BMC Rheumatology最新文献

Background: Supporting adherence to medication is an essential part of the treatment and care of patients with rheumatic and musculoskeletal diseases. The Compliance Questionnaire Rheumatology (CQR) measures adherence in rheumatic diseases through 19 items covering drug-taking behaviour to identify the reasons for adhering to treatment and the factors that contribute to suboptimal adherence. The objective of this study was to present the translation of the CQR into Danish and the face validity and reliability test.

Methods: The CQR was translated into Danish according to international guidelines, followed by a face validity test among 10 patients with rheumatoid arthritis in 2009. The test-retest reliability of the Danish CQR was evaluated in 49 patients with rheumatoid arthritis in 2020 - 2021 using the standard error of the measurement (SEM) converted into the minimally detectable change (MDC) and the intraclass correlation coefficient (ICC). Questionnaires were administered with a minimum of 10 days between assessments.

Results: The participants in the reliability test had a mean age of 57.4 years (SD 16.1) and a mean disease duration of 1.13 years (range 2 months-2 years). The mean CQR score in the test and retest was 62.7 (confidence interval (CI) 58.8; 66.6) and 62.5 (CI 58.9; 66.1), respectively, with a SEM of 8.59 (7.16; 10.73) and an MDC of 16.83. A satisfactory test-retest reliability was confirmed by an ICC value of 0.79 (CI 0.68; 0.89).

Conclusion: The Danish CQR has satisfactory test-retest reliability in patients newly diagnosed with rheumatoid arthritis and is considered a reliable tool to measure adherence in this group.

Background: To determine the safety and efficacy of biological agents used in the treatment of systemic lupus erythematosus (SLE) in adults.

Methods: Systematic review and meta-analysis following PRISMA guidelines.

Data sources: MEDLINE (through Pubmed), EMBASE, Cochrane library, Clinicaltrials.gov, Australianclinicaltrials.gov.au, ANZCTR.org.au and WHO International Clinical Trials Registry Platform for studies published from 20 May 2021 and 15 years prior. A grey literature search was performed and completed on 31 May 2021.

Study criteria: Phase II, III or quasi randomised controlled trials, studies with only cerebral or cutaneous lupus were excluded.

Data extraction: Two authors independently screened studies for eligibility, extracted, reviewed data for accuracy, and used the Cochrane tool to assess risk of bias.

Results: Forty-four studies were identified, consisting of 15 groups of drugs and 25 different biological agents, totalling 16,889 patients. The main outcomes assessed included Systemic Lupus Erythematosus Responder Index (SRI), BILAG-Based Composite Lupus Assessment (BICLA) and combined combined/partial renal remission (CRR/PRR). Four groups of biologics were found to improve outcomes. Anti-interferons: Anifrolumab increased BICLA response and SRI 5 to 8, decreased prednisone dosages, with increased herpes zoster infections, but fewer serious adverse events. Sifalimumab improved SRI but also increased herpes zoster infections. Anti BAFF/BLyS and/or APRIL: Belimumab consistently improved SRI 4, decreased prednisone dosages, increased combined CRR/PRR, and had no adverse safety outcomes. Tabalumab increased SRI 5 at 52 weeks with no steroid sparing effect but was associated with increased infusion related adverse events. Telitacicept improved SRI 4 at 52 weeks, with no increased adverse events, though data was rather sparse. Anti CD-20 monoclonal antibody, Obinutuzumab increased combined CRR/PRR at 1 and 2 years. Anti IL12/23 monoclonal antibody, Ustekinumab, increased SRI 4 to 6, but not BICLA at 24 weeks, with no concerning safety outcomes.

Conclusion: Multiple biologic agents are shown in high quality studies to have a significant therapeutic impact on outcomes in SLE.

Background: While the integration of patient and public involvement (PPI) in clinical research is now widespread and recommended as standard practice, meaningful PPI in pre-clinical, discovery science research is more difficult to achieve. One potential way to address this is by integrating PPI into the training programmes of discovery science postgraduate doctoral students. This paper describes the development and formative evaluation of the Student Patient Alliance (SPA), a programme developed at the University of Birmingham that connects PPI partners with doctoral students.

