with no serious adverse events or instances of macrophage activation syndrome (MAS). The study also noted a steroid-sparing effect of TOC, with a reduction in glucocorticoid usage among the cohort. Tocilizumab demonstrates substantial efficacy in reducing disease activity and improving clinical outcomes in patients with pJIA and sJIA, coupled with a favorable safety profile. These findings reinforce the role of TOC as a critical component of the therapeutic arsenal for JIA, offering hope for improved quality of life and disease management in this patient population.

Interferon-Gamma-Inducible Protein-10 (IP-10) and Tumor Necrosis Factor-α (TNF-α) as Serological Predictors of Active Disease Status in Localized Scleroderma.

We investigated the ability of a panel of immune-related cytokines and chemokines to predict the disease activity state in localized scleroderma (LS) subjects followed longitudinally. A total of 194 sera samples were obtained from 45 LS subjects with diverse types of LS (40% linear, 20% mixed, 16% craniofacial, 13% generalized, and 11% circumscribed) in our cohort. Cytokines/chemokines that were significantly elevated at the baseline active disease visit compared to the inactive disease state at follow-up were Interferon-Gamma-Inducible Protein (IP)-10 (p < 0.021) and Tumor Necrosis Factor (TNF)-α (p < 0.033). Mixed effect logit modeling identified IP-10 (Odds Ratio (OR) [95% confidence interval] = 2.1 [1.4, 3.2], p < 0.001), TNF-α (OR = 1.8 [1.1, 3.0], p = 0.016), and Monocyte Chemoattractant Protein (MCP)-1 (OR = 2.0 [1.1, 3.9], p = 0.034) as significant predictors of active disease status. These findings support earlier correlations between IP-10 and TNF-α with disease activity parameters in a cross-sectional Luminex™ serological study and may enhance clinical decision-making when disease activity is challenging to assess by clinical examination alone.

Plasma proteome profiling in giant cell arteritis

ObjectivesThis study aimed to identify plasma proteomic signatures that differentiate active and inactive giant cell arteritis (GCA) from non-disease controls. By comprehensively profiling the plasma proteome of both patients with...

Validation of the EQ-5D-Y-5L parent-proxy version among children with juvenile idiopathic arthritis

ObjectivesJuvenile idiopathic arthritis (JIA) is the most common type of arthritis among children. It can cause joint pain and permanent physical damage, which affects mobility and daily activities. The EQ-5D-Y-3L self-report version has been validated in JIA, but the validity of EQ-5D-Y-5L remains unknown. We examined the psychometric properties of the EQ-5D-Y-5L parent-proxy version among children with JIA.MethodsWe used data from the Understanding Childhood Arthritis Network Canadian-Dutch collaboration study cohort, including patients with new-onset JIA, and those starting or stopping biologics. Clinical data and the parent-proxy version of the childhood health assessment questionnaire (CHAQ) and EQ-5D-Y-5L were collected. We evaluated the ceiling and floor effect; convergent and divergent validity using Spearman’s rank correlation; known-group validity using one-way ANOVA (Analysis of Variance) and effect size; and informativity using Shannon’s evenness index.Results467 patient visits representing 407 patients were analyzed. The EQ-5D-Y-5L had no ceiling/floor effect. The EQ-5D-Y-5L showed good convergent (e.g., EQ-5D-Y-5L pain/discomfort dimension vs. CHAQ pain index (Spearman’s r = 0.74, 95% confidence interval (C.I.): 0.69–0.79)), divergent (e.g., EQ-5D-Y-5L pain/discomfort dimension vs. CHAQ eating dimension (Spearman’s r = 0.19, 95% C.I.: 0.09–0.29)) and known-group validity (e.g., mean EQ-5D-Y-5L level summary score for patients with inactive versus active disease status, 6.34 vs. 10.52 (p < 0.001, effect size = 1.20 (95% C.I.: 0.95–1.45)). Shannon’s evenness index ranged from 0.52 to 0.88, suggesting most dimensions had relatively even distributions.ConclusionsIn this patient sample, EQ-5D-Y-5L parent-proxy version exhibited construct validity and informativity, suggesting the EQ-5D-Y-5L can be used to measure the quality of life of children with JIA.

