Rheumatology and Therapy最新文献

{"title":"Factors Influencing Treatment Satisfaction and Recognition Gaps Between Physicians and Patients with Systemic Sclerosis.","authors":"Yoshihito Shima, Mona Uchida-Yamada, Sei-Ichiro Motegi, Taku Shimada, Haruka Ishii, Yoshito Ohya, Yasumasa Kanai","doi":"10.1007/s40744-025-00745-x","DOIUrl":"10.1007/s40744-025-00745-x","url":null,"abstract":"<p><strong>Introduction: </strong>It is important to understand the differences in patient-physician perceptions and factors affecting satisfaction with treatment in patients with systemic sclerosis (SSc).</p><p><strong>Methods: </strong>This web-based survey (conducted in Japan in March 2023) targeted patients aged ≥ 18 years with SSc and physicians in hospitals with ≥ 20 beds and seeing ≥ 3 patients with SSc monthly. Physicians and patients answered similar questions.</p><p><strong>Results: </strong>Responders were 301 patients (63.8% female; 47.5% limited cutaneous SSc; 44.9% diffuse cutaneous SSc) and 129 physicians (51.2% rheumatologists; 20.9% dermatologists). The most common problematic symptoms reported by patients having each symptom were Raynaud's phenomenon (RP) (59.5%), skin tightening (47.4%), and malaise (45.5%). Physicians also perceived RP as the common problematic symptoms (46.5%). Conversely, there was a large gap in the perception of malaise as problematic (5.4%). There was a ≥ 20% difference in the percentage of respondents who felt that treatments improved symptoms of reflux esophagitis (48.8% in patients vs. 76.7% in physicians), dysphagia (25.0% vs. 52.7%), constipation (35.1% vs. 62.8%), diarrhea (36.1% vs. 62.8%), and pain (47.6% vs. 69.0%). Patient characteristics associated with high satisfaction with treatment included treatment responsiveness, age ≥ 50 years, being anti-topoisomerase I antibody positive, having dermatological or digestive symptoms as problematic symptoms, and not feeling they should have seen their physician earlier.</p><p><strong>Conclusions: </strong>Patients and physicians had different perceptions of symptoms and treatment response. Patients' perception of improvement affected their satisfaction with treatment. Reviewing treatment goals and content between patients and physicians is necessary to improve treatment satisfaction.</p><p><strong>Trial registration: </strong>UMIN000050368.</p>","PeriodicalId":21267,"journal":{"name":"Rheumatology and Therapy","volume":" ","pages":"297-314"},"PeriodicalIF":2.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11920495/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143190491","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Prevalence, Incidence and Management of Systemic Lupus Erythematosus in Germany: A Retrospective Claims Data Analysis.","authors":"Tobias Alexander, Philipp Sewerin, Anja Strangfeld, Marcus Schulte, Julia Borchert, Tarcyane Barata Garcia, Eva Schrom","doi":"10.1007/s40744-024-00735-5","DOIUrl":"10.1007/s40744-024-00735-5","url":null,"abstract":"<p><strong>Introduction: </strong>This study evaluated the prevalence and incidence of systemic lupus erythematosus (SLE) in Germany and explored real-world data on sequence of therapy (SOT; sequence of drugs as prescribed in clinical practice).</p><p><strong>Methods: </strong>This retrospective, observational, longitudinal cohort study using German claims data from the WIG2 GmbH Scientific Institute for Health Economics and Health System Research database (January 2011-December 2019), extrapolated to the statutory health insurance (SHI)-insured population, evaluated prevalence and incidence in an epidemiological analysis group and SLE treatment patterns in an incident cohort (subgroup ≥ 18 years of age with incident disease and ≥ 24-month follow-up post index date). Analyses were descriptive.</p><p><strong>Results: </strong>Based on the epidemiological analysis (N = 3017), annual SLE prevalence per 100,000 gradually increased from 40.47 in 2012 to 59.87 in 2019 in the SHI population. In contrast, annual SLE incidence was relatively stable, ranging from 8.83 in 2012 to 8.86 in 2019. In the incident cohort (n = 941), based on SOT analysis (n = 681), treatment gaps of > 60 days were common: 67.1%, 51.2% and 54.9% in SOT1, SOT2 and SOT3, respectively. Corticosteroids were the most frequent monotherapy in SOT1 (31.0% vs 0% in SOT2/SOT3); 30.0-70.0% of patients received a corticosteroid combination therapy across SOTs. Over 50% of patients in each SOT received an antimalarial therapy (combination or monotherapies). The use of biologic disease-modifying drugs was low, ranging from 0.4% in SOT1 to 9.7% in SOT3.