Modern Rheumatology最新文献

Objectives: This subgroup analysis of the MITIGATE study aimed to evaluate whether the efficacy and safety of inebilizumab in Japanese patients with IgG4-related disease are consistent with those observed in the overall trial population.

Methods: MITIGATE was a randomized, double-blind, placebo-controlled, phase 3 study conducted in 22 countries, and this subgroup analysis included Japanese patients enrolled in that trial.

Results: In total, 27 Japanese patients (inebilizumab, N = 20; placebo, N = 7) were included in this subgroup analysis. The organs most frequently affected were the submandibular gland (66.7%), lacrimal gland (59.3%), and pancreas (51.9%). Despite the discontinuation of glucocorticoids within 8 weeks of study initiation, there were no adjudication committee-determined flares requiring treatment in the inebilizumab group. Flares were observed in four patients (57.1%) in the placebo group, with a median time to first flare of 246.0 days. Inebilizumab reduced annualized flare rate, increased flare-free complete remission, and lowered glucocorticoid use. Safety profiles were similar between treatment groups; most treatment-emergent adverse events were non-serious.

Conclusions: Inebilizumab reduced flare risk and glucocorticoid exposure and was well tolerated in Japanese patients with IgG4-related disease, consistent with overall MITIGATE results.

Trial registration: NCT04540497.

Objective: To assess pregnancy and neonatal outcomes in women with rheumatoid arthritis treated with an etanercept biosimilar, and provide real-world safety evidence.

Methods: This retrospective, single-centre study included 11 pregnant women with rheumatoid arthritis who received an etanercept biosimilar at the National Center for Child Health and Development (Tokyo) between October 2018 and March 2022. The clinical characteristics, disease activity, medications, delivery outcomes, and neonatal health were reviewed.

Results: All 11 pregnancies were live births. The median maternal age was 34 years, the gestational age at delivery was 39.7 weeks, and the birth weight was 2,929 g. One preterm birth (9.1%) and two caesarean deliveries (18.2%) occurred. No cases of low birth weight or neonatal intensive care unit admission were reported. One case of patent foramen ovale was noted. All neonates had 5-minute Apgar scores ≥ 9. The etanercept biosimilar was discontinued before delivery in four cases to allow infant vaccination.

Conclusion: Use of the etanercept biosimilar during pregnancy was associated with favourable outcomes, supporting its safety and utility in managing rheumatoid arthritis during pregnancy.

Objectives: This study aimed to evaluate the knowledge, experience, and challenges faced by care and welfare professionals in supporting patients with rheumatoid arthritis (RA) in Japan, and to identify issues related to coordination between medical and welfare services.

Methods: A nationwide questionnaire survey was conducted using stratified regional sampling. The target population included 1000 members each from the Japan Care Manager Association (JCMA) and the Japanese Association of Certified Social Workers (JASW). The survey assessed respondents' knowledge of RA, support experience, actual practices, views on interdisciplinary collaboration, and perceptions of patients' understanding of available systems.

Results: Valid responses were obtained from 390 JCMA members and 330 JASW members. While basic knowledge of RA symptoms and disease course was common, awareness of RA-specific medications was limited, especially among JASW respondents. Many participants had little experience supporting RA patients. The reported challenges included insufficient information from physicians, a lack of structured information sharing, and underestimation of care needs in long-term care certification. Patients and families also showed limited understanding of healthcare and welfare systems.

Conclusions: The findings highlight the need for improved education, better interdisciplinary coordination, and enhanced information sharing. Comprehensive efforts are essential to ensure continuous, high-quality care for RA patients in community settings.

Objectives: The present study aimed to investigate the relationship between locomotive syndrome (LS) and physical function in rheumatoid arthritis (RA) patients.

Methods: A total of 324 patients enrolled in a prospective cohort study on frailty in RA patients conducted between 2021 and 2022 were analysed. The 25-question Geriatric Locomotive Function Scale was used to assess LS, defined as stage ≥2 (0: < 7 points; 1: 7-15 points; 2: 16-23 points; 3: ≥24 points). Analysis of variance was used for group comparisons, and receiver operating characteristic analysis for identifying LS-related cut-off values.

Results: Mean age was 63 ± 11 years, mean disease duration was 13.1 ± 9.9 years, and 87% of patients were female. The proportion of patients with LS was 38%. Walking speed (odds ratio [OR]: 0.01, 95% confidence interval [CI]: 0.02-0.07), 2-step test (OR: 0.06, 95% CI: 0.01-0.40), and 5 Times Sit-to-Stand (5TSS) test (OR: 1.18, 95% CI: 1.08-1.29) were associated with LS. Cut-off values were 1.21 m/sec for walking speed, 1.2 for 2-step test score, and 10.7 seconds for the 5TSS test.

Conclusions: This study clarified the relationship between LS progression and lower limb function in RA patients, and identified cut-off values for LS.

Objectives: To examine the real-world effectiveness and safety of peficitinib (PEF) and abatacept (ABT) in patients with rheumatoid arthritis (RA).

Methods: In this multicentre, retrospective cohort study, we examined patients who were administered PEF or ABT between July 2019 and July 2022. The primary endpoint was the 1-year retention rate of PEF or ABT. The primary analyses were performed using treatment persistence, disease activity measurements, and safety parameters. The inverse probability of treatment weighting (IPTW) was used to adjust for confounding variables.

Results: A total of 219 patients were enrolled, with 64 receiving PEF and 155 receiving ABT. The 1-year retention rates were 42.8% in the PEF group and 61.0% in the ABT group after IPTW (P = .0083). Two years following the initiation of treatment, the composite disease activity measures showed comparable improvement in both groups after IPTW. Treatment discontinuation occurred primarily because of an inadequate therapeutic response (PEF: 41.3%, ABT: 28.1%) and adverse reactions (PEF: 11.1%, ABT: 3.8%).

