BMC Rheumatology最新文献

Background: Considerable knowledge gaps remain regarding the cause, prevention, and management of rheumatoid arthritis (RA), with limited systematic effort to establish research priorities that truly align with the preferences of those impacted by RA.

Objectives: To identify and prioritize unanswered questions about RA, capturing insights from people living with RA and healthcare professionals involved it its management.

Methods: A James Lind Alliance (JLA) Priority Setting Partnership was established and followed six steps: (1) form a steering group, (2) define scope, (3) collect evidence uncertainties using focus groups, (4) collate evidence uncertainties and verify by checking existing research, (5) shortlist evidence uncertainties in an online survey, and (6) identify the top 10 research priorities through a priority-setting workshop.

Results: A total of 212 questions were generated from three focus group interviews and distilled into 36 questions for a survey distributed to people with RA and healthcare professionals. Among 554 responses (mean age 58 [SD 13] years; 481 [87%] women), 449 (81%) identified as people with RA, and 105 (19%) as healthcare professionals, with some reporting dual roles. The ranking process resulted in a shortlist of 26 questions, which were further narrowed down to the top 10 research priorities. The top 10 research priorities addressed strategies for RA prevention, rapid diagnosis, identifying effective treatments, reducing treatment side effects, and holistic management approaches.

Conclusion: Adopting the JLA method, this study mapped out core research priorities in RA, offering valuable insights that can help researchers, policymakers, and funders align future RA research with patient and clinical needs.

Clinical trial number: Not applicable.

Background: Biologic and targeted synthetic disease-modifying antirheumatic drugs (b/tsDMARDs) have improved outcomes in rheumatoid arthritis (RA). However, heterogeneity in treatment response remains a significant challenge. Machine learning (ML) may enable improved prediction, but the comprehensive review of ML applications in RA is fragmented and limited. This scoping review synthesizes the literature on ML methods for predicting treatment response to b/tsDMARDs in RA.

Methods: Following the Preferred Reporting Items for Systematic Reviews and Meta-Analysis Extension for Scoping Reviews (PRISMA-ScR) guidelines, we systematically searched PubMed, MEDLINE, and Embase (from databases' inception through March 2024). Using the Covidence online platform, two reviewers independently screened titles, abstracts, and full texts for eligibility. Studies were included if they applied ML methods in predicting treatment response to b/tsDMARD in RA. We provided a qualitative synthesis of databases used, study design, population, outcomes, predictors, and model validation. Risk of bias was assessed using Quality in Prognosis Studies (QUIPS), and reporting quality was evaluated using TRIPOD guidelines.

Results: Of 294 citations reviewed, 24 studies met the inclusion criteria. Most used real-world data from registries (N = 12, 50%), followed by electronic health records (N = 4, 17%). Study sample sizes ranged from 39 to 7,300 (Median = 494). ML models-especially boosted trees, random forests, support vector machines, and regularized regression-were most frequently applied. Study outcomes included remission, low disease activity, and treatment non-response. Common baseline predictors were disease activity, biomarkers, functional status, and patient-reported measures. AUCs ranged from 0.54 to 0.92 (Mean = 0.71), with boosted trees and neural networks often performing best. External validation was rare (N = 7, 17.5%), and most studies showed a low-to-moderate risk of bias (N = 32, 80%).

Conclusion: ML methods are increasingly used to predict RA treatment response, but vary widely in methodology and performance. Standardization, external validation, and transparent reporting are critical for advancing clinical application.

Clinical trial number: Not Applicable (NA).

Background: We estimated the frequency of catastrophic healthcare expenditure(CHE), and their determinants in Indian patients with systemic lupus erythematosus(SLE).

Methods: This was a cross-sectional, questionnaire-based survey conducted by the Lupus Special Interest Group of the Indian Rheumatology Association across 14 centers. Patients with SLE diagnosed as per SLICC-2012 on follow-up for at least 1-year were interviewed regarding annual disease-related expenditures including direct (medical and non-medical) and indirect costs. CHE was defined as > 20% of the annual income. Results are presented in Indian currency (INR), wherein 100 INR = 1.19 USD = 1.1 EURO.

Results: We included 655 patients with SLE [92.7% women], with a mean age of 32.9 ± 11.6 years. The median direct annual expenditure was INR 52400(30810,96300), largest component being cost of medications [INR 24000(12000,40000) and hospitalizations [INR 35000(14400,90000)] One-third of patients(n = 237,36.2%) suffered CHE; they were older [AOR1.01(0.99,1.03)], had lower level of education [AOR1.95(1.01,3.81)], belonged to lower socio-economic-strata[AOR 9.63(5.66,16.4)], had renal and/or neuropsychiatric lupus [AOR1.42(0.99,2.06)] and higher damage(SDI) [AOR1.84(1.22,2.77)]. The median annual indirect cost was INR16416(5016,52896). Three-fourths(73.7%) of the participants incurred out-of-pocket expenses for their healthcare. The employed population was low(n = 187;28.3%), and the absenteeism rate was 24%.

Conclusion: Hospitalization and medication costs are major factors driving exponentially high out-of-pocket expenses, resulting in CHE in one-third of patients with SLE in India.

Clinical trial number: Not applicable.

Background: This study evaluated the disease phenotype and treatment of axial psoriatic arthritis among patients from Japan compared with those from different geographic regions.