</p><p><strong>Conclusions: </strong>Our data demonstrate an increased prevalence of SLE with stable incidence in Germany, suggesting improved survival of affected patients. Nevertheless, suboptimal treatment patterns, including limited use of biologics, reflect a high unmet need for optimised and personalised therapies in patients with SLE.</p>","PeriodicalId":21267,"journal":{"name":"Rheumatology and Therapy","volume":" ","pages":"237-254"},"PeriodicalIF":2.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11920567/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142954156","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Osteoporosis Treatment Gap and Costs in Spain: Data from Women with a First Fragility Fracture or Diagnosis of Postmenopausal Osteoporosis.","authors":"Antoni Sicras-Mainar, Francesc Sorio-Vilela, Marta Sacrest-Soy, Sonia Gatell, Aram Sicras-Navarro, Renata Villoro-Valdés, Elena Rebollo-Gómez, Ignacio Hernández","doi":"10.1007/s40744-024-00743-5","DOIUrl":"10.1007/s40744-024-00743-5","url":null,"abstract":"<p><strong>Introduction: </strong>Postmenopausal osteoporosis (PMO) increases the risk of fragility fractures (FF), leading to disability, higher mortality, and elevated healthcare costs. Despite available treatments, osteoporosis (OP) remains undertreated, especially in women over 50 years at high risk for FF. Real-world data on OP care in Spain are limited. This study aims to assess the OP treatment gap, healthcare resource utilisation (HCRU), and costs among Spanish women following a first FF or PMO diagnosis.</p><p><strong>Methods: </strong>This retrospective study used data from the BIG-PAC^® administrative database on women aged ≥ 50 years with a first FF (cohort 1) or newly diagnosed PMO (cohort 2) between 2014 and 2018. Patients were followed for 2 years after the index event. The primary outcome was the proportion of women not prescribed OP medication within 6 months after the index event (treatment gap). Secondary outcomes included fracture incidence, mortality, HCRU, and costs.</p><p><strong>Results: </strong>The study included 22,142 women: 3190 in cohort 1 and 18,952 in cohort 2. The OP treatment gap was higher in cohort 1 vs cohort 2 (41.5% vs 23.6%). In cohort 1, 59.2% were diagnosed with PMO after the first FF, with 88% experiencing subsequent fracture(s). OP treatment persistence decreased over time in both cohorts. Fracture rates were lower in women prescribed OP treatment vs those who were not (8.35 vs 13.8 per 1000 patient-years) and in those who showed 24-month-persistence and 12-month adherence to treatment vs those who did not (8.98 and 7.66 vs 10.79 and 10.76). The 2-year mean cost per patient was higher in cohort 1 (€10,601) than in cohort 2 (€1659), with the highest costs incurred for hip (€15,833) and vertebral (€10,593) fractures.</p><p><strong>Conclusion: </strong>This study highlights a significant treatment gap in Spanish women aged ≥ 50 with a first FF or newly diagnosed PMO. Costs are particularly high for those with a first FF, especially for hip or vertebral fractures. Improving treatment adherence could reduce fracture risk, healthcare costs, and resource utilisation.</p>","PeriodicalId":21267,"journal":{"name":"Rheumatology and Therapy","volume":" ","pages":"315-335"},"PeriodicalIF":2.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11920490/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143391663","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World-Evidence of Digital Health Applications (DiGAs) in Rheumatology: Insights from the DiGAReal Registry.","authors":"Alexander Albrecht, Jule Taubmann, Ioanna Minopoulou, Lukas Hatscher, Stefan Kleinert, Felix Mühlensiepen, Martin Welcker, Jan Leipe, Nils Schulz, Philipp Klemm, Axel Hueber, Georg Schett, Sebastian Kuhn, Hannah Labinsky, Johannes Knitza","doi":"10.1007/s40744-025-00744-y","DOIUrl":"10.1007/s40744-025-00744-y","url":null,"abstract":"<p><strong>Introduction: </strong>Prescribable digital health applications (DiGAs) present scalable solutions to improve patient self-management in rheumatology, however real-world evidence is scarce. Therefore, we aimed to assess the effectiveness, usage, and usability of DiGAs prescribed by rheumatologists, as well as patient satisfaction.</p><p><strong>Methods: </strong>The DiGAReal registry includes adult patients with rheumatic conditions who received a DiGA prescription. Data at baseline (T0) and the 3-month follow-up (T1) were collected through electronic questionnaires. Study outcomes included DiGA-specific outcome assessments as well as generic outcome assessments, including the Patient Global Impression of Change (PGIC), Patient Activation Measure (PAM®), and the German Telehealth Usability and Utility Short Questionnaire (TUUSQ). Changes between T0 and T1 were analyzed using descriptive statistics and paired tests.