Serum Immunoglobulin Concentrations in Juvenile Idiopathic Arthritis Cases during Active and Inactive Disease States

Serum Immunoglobulin Concentrations in Juvenile Idiopathic Arthritis Cases during Active and Inactive Disease States

Fecal Urgency in Ulcerative Colitis: Impact on Quality of Life and Psychological Well-Being in Active and Inactive Disease States

Ulcerative colitis (UC) is a chronic, relapsing-and-remitting, potentially progressive form of inflammatory bowel disease (IBD) with multidimensional and often negative effects on patients' lives. Fecal urgency, the sudden and compelling desire to defecate, often accompanied by impaired bowel control leading to frequent and urgent trips to the bathroom, is a distressing symptom, experienced by more than 50% of patients with UC.1 Physicians frequently underestimate the burden of fecal urgency on patients' lives, with ramifications ranging from disruption in daily activities, social interactions, and emotional distress with resultant impairment in quality of life (QoL).2,3.

NMR-based metabolomics in giant cell arteritis and polymyalgia rheumatica sequential sera differentiates active and inactive disease.

Giant Cell Arteritis-(GCA) is an inflammatory disease following a chronic, relapsing course. The metabolic alterations related to the intense inflammatory process during the active phase and to the rapid impact of steroid treatment, remain unknown. The study aims to investigate the serum metabolome in active and inactive disease state. 110 serum samples from 50 patients [33-GCA and 17-Polymyalgia rheumatica-(PMR)] at 3 time points, 0-(V1: active disease), 1 and 6 months-(V2 and V3: remission) of treatment with glucocorticosteroids (GCs), were subjected to Nuclear Magnetic Resonance (NMR)-based metabolomic analysis. Multi- and univariate statistical analyses were utilized to unveil metabolome alterations following treatment. Distinct metabolic profiles were identified between activity and remission, independently to disease type. N-acetylglycoproteins and cholines of bound phospholipids, emerged as predictive markers of disease activity. Altered levels of 4 out of the 21 small molecules were also observed, including increased levels of phenylalanine, and decreased of glutamine, alanine, and creatinine in active disease. Metabolic fingerprinting discriminated GCA from PMR in remission. GCA and PMR patients exhibited characteristic lipid alterations as a response and/or adverse effect of GCs treatment. Correlation analysis showed that several identified biomarkers were further associated with acute phase reactants, C-Reactive Protein and Erythrocyte Sedimentation Rate. The NMR profile of serum metabolome could identify and propose sensitive biomarkers of inflammation. Metabolome alterations, following GCs treatment, could provide predictors for future steroid-induced side effects.

Opening Lecture

Ankylosing spondylitis (AS), also referred to as radiographic axial spondyloarthritis (r-axSpA), is a challenging, inflammatory rheumatic condition primarily impacting the sacroiliac joints and often extending to the spine. The prevalence of axSpA, encompassing both radiographic AS and non-radiographic axSpA, is estimated to vary between 0.2% and 0.5% among Chinese adults, depending on the classification criteria employed. Treatment objectives for AS focus on improving the patient’s quality of life, function, and social involvement by managing inflammation and other disease symptoms. To achieve these goals, the American College of Rheumatology (ACR), the Spondylitis Association of America (SAA), and the Spondyloarthritis Research and Treatment Network (SPARTAN) advocate the use of non-steroidal anti-inflammatory drugs (NSAIDs) as the first-line treatment for AS, followed by biologic disease-modifying anti-rheumatic drugs (bDMARDs) like TNF inhibitors. While it has been common practice to initiate treatment with a TNF inhibitor or IL-17 inhibitor, the updated recommendations from the Assessment of SpondyloArthritis International Society (ASAS) and the European League Against Rheumatism (EULAR) now suggest the consideration of Janus kinase (JAK) inhibitors for patients with persistent high disease activity despite conventional therapy. Furthermore, the updated guidelines propose switching to another biologic DMARD (TNF inhibitor or IL-17 inhibitor) or JAK inhibitor if the first biologic DMARD proves ineffective. Despite recent advancement, there remains an unmet need for effective therapies in the treatment of AS. Across various biologic studies, only 40-50% of patients achieve an ASAS40 response. Moreover, approximately one-third of patients fail to attain low disease activity according to the Ankylosing Spondylitis Disease Activity Score (ASDAS), and 65% do not achieve inactive disease status on ASDAS after one year of bDMARD therapy. There is a lack of oral treatment options for AS beyond NSAIDs, as all current biologics are administered via injection or infusion. Targeting JAK-mediated pathways with an oral option holds promise as a potential approach for treating adult AS patients.