Methods: Following a successful pilot of the SPA by the Rheumatology Research Group at the University of Birmingham, the scheme was implemented across several collaborating Versus Arthritis / Medical Research Council (MRC) centres of excellence. Doctoral students were partnered with PPI partners, provided with initial information and guidance, and then encouraged to work together on research and public engagement activities. After six months, students, their PPI partners and the PPI coordinators at each centre completed brief surveys about their participation in the SPA.

Results: Both doctoral students and their PPI partners felt that taking part in SPA had a positive impact on understanding, motivation and communication skills. Students reported an increased understanding of PPI and patient priorities and reported improved public engagement skills. Their PPI partners reported a positive impact of the collaboration with the students. They enjoyed learning about the student's research and contributing to the student's personal development. PPI coordinators also highlighted the benefits of the SPA, but noted some challenges they had experienced, such as difficulties matching students with PPI partners.

Conclusions: The SPA was valued by students and PPI partners, and it is likely that initiatives of this kind would enhance students' PPI and public engagement skills and awareness of patients' experiences on a wider scale. However, appropriate resources are needed at an institutional level to support the implementation of effective programmes of this kind on a larger scale.

Background: There is increasing research interest in the development of preventive treatment for individuals at risk of rheumatoid arthritis (RA). Previous studies have explored the perceptions of at-risk groups and patients about predictive and preventive strategies for RA, but little is known about health care professionals' (HCPs) perspectives.

Methods: One-to-one semi-structured qualitative interviews were conducted (face-to-face or by telephone) with HCPs. Audio recordings of the interviews were transcribed, and the data were analysed by thematic analysis.

Results: Nineteen HCPs (11 female) were interviewed, including ten GPs, six rheumatologists and three rheumatology nurse specialists. The thematic analysis identified four organising themes: 1) Attributes of predictive and preventive approaches; 2) Ethical and psychological concerns; 3) Implementation issues and 4) Learning from management of other conditions. Theme 1 described necessary attributes of predictive and preventive approaches, including the type and performance of predictive tools, the need for a sound evidence base and consideration of risks and benefits associated with preventive treatment. Theme 2 described the ethical and psycho-social concerns that interviewees raised, including the potential negative economic, financial and psychological effects of risk disclosure for 'at-risk' individuals, uncertainty around the development of RA and the potential for benefit associated with the treatments being considered. Theme 3 describes the implementation issues considered, including knowledge and training needs, costs and resource implications of implementing predictive and preventive approaches, the role of different types of HCPs, guidelines and tools needed, and patient characteristics relating to the appropriateness of preventive treatments. Theme 4 describes lessons that could be learned from interviewees' experiences of prediction and prevention in other disease areas, including how preventive treatment is prescribed, existing guidelines and tools for other diseases and issues relating to risk communication.

Conclusions: For successful implementation of predictive and preventative approaches in RA, HCPs need appropriate training about use and interpretation of predictive tools, communication of results to at-risk individuals, and options for intervention. Evidence of cost-efficiency, appropriate resource allocation, adaptation of official guidelines and careful consideration of the at-risk individuals' psycho-social needs are also needed.

Introduction: Clinically significant pericardial effusions and cardiac tamponade in systemic sclerosis (SSc) patients is uncommon and the factors that contribute to progression of pericardial involvement in SSc patients have not been well established.

Methods: A review of the national inpatient sample database was performed looking SSc related hospitalizations between 2002 and 2019. Data was collected on patients with pericardial effusions and cardiac tamponade and analyzed to identify and describe patient characteristics and comorbidities.

Results: Out of a total of 523,410 SSc hospitalizations, with an overall inpatient mortality rate of 4.7% (24,764 patients), pericardial effusion was identified in 3.1% of all hospitalizations (16,141 patients) out of which 0.2% (838 patients) had a diagnosis of cardiac tamponade. Patients with pericardial effusion were significantly more likely to have pulmonary circulatory disease (p = < 0.0001), congestive heart failure (p = < 0.0001) end stage renal disease (p = < 0.0001), diabetes (p = 0.015), and hypothyroidism (p = 0.025). Patients with cardiac tamponade were significantly more likely to have a history of coronary artery bypass graft surgery (p = 0.001) or atrial fibrillation (p = < 0.0001). Hospitalized patients with cardiac tamponade had a significantly increased mortality rate of 17.7% compared to 8.8% in patients with pericardial effusions without a tamponade physiology, with an odds ratio of 2.3 (1.97-2.86), p = < 0.0001.