</p><p><strong>Results: </strong>A total of 191 patients were included between June 2022 and April 2023. Of these, 127 completed the 3-month follow-up, and 114 reported using the prescribed DiGA, with 66% reporting weekly use and 15% completing the full DiGA program. The most commonly prescribed DiGAs targeted pain management (53%). Symptom improvement was reported by 51% of patients using a DiGA, with significant reductions in exhaustion levels (p = 0.03). Significant DiGA-specific improvements were observed for DiGAs addressing back pain (p = 0.05) and insomnia (p = 0.006). However, no overall significant changes were detected in patient activation, health literacy, pain, overall health, or disease activity. Back pain and weight management DiGAs were the most effective, frequently used, and best-rated DiGAs, with symptom improvements reported by 50% to 82% of patients.</p><p><strong>Conclusion: </strong>The findings suggest that DiGAs can improve symptom management in rheumatic patients, especially for conditions like back pain and weight control. Further real-world evidence is needed and may support value-based digital health efforts and reimbursement frameworks.</p>","PeriodicalId":21267,"journal":{"name":"Rheumatology and Therapy","volume":" ","pages":"267-282"},"PeriodicalIF":2.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11920491/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143010846","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Improvement of Fatigue in Patients with Ankylosing Spondylitis Receiving Tofacitinib: Analyses of a Phase 3 Randomized Controlled Trial.","authors":"Laure Gossec, Jessica A Walsh, Raj Sengupta, Andrew G Bushmakin, Joseph C Cappelleri, Arne Yndestad, Oluwaseyi Dina, David Cella","doi":"10.1007/s40744-024-00727-5","DOIUrl":"10.1007/s40744-024-00727-5","url":null,"abstract":"<p><strong>Introduction: </strong>Fatigue is a key symptom in patients with ankylosing spondylitis (AS). The objective of this analysis was to estimate the median time to initial and stable improvement events in fatigue in patients with AS receiving tofacitinib.</p><p><strong>Methods: </strong>This post hoc analysis used data from a phase 3 trial (NCT03502616) in patients with active AS receiving tofacitinib 5 mg twice daily or placebo. Time to improvement in fatigue was assessed using the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) total score, experience domain score, and impact domain score. The rapidity of improvement was assessed by time-to-event analyses (nonparametric Kaplan-Meier models); initial improvement events (i.e., time to first week of FACIT-F improvement) and stable improvement events (i.e., time to first week of FACIT-F improvement, sustained to 16 weeks) were examined.</p><p><strong>Results: </strong>Overall, 269 patients were assessed (mean disease duration: 14.2 [standard deviation (SD): 9.8] years; mean baseline FACIT-F total score: 27.2 [SD: 9.3]). Median times to initial and stable improvement events in FACIT-F total and domain scores were significantly shorter and occurred in more patients receiving tofacitinib than placebo. Median time to initial and stable improvement events of 6 points in FACIT-F total score were 8 and 12 weeks with tofacitinib, respectively (placebo: not reached); 70.0% versus 48.5% of patients receiving tofacitinib versus placebo, respectively, experienced initial improvements of 6 points in FACIT-F total score within 16 weeks.</p><p><strong>Conclusions: </strong>Improvements in fatigue occurred more rapidly with tofacitinib than with placebo. These results may be useful for healthcare providers when discussing tofacitinib treatment expectations with patients.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov: NCT03502616 (June 7, 2018).</p>","PeriodicalId":21267,"journal":{"name":"Rheumatology and Therapy","volume":" ","pages":"85-98"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751249/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142819030","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluation of the \"Multivariable Psoriatic Arthritis Risk Estimation Tool\" in a Cohort of Patients with Psoriasis: Preliminary Results of a Prospective Observational Study.","authors":"Ennio Lubrano, Filomena Mandato, Marcella Antenucci, Fabio Massimo Perrotta","doi":"10.1007/s40744-024-00729-3","DOIUrl":"10.1007/s40744-024-00729-3","url":null,"abstract":"<p><strong>Introduction: </strong>An intriguing aspect that emerged in recent years is the transition phase from psoriasis (PsO) to psoriatic arthritis (PsA). The PRESTO instrument allows estimating a patient's risk of developing PsA based on a few clinical items. The aim of this study was to apply and evaluate the performance of the PRESTO tool in a cohort of patients with PsO.