Comparative Assessment of the Immunological Significance of Semaphorin 5A and Anti-Cyclic Citrullinated Peptide Antibodies in Iraqi Patients with Rheumatoid Arthritis

The objective of this research was to assess the predictive potential of Semaphorin 5A in comparison to Anti-Cyclic Citrullinated Peptide antibodies for forecasting disease progression and treatment responses among Iraqi patients with Rheumatoid Arthritis (RA). Conducted as a case-control study, the investigation encompassed a total of 150 participants, comprising 100 RA patients and 50 healthy individuals. The study took place at Baghdad Teaching Hospital over the period from November 2021 to February 2022. Enrolled participants were selected based on the 2010 criteria established by the American College of Rheumatology. The levels of biomarkers were assessed using the enzyme-linked immunosorbent assay (ELISA) method. The findings demonstrated a noteworthy elevation in the levels of both ACCP and Semaphorin 5A among RA patients compared to the control group (p&lt;0.001). Furthermore, these levels were found to be higher in individuals with active disease as opposed to those with inactive disease. In both active and inactive disease states, the levels of both ACCP and Semaphorin 5A remained considerably higher compared to the levels observed in the healthy control group (p&lt;0.001). In patients who had not undergone treatment, the levels of both ACCP and Semaphorin 5A exhibited considerably greater significance compared to those who received Methotrexate or etanercept (p&lt;0.001). A noteworthy and positive correlation was observed between ACCP and Semaphorin 5A, with a correlation coefficient of 0.476 (p&lt;0.001). The sensitivity for ACCP and Semaphorin 5A was determined to be 72% and 83% respectively, while the specificity values were 98% and 76%. The study's conclusion highlighted the promising prognostic potential of both ACCP and Semaphorin 5A, establishing them as potential biomarkers for distinguishing between Rheumatoid Arthritis patients and healthy individuals. Predict disease activity and response to methotrexate or etanercept. Active disease patients and without treatments patients ACCP and Semaphorin5A levels were higher than inactive and received treatments (methotrexate or etanercept).

Potential for sustaining remission in ankylosing spondylitis patients upon netakimab discontinuation

Introduction. Biological disease modifying drugs (bDMARD) in the treatment of ankylosing spondylitis (AS) have shown good results with the achievement and long-term preservation of remission. There is a discussion about the withdrawal of drugs without loss of effect in order to reduce the economic burden, drug load, adverse events, the possibility of interrupting therapy during surgical treatment.Aim. To evaluate the potential for sustaining the therapeutic effect of netakimab (NTK) after its discontinuation in patients with AS who have achieved remission.Materials and methods. A cohort of 11 patients diagnosed with ankylosing spondylitis (AS) who had achieved remission was included in this study. The patients were closely observed for 52 weeks after discontinuing NTK treatment. AS exacerbations, pain intensity, disease activity scores (BASDAI, ASDAS), enthesitis evaluations (MASES), functional impairments (BASMI and BASFI), C-reactive protein (CRP) and erythrocyte sedimentation rate (ESR) levels were documented, as well as radiographic and MRI assessments of the sacroiliac joints and spine were performed.Results. Out of the 11 patients, 5 (45.5%) experienced AS exacerbations within the 12-month observation period. Patients who developed flare-ups had higher baseline levels of BASDAI, ASDAS, BASMI, and CRP at the time of NTK discontinuation. They also had a longer disease duration and were older compared to patients without relapse (p &lt; 0.05). The presence of flare-ups was significantly associated (p &lt; 0.05) with a history of peripheral arthritis, previous treatment with IFN-alpha, and the number of comorbidities. By week 52 of the observation period, patients demonstrated a deterioration in both activity and functional limitations (p &lt; 0.05). Elevated ASDAS-CRP levels were found to be correlated (p &lt; 0.05) with higher radiographic stages of sacroiliitis, the presence of syndesmophytes, functional limitations based on BASMI at the time of drug discontinuation, and the absence of continuous NSAID use. Significant prolongation of remission was associated with a substantial decline in ASDAS-CRP under NTK treatment (rSp = 0.996; p &lt; 0.05), especially among younger patients (rSp = 0.607; p &lt; 0.05).Conclusions. Approximately half of the patients who discontinued NTK therapy after achieving clinical and laboratory remission were able to sustain it. Maintenance of remission for 1 year was more prevalent in younger patients with shorter duration of AS, achieving inactive disease status based on ASDAS-CRP, fewer functional limitations, absence of peripheral arthritis, and comorbidities. Nevertheless, regular patient monitoring is necessary to promptly identify disease recurrence.