Conclusion: Pericardial effusion and tamponade are associated with increased morbidity and mortality in SSc patients. Further studies are required to explore the role of patient comorbidities and characteristics in development into pericardial effusions or tamponade.

Objectives: Rheumatic and musculoskeletal diseases (RMDs) require a tailored follow-up that can be enhanced by the implementation of innovative tools. The Digireuma study aimed to test the feasibility of a hybrid follow-up utilizing an electronic patient reported outcomes (ePROs)-based monitoring strategy in patients with RMDs.

Methods: Adult patients with rheumatoid arthritis (RA) and spondyloarthritis (SpA) were recruited for a 6-month bicentric prospective follow-up consisting of face-to-face and digital assessments. Patients were asked to report disease-specific ePROs on a pre-established basis, and could also report flares, medication changes, and recent infections at any time. Four rheumatologists monitored these outcomes and contacted patients for interventions when deemed necessary. Results from face-to-face and digital assessments were described.

Results: Of 56 recruited patients, 47 (84%) submitted any ePROs to the digital platform. Most patients with RA were female (74%, median age of 47 years), while 48% of patients with SpA were female (median age 40.4 years). A total of 3,800 platform visits were completed, with a median of 57 and 29 visits in patients with RA and SpA, respectively. Among 52 reported alerts, 47 (90%) needed contact, of which 36 (77%) were managed remotely. Adherence rates declined throughout the study, with around half of patients dropping out during the 6 months follow-up.

Conclusion: The implementation of a hybrid follow-up in clinical practice is feasible. Digital health solutions can provide granular knowledge of disease evolution and enable more informed clinical decision making, leading to improved patient outcomes. Further research is needed to identify target patient populations and engagement strategies.

Background: Traditionally rheumatoid arthritis (RA) trials classify patients as responders and non-responders; they ignore the potential range of treatment responses. Group Based Trajectory Models (GBTMs) provide a more refined approach. They identify patient subgroups with similar outcome trajectories. We used GBTMs to classify patients into subgroups of varying responses and explore factors associated with different responses to intensive treatment in a secondary analysis of intensive treatment in the TITRATE clinical trial.

Methods: The TITRATE trial enrolled 335 patients with RA: 168 patients were randomised to receive intensive management, which comprised monthly assessments including measures of the disease activity score for 28 joints (DAS28), treatment escalation when patients were not responding sufficiently and psychosocial support; 163 of these patients completed the trial. We applied GBTMs to monthly DAS28 scores over one year to these patients who had received intensive management. The control group had standard care and were assessed every 6 months; they had too few DAS28 scores for applying GBTMs.

Results: GBTMs identified three distinct trajectories in the patients receiving intensive management: good (n = 40), moderate (n = 76) and poor (n = 47) responders. Baseline body mass index (BMI), disability, fatigue and depression levels were significantly different between trajectory groups. Few (10%) good responders were obese, compared to 38% of moderate, and 43% of poor responders (P = 0.002). Few (8%) good responders had depression, compared to 14% moderate responders, and 38% poor responders (P < 0.001). The key difference in treatments was using high-cost biologics, used in only 5% of good responders but 30% of moderate and 51% of poor responders (P < 0.001). Most good responders had endpoint remissions and low disability, pain, and fatigue scores; few poor responders achieved any favourable outcomes.

Conclusion: GBTMs identified three trajectories of disease activity progression in patients receiving intensive management for moderately active RA. Baseline variables like obesity and depression predicted different treatment responses. Few good responders needed biologic drugs; they responded to conventional DMARDs alone. GBTMs have the potential to facilitate precision medicine enabling patient-oriented treatment strategies based on key characteristics.

Registration: TITRATE Trial ISRCTN 70160382.