</p><p><strong>Methods: </strong>Consecutive patients with PsO were enrolled. Dermatological and rheumatological assessment was carried out in order to evaluate clinical features of PsO, to exclude the diagnosis of PsA, and to administer the PRESTO tool.</p><p><strong>Results: </strong>Between January 1, 2024 and April 1, 2024, 100 patients were assessed. Eight-four patients found the questionnaire to be very useful and easy. The estimated risk (median/IQR) of 1-year progression to PsA found in our group was 2.45% at 1 year (1.1-4).</p><p><strong>Conclusions: </strong>The PRESTO instrument was feasible and well accepted by patients. The 1-year risk assessed by PRESTO tools is consistent with other reports in the literature.</p>","PeriodicalId":21267,"journal":{"name":"Rheumatology and Therapy","volume":" ","pages":"203-209"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751210/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142785732","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Tofacitinib Efficacy/Safety in Patients with Ankylosing Spondylitis by Baseline Body Mass Index: A Post Hoc Analysis of Phase 2/3 Trials.","authors":"Hillary Norton, Paula Sliwinska-Stanczyk, Tomas Hala, Bassel El-Zorkany, Lori Stockert, Rajiv Mundayat, Lisy Wang, Christopher T Ritchlin","doi":"10.1007/s40744-024-00726-6","DOIUrl":"10.1007/s40744-024-00726-6","url":null,"abstract":"<p><strong>Introduction: </strong>We assessed tofacitinib efficacy and safety in ankylosing spondylitis (AS) by body mass index (BMI) category.</p><p><strong>Methods: </strong>Data were pooled from phase 2/3 trials; analyses included patients with active AS randomized (1:1) to tofacitinib 5 mg twice daily or placebo, who were stratified by baseline BMI into < 25, ≥ 25 to < 30, and ≥ 30 kg/m² categories. Efficacy was assessed at week 12 and safety to week 16.</p><p><strong>Results: </strong>Of 370 patients, 153, 131, and 86 had a baseline BMI  of < 25, ≥ 25 to < 30, and ≥ 30 kg/m², respectively. At baseline, patients with BMI < 25 kg/m² were younger and more likely to be current smokers/Asian, and patients with BMI ≥ 30 kg/m² had higher mean waist circumference/swollen joint count (SJC) and were more likely to have enthesitis, high-sensitivity C-reactive protein (hsCRP) > 5 mg/L, an inadequate response to tumor necrosis factor inhibitors (TNFi), and prior biologic disease-modifying anti-rheumatic drug (bDMARD) use versus other categories. Across categories, tofacitinib responses/improvements were greater than with placebo, except for ≥ 40% Assessment of SpondyloArthritis international Society improvement (ASAS40), ASAS partial remission, 50% improvement from baseline in the Bath Ankylosing Spondylitis Disease Activity Index score (BASDAI50), and Ankylosing Spondylitis Disease Activity Score using C-reactive protein (ASDAS-CRP) inactive disease rates, which were similar for tofacitinib and placebo in the BMI ≥ 30 kg/m² category. Treatment effects were similar across categories, except for BASDAI50, which was smaller in the BMI ≥ 30 category versus  the < 25 kg/m² category. More adverse events (AEs) and serious adverse events (SAEs) with tofacitinib were reported in the BMI < 25 kg/m² category, which had a higher proportion of current smokers versus other categories.</p><p><strong>Conclusions: </strong>Regardless of baseline BMI category, efficacy was greater with tofacitinib versus placebo in patients with AS, and no treatment effect differences between categories were observed, with exceptions for BMI ≥ 30 kg/m² (more active/treatment-refractory disease and a smaller sample size). Overall, tofacitinib safety was generally comparable across categories; however, AE/SAE rates with tofacitinib were higher in the BMI < 25 kg/m² category (which had more current smokers). This post hoc analysis demonstrates that tofacitinib can be considered as a treatment option for AS, regardless of baseline BMI category; however, interpretation was limited by small sample sizes and differences in sample sizes and baseline characteristics across categories.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifiers, NCT01786668 and NCT03502616.