Efficacy of anakinra treatment in pediatric rheumatic diseases: Our single-center experience.

This study aims to present our experience on anakinra, a recombinant interleukin-1 (IL-1) receptor antagonist, and efficacy results in pediatric rheumatic diseases in our clinic. Between July 1st, 2016 and July 1st, 2020, a total of 33 pediatric patients (18 males, 15 females; mean age: 6±3.4 years; range 4 to 13 years) with pediatric rheumatic diseases who were treated with anakinra were retrospectively analyzed. The patients with over one-month treatment period and followed for at least one year were included. Demographic and clinical findings, outcomes, adverse events, prior and/or additional treatments were collected at baseline, at 3 and 12 months of therapy. There were 33 patients with different pediatric rheumatic diseases (11 with systemic juvenile idiopathic arthritis [sJIA] complicated by macrophage activation syndrome [MAS], six with hyperimmunoglobulin-D syndrome, five with cryopyrin-associated periodic syndrome, five with familial Mediterranean fever, four with idiopathic recurrent pericarditis, one with NLRP12-associated periodic fever syndrome and one with unclassified systemic autoinflammatory disease), in the study group. The complete response was observed 69.7% of patients, partial response in 24.2%, and no response in 6.1% at three months of treatment. Inactive disease status was achieved in 45.5% of the patients with remission-on medication and 18.2% of the patients with remission-off medication at the end of a year. Anakinra was switched to other biological treatments in 51.5% of patients (n=17). Biological switch to canakinumab and tocilizumab were observed in 70.6% and 29.4% of these patients. Except for local reactions (n=2), no adverse events were observed in any of the patients. Anakinra appears to be a promising treatment alternative owing to its rapid effect as a result of its short half-life in autoinflammatory conditions. While short-term therapy seems to be sufficient for the sJIA complicated by MAS, the patients with systemic autoinflammatory diseases maintenance a more anakinra-dependent course.

Investigation of Inactive Disease States Among Patients With Juvenile Idiopathic Arthritis in the Childhood Arthritis and Rheumatology Research Alliance Registry.

ObjectiveInactive disease is the treatment goal for juvenile idiopathic arthritis (JIA), but there are multiple measures for disease activity. The objective was to compare individuals with JIA who meet different definitions for inactive disease.MethodsDisease activity measures were determined at the 1‐year follow‐up visit for all patients with JIA enrolled in a North American multicenter registry from 2015 to 2019, the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry. Patient and disease characteristics between those who met only one composite definition of inactive disease were compared by χ2 for categorical variables and Wilcoxon rank sum for continuous variables. The Spearman correlation coefficient was calculated for simple disease measures.ResultsAmong all 2904 patients with JIA enrolled in the CARRA Registry with 1‐year visit data, 1984 (68%) had no active joints, 1485 (51%) had a physician global score of 0, 1366 (47%) had a patient/parent global score of 0, 1293 (45%) met the American College of Rheumatology provisional criteria for clinical inactive disease (ACR CID), and 1325 (46%) had a clinical Juvenile Arthritis Disease Activity Score (cJADAS10) of 1 or less. Almost half (47%) did not meet either composite definition of inactive disease, and 38% met both ACR CID and cJADAS10 of 1 or less.ConclusionIn a multicenter cohort of patients with JIA in North America, a large proportion of patients had inactive disease by single or composite measures after 1 year of observation in the Registry. There was significant overlap between patients who met ACR CID criteria and those who had a cJADAS10 of 1 or less. Additional studies are needed to evaluate the reasons for discordance in inactive disease measures.