Background: Although reduced work ability is a substantial problem among people with inflammatory arthritis (IA), work ability is an underexposed area in clinical practice. Evidence on vocational interventions in IA is limited, but favourable results of delivery by a physiotherapist (PT) warrant the need for further research. Therefore, we aim to evaluate the (cost-)effectiveness of a multimodal, PT-led, vocational intervention in (self-)employed people with IA compared to usual care.

Methods: This randomized controlled trial will include 140 people with rheumatoid arthritis (RA) or axial spondyloarthritis (axSpA) who are (self-)employed and have reduced work ability (Work Ability Index - Single Item Scale (WAS) ≤ 7/10) and/or RA/axSpA related sick leave (≤ 6 months). Participants will be randomized 1:1 to the intervention or control condition (usual care). The intervention, delivered by primary care PTs, will be personalized to each patient, consisting of 10 to 21 sessions over 12 months. The intervention will be multimodal, comprising of 1) exercise therapy and a physical activity plan, 2) education/self-management support, 3) work-roadmap to guide participants in finding relevant other care, with optionally 4) online self-management course and 5) workplace examination. Assessments will be performed at baseline and after 3, 6, and 12 months. The primary outcome measure of effectiveness is work ability, as measured with the WAS at 12 months. For the cost-effectiveness analysis, the EuroQol (EQ-5D-5L), self-reported healthcare use, sick leave and productivity while at work will be used to estimate the trial based cost-utility from a societal perspective. A process evaluation, including assessments of adherence and treatment fidelity, will be undertaken using the registrations of the PTs and semi-structured interviews at 12 months follow-up in a random sample of the intervention group.

Discussion: The results of this study will provide insights in the (cost-)effectiveness of a multimodal, PT-led, vocational intervention in people with IA and a reduced work ability.

Trial registration: This study is registered in the International Clinical Trial Registry Platform (ICTRP) under number NL9343.

Background: Cardiac tumours are rare, and clinical manifestations depend on the anatomical location. Symptoms can be the result of cardiac outflow anomalies, constitutional features such as fever, loss of weight, and/or paraneoplastic manifestations such as arthritis. To date, there has only been one other case report in the literature of cardiac sarcoma presenting as paraneoplastic arthropathy.

Case presentation: A 52-year-old woman presented with acute onset corticosteroid-resistant inflammatory polyarthralgia, clubbing and a systolic murmur. Transthoracic echocardiogram revealed a dilated left atrium with an echogenic mass and brain magnetic resonance imaging revealed multiple embolic infarcts. Histopathology following emergency resection showed a Grade 3 left atrial intimal sarcoma. The polyarthralgia and clubbing resolved soon after tumour removal. The patient went on to receive chemotherapy and remains in remission.

Conclusions: This case highlights the rare paraneoplastic association of cardiac sarcoma and arthropathy.

Background: The study aimed to investigate novel biomarkers from the C1q TNF superfamily and evaluate their role in autoimmune inflammatory rheumatic diseases with the goal of identifying an effective biomarker to measure clinical disease activity and assess treatment efficacy.

Methods: Sixty-one Axial spondyloarthritis (AxSpa) patients and 30 healthy controls were enrolled in the study. The serum biomarkers subfatin, CTHRC1, CTRP3, CTRP6, IL-6, IL-17, and TNF-α and the disease indices BASDAI, BASFI, MASES, and ASDAS-ESR/CRP were evaluated and compared. The patients were then classified, and their serum biomarkers were assessed according to their ASDAS scores and their treatment regimens.

Results: Among the studied biomarkers, none showed a significant difference between the patients and the healthy controls. Although the difference was not statistically significant, the median values of serum subfatin, CTHRC1, CTRP3, CTRP6, IL-6, IL-17, and TNF-α were all found to be lower in the AxSpa patients than in the healthy controls. Furthermore, once the patients were classified regarding their disease activity, no correlation between the study biomarkers and levels of clinical disease indices was observed. Finally, biological treatments were found to affect the serum concentration of these biomarkers regardless of the level of disease activity.

Conclusion: Novel adipokines and known modulators of inflammation, circulating subfatin, CTHRC1, CTRP3, CTRP6, IL-6, IL-17, and TNF-α levels may play a role in assessing treatment efficacy, especially in those treated with TNF-inhibitors. However, we failed to demonstrate a correlation between clinical disease activity and serum biomarker levels.