</p>","PeriodicalId":21267,"journal":{"name":"Rheumatology and Therapy","volume":" ","pages":"67-84"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751278/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142786119","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"High-Titer Rheumatoid Factor is Associated with Worse Clinical Outcomes and Higher Needs for Advanced Therapies in Rheumatoid Arthritis Under Real-Life Conditions.","authors":"Victor Davi R S Oliveira, Ana Paula M G Reis, Claiton V Brenol, Ivânio A Pereira, Karina R Bonfiglioli, Letícia R Pereira, Manoel B Bértolo, Maria de Fátima L C Sauma, Maria Fernanda B R Guimarães, Paulo Louzada-Júnior, Rina D N Giorgi, Sebastião C Radominski, Licia Maria H Mota, Cleandro P Albuquerque, Geraldo R Castelar-Pinheiro","doi":"10.1007/s40744-024-00730-w","DOIUrl":"10.1007/s40744-024-00730-w","url":null,"abstract":"<p><strong>Introduction: </strong>Rheumatoid factor (RF) plays an important role in rheumatoid arthritis (RA) pathophysiology, yet the differential effects of varying RF titers remain understudied. We evaluated associations between different RF titers and clinical outcomes in long-standing RA.</p><p><strong>Methods: </strong>This multicenter, cross-sectional study included adults meeting ACR/EULAR (2010) criteria for RA. Circulating RF titers and clinical-epidemiological characteristics were evaluated. Bivariate (Student's t and chi-squared tests) tests and multiple logistic and linear regression analyses were conducted.</p><p><strong>Results: </strong>We included 1097 participants; 78.7% had positive RF, with high titers (≥ 3 × the upper limit of normality) in 56.2%. Negative vs. low-positive RF groups performed similarly concerning all clinical outcomes, being subsequently aggregated as \"non-high\" RF group. High RF titers (compared to \"non-high\") were associated with tobacco use (odds ratio, OR [95% confidence interval, CI]: 2.04 [1.35, 3.08]; p < 0.001), multiraciality (OR [95% CI] 1.31 [1.03, 1.67]; p = 0.028, compared to White race), and higher body mass index (mean difference [95% CI] 0.69 [0.05, 1.33] kg/m²; p = 0.033). In multivariate analyses, high-titer RF was independently associated with higher disease activity (Clinical Disease Activity Index, CDAI: β = 2.44 [0.89, 3.99], p = 0.002), worse functional capacity (Health Assessment Questionnaire Disability Index, HAQ-DI: β = 0.112 [0.018, 0.205], p = 0.020); extra-articular manifestations (OR 1.48 [1.09, 2.00], p = 0.011); increased corticosteroid (OR 1.53 [1.19, 1.96], p = 0.001) and biological disease-modifying antirheumatic drugs (bDMARD) use (OR 1.41 [1.08, 1.84], p = 0.011).</p><p><strong>Conclusions: </strong>High RF titers in long-standing RA were associated with worse disease activity, lower physical functionality, increased extra-articular manifestations, and higher usage of corticosteroids and bDMARDs. Comparing high vs. non-high RF titers (rather than positive vs. negative RF) seems more useful for evaluating the clinical effects of RF in RA. This approach should be considered in future studies of RF.</p>","PeriodicalId":21267,"journal":{"name":"Rheumatology and Therapy","volume":" ","pages":"123-136"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751350/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142855515","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Safety and Effectiveness of Canakinumab in Patients with MKD/HIDS: Interim Analysis of the RELIANCE Registry.","authors":"Prasad T Oommen, Tilmann Kallinich, Juergen Rech, Norbert Blank, Julia Weber-Arden, Jasmin B Kuemmerle-Deschner","doi":"10.1007/s40744-024-00733-7","DOIUrl":"10.1007/s40744-024-00733-7","url":null,"abstract":"<p><strong>Introduction: </strong>Interim analysis of the long-term safety and effectiveness of canakinumab, at a patient level, in the mevalonate kinase deficiency/hyperimmunoglobulin-D syndrome (MKD/HIDS) cohort of the RELIANCE registry.</p><p><strong>Methods: </strong>From June 2018, the RELIANCE registry enrolled paediatric (aged ≥ 2 years) and adult patients (aged ≥ 18 years) with MKD/HIDS who were receiving canakinumab as part of their routine medical care. Safety, physician- and patient-reported measures of disease activity and dosing patterns were evaluated at baseline and every 6 months until end-of-study visit.