Myeloid-related protein 8/14 in plasma and serum in patients with new-onset juvenile idiopathic arthritis in real-world setting in a single center

ObjectiveThe aim of this study was to analyze the usefulness of myeloid-related protein 8/14 (MRP8/14) in the prediction of disease course in a real-world setting for patients with new-onset juvenile idiopathic arthritis (JIA), to identify the relationship between MRP8/14 and disease activity using the physician’s global assessment of disease activity (PGA), and determine whether the MRP8/14 levels measured in serum and plasma are equally useful.MethodsIn this prospective follow-up study, 87 new-onset non-systemic JIA patients were studied. Blood and synovial fluid samples were collected prior to any antirheumatic medication use. MRP8/14 was measured from serum (S-MRP8/14), plasma (P-MRP8/14), and synovial fluid samples using ELISA.ResultsThe baseline MRP8/14 blood levels were significantly higher in patients using synthetic antirheumatic drugs than in patients with no systemic medications at 1 year after diagnosis in serum (mean 298 vs. 198 ng/ml, P < 0.001) and in plasma (mean 291 vs. 137 ng/ml, P = 0.001). MRP8/14 levels at the time of JIA diagnosis were higher in patients who started methotrexate during 1.5-year follow-up compared to those who achieved long-lasting inactive disease status without systemic medications (serum: mean 298 vs. 219 ng/ml, P = 0.006 and plasma: 296 vs. 141 ng/ml, P = 0.001). P-MRP8/14 was the most effective predictive variable for disease activity (by PGA) in linear multivariate regression model (combined to ESR, CRP, leukocytes, and neutrophils), whereas S-MRP8/14 was not significant.ConclusionBlood MRP8/14 levels at baseline seem to predict disease course in new-onset JIA patients. P-MRP8/14 might be better than S-MRP8/14 when assessing disease activity at the time of JIA diagnosis.

OP0023 A RANDOMIZED, DOUBLE-BLIND TRIAL COMPARING SECUKINUMAB 300 MG AND 150 MG AT WEEK 52 IN PATIENTS WITH ANKYLOSING SPONDYLITIS WHO DID NOT ACHIEVE INACTIVE DISEASE DURING AN INITIAL 16 WEEKS OF OPEN-LABEL TREATMENT WITH SECUKINUMAB 150 MG

OP0023 A RANDOMIZED, DOUBLE-BLIND TRIAL COMPARING SECUKINUMAB 300 MG AND 150 MG AT WEEK 52 IN PATIENTS WITH ANKYLOSING SPONDYLITIS WHO DID NOT ACHIEVE INACTIVE DISEASE DURING AN INITIAL 16 WEEKS OF OPEN-LABEL TREATMENT WITH SECUKINUMAB 150 MG

Soluble interleukin-2 receptor serum levels facilitate prediction of relapses in subgroups of patients with juvenile idiopathic arthritis.

JIA is characterised by a chronic disease course. Once patients achieve a state of inactive disease, there are no established biomarkers to predict the further course of inflammation for these patients. Therefore, the purpose of this study was to quantify serum biomarkers during quiescent disease to evaluate their use in identifying JIA patients at risk for future disease flare. Patients with non-systemic JIA reaching inactive disease status were divided into two groups: 92 patients with future active disease after a median period of 6 months (range 3-9) and 80 patients with persistent inactive disease for the following period (median 11 months, range 7-16) according to the juvenile arthritis DAS (JADAS). Clinical parameters and serum levels of various biomarkers were measured in the state of inactive disease using immunoassays in both groups and were analysed for their potential to predict the further course of disease. Soluble interleukin-2 receptor (sIL-2R) serum levels were significantly higher in patients with future active disease (P = 0.021), which especially applied to patients with RF-negative polyarticular and extended oligoarticular JIA (P < 0.001). Higher sIL-2R serum levels during inactive disease were associated with a greater number of active joints at future active disease. Patients without clinical signs of disease activity already presented with increased sIL-2R serum levels several months before disease relapses, whereas conventional inflammation parameters were not elevated. Determination of sIL-2R serum levels during inactive disease may facilitate identifying patients with subclinical disease activity at risk for future active disease.