Methods: Data from the ASAS-PerSpA study were analyzed. Patients with psoriatic arthritis (PsA) with axial involvement, according to a rheumatologist´s judgment, were included. Patients were further categorized by four geographic regions: Europe/North America, Latin America, Middle East/North Africa, and Asia, split into Japan and other Asian countries. Disease and patient characteristics, disease activity, function, and treatment were compared by region.

Results: Of the 4,465 patients with SpA, 1,033 (23%) were diagnosed with PsA by their rheumatologist. Among those with PsA, 367 (35.5%) had axial involvement (axPsA). Disease activity and function ranges were 4.1-5.4 for BASDAI, 2.5-3.2 for ASDAS, and 3.0-4.7 for BASFI, by regions. In Japan, disease activity and function were relatively lower, indicated by a mean BASDAI of 3.5 (SD 2.4), ASDAS of 2.2 (SD 1.0), and BASFI of 1.6 (SD 2.3). These indexes were also significantly lower than those in other Asian countries, with scores of 4.8 (SD 3.0), 2.2 (SD 2.4), and 3.2 (SD 1.5) respectively. All regions showed variations in the use of csDMARDs and bDMARDs, the utilization rate of csDMARDs was significantly lower in Japan than in other Asian countries (51.4% vs. 78.1%, p = 0.02).

Conclusion: Patients with axPsA in Japan showed relatively lower disease activity and function than those from different geographic regions, especially in other Asian countries with less frequent csDMARD use.

Background: Granulomatosis with polyangiitis (GPA) is a rare form of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis that can involve multiple organ systems, including the gastrointestinal (GI) tract. Although GI manifestations are relatively uncommon, they may be associated with serious complications and adverse outcomes. This study aimed to assess the frequency and types of GI involvement in patients with GPA and to examine their relationship with disease severity, prognosis, and treatment response.

Methods: In this retrospective cohort study, clinical records of 220 patients with a confirmed diagnosis of GPA who were referred to Amir Alam Hospital between 2013 and 2021 were reviewed. Data on demographic characteristics, GI symptoms, Birmingham Vasculitis Activity Score (BVAS), therapeutic response, relapse rates, and mortality were collected and analyzed.

Results: GI involvement was observed in 18 patients (8.2%). The most common manifestations included hepatitis, mesenteric ischemia, diarrhea, pancreatitis, and elevated liver enzymes. Patients with GI involvement had significantly higher BVAS scores (mean 21 vs. 15.8, p = 0.004), a markedly increased mortality risk (hazard ratio = 3.24, p < 0.001), and a shorter time to first relapse (mean 5.2 vs. 10.3 months, p = 0.041) compared to those without GI symptoms.

Conclusion: Gastrointestinal involvement in GPA is associated with more severe disease activity, diminished treatment response, and increased mortality. Early detection and appropriate management of GI manifestations may improve clinical outcomes. Further prospective studies are warranted to elucidate the underlying mechanisms and optimize treatment strategies for this high-risk subgroup.

Background: SLE is a chronic autoimmune illness that affects several systems. Gastrointestinal abnormalities, although uncommon, can be dangerous and frequently go undiagnosed due to vague symptoms. Lupus enteritis and peritonitis are serious consequences. Lupus enteritis affects up to 9.7% of SLE patients and causes stomach discomfort, nausea, and diarrhea. It frequently involves intestinal vasculitis, which results in gut wall thickening and, in extreme instances, ischemia or perforation. Lupus peritonitis is very uncommon. Differentiating them from other causes of stomach discomfort is critical. This case emphasizes the diagnostic problems, imaging results, and the significance of early, focused therapy for better outcomes.

Case presentation: A 38-year-old lady from Hebron, Palestine, reported 3 days of increasing, colicky abdominal discomfort, bloody vomiting, and black feces. She had a three-year history of migrating polyarthralgia, photosensitivity, alopecia, and two first-trimester losses. Her cousins' family history indicated rheumatoid arthritis and hypothyroidism. Upon examination, she seemed pale and unwell, with stomach discomfort and right basal lung crepitations. Laboratory results revealed microcytic anemia, lymphopenia, hypokalemia, hypophosphatemia, increased ESR and CRP, and a positive Coombs test. High levels of ANA, anti-dsDNA, and anti-Sm antibodies proved systemic lupus erythematosus (SLE). The imaging indicated a pulmonary embolism, pleural effusion, and thickening of the jejunal wall. Methylprednisolone and anticoagulant treatment were started. Gastroscopy indicated hemorrhagic gastritis, and paracentesis revealed exudative ascitic fluid due to ascites worsening. The patient was given cyclophosphamide with mesna, which showed improvement. The patient improved on treatment with prednisolone, hydroxychloroquine, and a proton pump inhibitor after discharge. Follow-up showed continued recovery without recurrence of symptoms.

Conclusion: Systemic lupus erythematosus (SLE) is an autoimmune illness that causes uncommon gastrointestinal symptoms such as lupus enteritis and peritonitis. This case demonstrates their simultaneous occurrence, underscoring the need to include SLE in the workup for gastrointestinal symptoms. Proper diagnosis by laboratories, imaging, and gastroscopy is critical since early intervention dramatically improves patient outcomes.

Clinical trial number: Not applicable.