</p><p><strong>Results: </strong>At the analysis cut-off date of December 2022, eight patients with MKD/HIDS were enrolled. Five (62.5%) were children (< 18 years) and five (62.5%) were female. The median patient age was 8.0 (range 2.0-39.0) years, and all patients were pre-treated with canakinumab prior to enrolment (median duration of canakinumab treatment: 3.8 years). Canakinumab was well tolerated, with seven (87.5%) patients reporting 48 adverse events (incidence rate/100 patient years: 218.1). No serious adverse drug reactions were reported. Patients continued to receive vaccinations during long-term treatment with canakinumab. Disease activity, evaluated by physician-reported (physician's global assessment, disease remission, C-reactive protein, serum amyloid A, erythrocyte sedimentation rate) and patient-reported (autoinflammatory disease activity index diary, disease activity, fatigue, impact on social life) measures, was generally well controlled throughout the study. Over 50.0% of patients maintained disease remission from baseline to month 24, and medians of all inflammatory markers remained within normal limits throughout the study. Most patients received higher than the recommended starting dose of canakinumab throughout the study.</p><p><strong>Conclusion: </strong>Data from this interim analysis of a unique registry of patients with a rare disease support the long-term safety and effectiveness of the IL-1-blocking agent canakinumab for the treatment of MKD/HIDS.</p>","PeriodicalId":21267,"journal":{"name":"Rheumatology and Therapy","volume":" ","pages":"137-155"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751260/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142897058","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Persistence and Effectiveness of Upadacitinib versus Other Janus Kinase Inhibitors and Tumor Necrosis Factor Inhibitors in Australian Patients with Rheumatoid Arthritis.","authors":"Peter Youssef, Sabina Ciciriello, Talib Tahir, Joanna Leadbetter, Belinda Butcher, Miriam Calao, Nicole Walsh, Catherine O'Sullivan, Tegan Smith, Geoffrey Littlejohn","doi":"10.1007/s40744-024-00736-4","DOIUrl":"10.1007/s40744-024-00736-4","url":null,"abstract":"<p><strong>Introduction: </strong>This study sought to describe treatment patterns, persistence, and effectiveness of upadacitinib (UPA) alone and compared to other Janus kinase inhibitors (JAKis) or tumor necrosis factor inhibitors (TNFis) in patients with rheumatoid arthritis (RA).</p><p><strong>Methods: </strong>This retrospective, non-interventional study used the OPAL dataset, derived from electronic medical records. Patients initiated UPA (N = 2624), other JAKis (baricitinib and tofacitinib [N = 925]), or TNFis (adalimumab, etanercept, certolizumab, golimumab, infliximab [N = 3540]) between May 2020 and March 2023. Median persistence (Kaplan-Meier) and effectiveness (Disease Activity Score 28-joint C-reactive protein, three variables [DAS28CRP{3}]) were evaluated for UPA-treated patients and in three propensity score-matched cohorts: UPA monotherapy versus combination therapy, UPA versus other JAKis, and UPA versus TNFis.</p><p><strong>Results: </strong>In patients prescribed UPA, 41.3% were ≥ 65 years old, 33.8% were prescribed as first-line advanced therapy, and 27.2% were prescribed monotherapy. Persistence on UPA was 26.6 months (95% confidence intervals: 24.4, 29.9) and longest in earlier lines of therapy. The DAS28CRP(3) remission rate was 73% at 3 months, with improvements observed across lines of therapy. UPA monotherapy and combination therapy had similar persistence (27.8 [23.5, 33.4] versus 30.4 months [22.1, 35.3], p = 0.84) and effectiveness. UPA showed longer persistence than other JAKis (28.8 [25.6, 32.4] versus 17.2 months [14.9, 19.8], p < 0.001) and TNFis (26.6 [24.9, 30.8] versus 13.3 months [11.5, 14.5], p < 0.001). DAS28CRP(3) remission rates were greater at 3 months for UPA than other JAKis (75.0% versus 61.5%) and TNFis (72.7% versus 59.5%). In unmatched subgroups, compared to cycling between TNFis, switching to UPA from other JAKis or TNFis resulted in longer persistence (JAKi-to-UPA: 25.3 [16.1, not reached]; TNFi-to-UPA: 27.8 [23.2, 35.4]; TNFi-to-TNFi: 9.6 [8.4, 10.7]) and greater DAS28CRP(3) remission rates over 9 months.</p><p><strong>Conclusions: </strong>Overall, the breadth and depth of data from this large real-world dataset continue to support a favorable clinical profile of UPA for the treatment of RA and may inform treatment choices in everyday clinical practice.</p>","PeriodicalId":21267,"journal":{"name":"Rheumatology and Therapy","volume":" ","pages":"173-202"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751354/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142932618","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}