Эффективность и безопасность отмены ингибиторов фактора некроза опухоли альфа при длительной ремиссии ювенильного идиопатического артрита без системных проявлений: когортное исследование

The biological agents when long-term clinical remission of juvenile idiopathic arthritis (JIA) is achieved is necessary to reduce the risk of side effects and costs and to minimize the non-economic burden of therapy on patients and their families. Objective. To evaluate the efficacy and safety of discontinuing inhibitors of tumor necrosis factor alpha (TNF-α) in patients with JIA without systemic manifestations due to long-term clinical remission. Patients and methods. A total of 137 patients with JIA (27.0% male, mean age: 10 years) were enrolled in this study, of whom 95 (69.3%) received etanercept (0.8 mg/kg/week) and the rest – adalimumab (24 mg/m2 once every two weeks). The retrospective study included patients with JIA without systemic manifestations and disease remission of ≥24 months, during which the patients received TNF-α inhibitors. The latter were discontinued as directed by a physician or due to unavailability of the drug. The primary outcome measure of the study was maintenance of clinically inactive disease status (according to American College of Rheumatology criteria) for 6 months (&lt;210 days including 30 days’ tolerance for deviation from the recommended rehospitalization time) after discontinuation of therapy with TNF-α inhibitors. Results. At the time of TNF-α withdrawal, the median duration of JIA was about 6 years, the duration of TNF-α therapy was 3.5 years, and remission lasted for 3 years. After TNF-α withdrawal, 60 (43.8%) patients continued therapy with traditional disease-modifying antirheumatic drugs (DMARs). In the first 210 days after TNF-α discontinuation, 92 (67.2%) patients with JIA maintained the inactive disease status. The median time for the development of JIA exacerbations in 45 patients was 123 (95; 150) days. Biological agents therapy was resumed in 42 of 45 patients, and the inactive disease status was achieved in 41 cases. DMARs were received by 23 (55%) of 42 patients on therapy with biological agents. Conclusion. In two thirds of patients with JIA, the discontinuation of TNF-α inhibitors due to long-term clinical remission did not lead to disease exacerbations in the next six months. The resumption of biological agents therapy in case of JIA worsening allows most patients to reach the status of clinically inactive disease. Key words: genetically engineered biopharmaceutical drugs, tumor necrosis factor inhibitors, juvenile idiopathic arthritis, therapy withdrawal, long-term remission, clinically inactive disease

Possibility of treatment modification in patients with ankylosing spondylitis achieved of partial remission on treatment with adalimumab: Real practice data

Objective . To assess the duration of remission or inactive disease status in patients with achieved partial remission due to treatment with adalimumab (ADA) after it discontinuation. Materials and methods . A dynamic observation was conducted of 26 patients with ankylosing spondylitis with partial remission achieved due to prolonged use (for 24 months or more) of subcutaneous injections of 40 mg ADA once every two weeks. The discontinuation of ADA was carried out after a 3-4 month period of its use in de-escalation mode in the form of 1 injection (40 mg) once every 4 weeks. After discontinuation of ADA, patients continued to take non-steroidal anti-inflammatory drugs, sulfasalazine at a dose of 1.5-2 g per day (11 patients) and methotrexate 10 mg per week (3 patients). Assessment of the clinical, laboratory and ultrasonographic parameters of ankylosing spondylitis was carried out in 3, 6 and 12 months after the abolition of the ADA. Results and discussion . Among the observed patients, partial clinical remission of ankylosing spondylitis maintained after 6 months in 12 (46.2%) patients, and after 12 months - in 10 (38.5%). A low degree of ankylosing spondylitis activity (BASDAI &lt;4) was maintained for 6 months after the abolition of ADA in 14 (53.8%) patients, and after 12 months - in 12 (46.2%). Conclusion . Within 12 months after cancellation of ADA, ankylosing spondylitis exacerbation was observed in 53.8% of patients with initially achieved partial clinical remission.

Early combination therapy with etanercept and methotrexate in JIA patients shortens the time to reach an inactive disease state and remission: results of a double-blind placebo-controlled trial