Conclusions: PEF treatment resulted in lower retention rates compared with ABT, whereas both medications demonstrated effectiveness in controlling disease activity for patients who were able to continue treatment. Our findings contribute to informed decision-making in RA treatment in actual clinical practice.

Objectives: VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is an autoinflammatory disease caused by somatic variants of the UBA1 gene. Due to uncertainty around treatment strategies and limited therapeutic options, this study aimed to characterise the clinical treatment and outcomes of VEXAS syndrome patients in Japan.

Methods: We retrospectively analysed data on clinical manifestations, treatments, and outcomes from 46 male patients diagnosed with VEXAS syndrome. Twelve patients at our institution were evaluated for remission using the French VEXAS group criteria.

Results: All patients (median age at onset: 71.4 years) received systemic glucocorticoid therapy (mean maximum dose: 47.4 mg/day; mean minimum dose: 8.5 mg/day). Most patients required continuous glucocorticoid treatment of ≥10 mg/day, and tapering was generally difficult. Among the patients followed at our institution, only 42% (5/12) achieved complete remission at least once during follow-up. Tocilizumab was the most frequently administered immunosuppressant (n = 24, 52.2%); Janus kinase inhibitors and azacitidine, reported to be effective overseas, were prescribed in only three and two cases, respectively.

Conclusions: This study highlights the difficulty of treating patients with VEXAS syndrome in Japan. The development of more effective treatments is urgently needed to reduce glucocorticoid dependence and improve patient outcomes.

Objectives: This study aimed to evaluate the diagnostic performance of the 2019 European League Against Rheumatism/American College of Rheumatology (EULAR/ACR-2019) classification criteria of systemic lupus erythematosus (SLE) and to clarify the clinical characteristics of Japanese childhood-onset SLE (cSLE).

Methods: We retrospectively analyzed clinical data registered in the Paediatric Rheumatology International Collaboration Unit Registry (PRICURE) version 2 up to March 31, 2023. Frequencies of individual items within the EULAR/ACR-2019 criteria were compared with those observed in a Japanese adult SLE cohort.

Results: A total of 105 patients with cSLE, 19 with Juvenile dermatomyositis (JDM), 27 with primary Sjögren's disease (pSjD), and 9 with mixed connective-tissue disease (MCTD) were included. The sensitivity of the EULAR/ACR-2019 criteria was 97.1%. The specificity was 94.7% for JDM, 92.6% for pSjD, 55.6% for MCTD, and 87.3% for all disease controls. cSLE patients in this cohort more frequently exhibited renal involvement, low serum C3 or C4 levels, and positivity for antiphospholipid and anti-double-stranded DNA antibodies, but joint symptoms were less common than in adult SLE patients.

Conclusions: Although the EULAR/ACR-2019 criteria are generally applicable, the limited specificity for MCTD necessitates careful differential diagnosis. Japanese cSLE is commonly characterized by renal involvement, hypocomplementemia, and SLE-related autoantibody positivity.

Objectives: Evaluate the long-term safety and effectiveness of mepolizumab in subgroups of patients with eosinophilic granulomatosis with polyangiitis, including: disease duration, oral corticosteroid (OCS) use, relapse, worsening or new-onset symptoms, anti-neutrophil cytoplasm antibodies (ANCA) status, and immunosuppressive therapy use.

Methods: Data from the 96-week MARS study in Japan were analysed for patients receiving mepolizumab for ≥96 weeks, focusing on adverse events (AEs), clinical symptoms, average daily OCS dose, and relapse.

Results: OCS exposure decreased in most subgroups, particularly in the ANCA negative subgroup. Patients on higher average OCS doses (>7.5 mg/day vs ≤7.5 mg/day, during the observation period) experienced more AEs, including infection-related AEs and clinical symptoms, and higher relapse rates. No relapses were observed in patients on an average dose of 0.0 mg/day OCSs. Reduced OCS use was associated with improved clinical outcomes, particularly in patients who remained relapse-free.

Conclusions: Mepolizumab was effective regardless of eosinophilic granulomatosis with polyangiitis duration, ANCA status, or immunosuppressant use. Higher OCS doses were linked to more AEs, clinical symptoms, and relapses compared to lower doses. In relapse-free patients, continued mepolizumab use led to fewer AEs, lower OCS doses, and improved symptoms.

Objective: This study aimed to evaluate treatment outcomes, complications, and the clinical course of thrombocytopenia in patients with TAFRO syndrome.

Methods: We conducted a retrospective analysis of patients treated at Nara Medical University Hospital from 2015 to 2023. Diagnosis of TAFRO syndrome was made according to the 2015 or 2019 criteria, and disease severity was assessed based on the updated disease severity classification. Clinical characteristics and laboratory and histological data were analysed.

Results: Seven patients were included, with four classified as very severe, two as severe, and one as slightly severe. All the patients received early combination therapy (various combinations of glucocorticoids, tocilizumab, cyclosporine, and rituximab), with the second therapy added a median of 5 days after the first. Six patients received eltrombopag. Despite intensive treatment, thrombocytopenia persisted for a prolonged period, with a median time to platelet count normalization of 54 days. Cytomegalovirus reactivation occurred in six patients during treatment but resolved with appropriate antiviral therapy. All the patients achieved remission and survived.

Conclusions: Thrombocytopenia in TAFRO syndrome persists long term despite intensive combination therapy. Early combination therapy was associated with excellent survival outcomes, suggesting that intensifying treatment solely for thrombocytopenia may not be necessary given its prolonged course.