BackgroundRemission is the primary objective of treating juvenile idiopathic arthritis (JIA). It is still debatable whether early intensive treatment is superior in terms of earlier achievement of remission. The aim of this study was to evaluate the effectiveness of early etanercept+methotrexate (ETA+MTX) combination therapy versus step-up MTX monotherapy with ETA added in refractory disease.MethodsA multi-centre, double-blind, randomized study in active polyarticular JIA patients treated with either ETA+MTX (n = 35) or placebo+MTX (n = 33) for up to 24 weeks, followed by a 24-week open-label phase. The efficacy endpoints included pedACR30 criteria improvement at week 12, inactive disease at week 24, and remission at week 48. Patients who failed to achieve the endpoints at week 12 or at week 24 escaped to open-label ETA+MTX. Safety was assessed at each visit.ResultsBy intention-to-treat analysis, more patients in the ETA+MTX group reached the pedACR30 response at week 12 (33 (94.3%)) than in the placebo+MTX group (20 (60.6%); p = 0.001). At week 24, comparable percentages of patients reached inactive disease (11 (31.4%) vs 11 (33.3%)). At week 48, 11 (31.4%) and eight (24.2%) patients achieved remission. The median (+/−IQR) times to achieve an inactive disease state in the ETA+MTX and placebo+MTX groups were 24 (14–32) and 32 (24–40) weeks, respectively. Forty-four (74/100 patient-years) adverse events (AEs) were reported, leading to treatment discontinuation in 6 patients.ConclusionsEarly combination therapy with ETA+MTX proved to be highly effective compared to the standard step-up regimen. Compared to those treated with the standard regimen, more patients treated with a combination of ETA+MTX reached the pedACR30 response and achieved inactive disease and remission more rapidly.

Myositis-specific autoantibodies and their associated phenotypes in juvenile dermatomyositis: data from a German cohort.

To describe a German cohort of patients with juvenile dermatomyositis (JDM) and to evaluate clinical manifestations, disease course and prognosis in JDM patients with a certain myositis-specific autoantibody. Cross-sectional data on patients with JDM documented in the National Paediatric Rheumatologic Database in Germany between 2014 and 2016 were analysed. In a subgroup of the cohort, MSAs were determined with a commercial multiplex array, and a retrospective chart review was conducted to specify the clinical phenotype and patient outcome. The total cohort consisted of 196 patients with JDM (mean age 12.2±4.0 years, mean disease duration 5.1±3.8 years, 70% female). Apart from typical skin changes and muscle weakness, 41% of patients also had arthritis and/or contractures, 27% had calcinosis and approximately 10% had interstitial lung disease. Immunoblot testing was performed on the sera of 91 (46%) patients, detecting MSAs in 44% of patients. Patient groups with specific MSAs differed in clinical characteristics such as calcinosis, dysphagia, and lung and joint involvement. The extent of muscle weakness evaluated by the Childhood Myositis Assessment Scale was significantly associated with an increased level of creatine kinase. Patients with anti-MDA5 were particularly affected by polyarthritis of the small joints. After 5 years, 51 patients of the MSA cohort (56.0%) achieved an inactive disease state, 12/51 (23.5%) were off therapy. Patients with JDM in Germany show a broad spectrum of clinical manifestations that can be grouped into homogeneous groups using MSA, which also helps to predict the course and prognosis of the disease.

Cochlear Implantation in Patients With Menière's Disease: Does Disease Activity Affect the Outcome?

Menière's disease (MD) is characterized by episodes of vertigo, tinnitus, and sensorineural hearing loss. In the setting of bilateral deafness due to MD alone or contralateral pathology, cochlear implantation (CI) improves hearing. Active MD is characterized by fluctuating auditory symptoms and vertigo; whereas remittance of vertiginous symptoms and severe, permanent sensorineural hearing loss characterizes the inactive disease state. This study evaluates outcomes for MD patients compared with the general CI population and assesses if disease activity affects implant outcomes. Retrospective chart review. Tertiary referral center. Twenty-four patients with MD that received CI (7 active, 16 inactive, and 1 Probable Menière's), and 24 age-matched controls. Cochlear implantation. Word Recognition Score, Sentence Recognition Score (SRS), and Speech Reception Threshold. Best-aided preoperative and postoperative audiometric data were compared per ear between MD patients and controls and stratified by disease status using descriptive statistics with mixed-effects modeling. Patients with MD derived significantly more benefit from CI than controls when comparing differences between preoperative and postoperative levels for Word Recognition Score (12.2%, p = 0.0236), SRS (12.8%, p = 0.0375), and Speech Reception Threshold (-14.4 dB, p = 0.0188). Active disease status does not negatively impact CI outcomes and patients with active MD may benefit from greater gains in SRS (23.5%, p = 0.0107). CI provides greater gains in functional hearing for patients with MD compared with age-matched controls. Patients with active MD seem to perform better with respect to SRS following CI than patients with inactive status.