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Zeitschrift Fur Rheumatologie Feb 2024This study aimed to update the prevalence estimates of inflammatory rheumatic diseases (IRD) in Germany.
OBJECTIVE
This study aimed to update the prevalence estimates of inflammatory rheumatic diseases (IRD) in Germany.
METHODS
A systematic literature search in PubMed and Web of Science (last search 08 November 2022) identified original articles (regional and nationwide surveys and claims data analyses for arthritides, connective tissue diseases, and vasculitides) on prevalences for the period 2014-2022. Data sources, collection period, case definition, and risk of bias are reported. Prevalences were estimated from available national data, with consideration of international data.
RESULTS
Screening by two authors yielded 263 hits, of which 18 claims data analyses and 2 surveys met the inclusion criteria. Prevalences ranged from 0.42 to 1.85% (rheumatoid arthritis), 0.32-0.5% (ankylosing spondylitis), 0.11-0.32% (psoriatic arthritis), 0.037-0.14% (systemic lupus erythematosus), 0.07-0.77% (Sjögren's disease/sicca syndrome), 0.14-0.15% (polymyalgia rheumatica, ≥ 40 years), 0.04-0.05% (giant cell arteritis, ≥ 50 years), and 0.015-0.026% (ANCA-associated vasculitis). The risk of bias was moderate in 13 and high in 7 studies. Based on the results, we estimate the prevalence of IRD in Germany to be 2.2-3.0%, which corresponds to approximately 1.5-2.1 million affected individuals. The prevalence of juvenile idiopathic arthritis was reported to be around 0.10% (0.07-0.10%) of 0-18-year-olds, corresponding to about 14,000 children and adolescents in Germany.
CONCLUSION
This systematic review shows an increase in the prevalence of IRD in Germany, which is almost exclusively based on claims data analyses. In the absence of multistage population studies, the available data are, overall, uncertain sources for prevalence estimates, with a moderate to high risk of bias.
Topics: Child; Adolescent; Humans; Prevalence; Arthritis, Rheumatoid; Spondylitis, Ankylosing; Polymyalgia Rheumatica; Sjogren's Syndrome; Lupus Erythematosus, Systemic; Rheumatic Fever; Giant Cell Arteritis; Rheumatic Diseases
PubMed: 36749363
DOI: 10.1007/s00393-022-01302-5 -
Advances in Therapy Dec 2022In light of the lack of an agreed international standard for how to conduct cost-effectiveness analyses (CEAs), including cost-utility analyses (CUAs) from a societal... (Review)
Review
INTRODUCTION
In light of the lack of an agreed international standard for how to conduct cost-effectiveness analyses (CEAs), including cost-utility analyses (CUAs) from a societal perspective, there is uncertainty regarding to what extent the inclusion of productivity losses/gains in economic evaluations can affect cost-effectiveness results and subsequently decisions on whether to recommend new health technologies. To investigate this, we conducted a systematic review of CEAs and CUAs of drug-based therapies for a set of chronic immune-mediated disorders to understand how cost elements and calculation methods related to productivity losses/gains are used, examine the impact on the incremental cost-effectiveness ratio (ICER) of including productivity costs, and explore factors that affect the inclusion of productivity loss.
METHODS
Databases (MEDLINE In-process, MEDLINE, Embase and Cochrane Library) were searched from January 2010 to October 2020 by two independent reviewers for all CEAs and CUAs in adults with any of the following conditions: ankylosing spondylitis, chronic idiopathic urticaria, Crohn's disease, fibromyalgia, juvenile idiopathic arthritis, psoriasis, rheumatoid arthritis, systemic lupus erythematosus and ulcerative colitis. Relevant study data were extracted and evidence was synthesized for both qualitative and quantitative analysis. Productivity cost elements including absenteeism, presenteeism, unemployment/early retirement, premature mortality and informal care were extracted, along with the method used to determine them. A multivariate analysis was performed to identify factors associated with the inclusion of productivity loss.
RESULTS
Our searches identified 5016 records, culminating in 198 unique studies from 234 publications following screening. Most of the studies investigated rheumatoid arthritis (37.0%) or psoriasis (32.0%). The majority were CUAs, with some including both a CEA and a CUA (73.0%). Most studies used a payer perspective only (28.5%) or a societal perspective only (21.0%). Of the 49 studies incorporating productivity losses/gains, 42 reported the type of cost element used; all of these used patient absenteeism, either alone or in addition with other elements. Only 16 studies reported the method used to value productivity changes, of which eight used a human capital approach, four used a friction cost approach and four used both approaches. Twenty-eight of the 49 studies (57.1%) reported inclusion of productivity losses/gains as contributing to more favourable cost-effectiveness outcomes and ICERs, while 12 (24.5%) reported no substantial impact. On the basis of a multivariate analysis, rheumatoid arthritis as the target disease had a statistically significant association with the inclusion of productivity loss compared with psoriasis and inflammatory bowel disease.
CONCLUSIONS
The results of our review suggest that incorporating productivity cost elements may positively affect cost-effectiveness outcomes in evaluations of therapeutics for immune-mediated disorders. Our work highlights the continued need for clarity when reporting how CEAs and CUAs in this disease area are conducted, in order to better inform healthcare decision-making.
Topics: Adult; Humans; Cost-Benefit Analysis; Efficiency; Arthritis, Rheumatoid; Absenteeism; Psoriasis
PubMed: 36205907
DOI: 10.1007/s12325-022-02321-z -
BMC Medicine Mar 2024Previous randomized controlled trials (RCTs) suggested that gut microbiota-based therapies may be effective in treating autoimmune diseases, but a systematic summary is... (Meta-Analysis)
Meta-Analysis
BACKGROUND
Previous randomized controlled trials (RCTs) suggested that gut microbiota-based therapies may be effective in treating autoimmune diseases, but a systematic summary is lacking.
METHODS
Pubmed, EMbase, Sinomed, and other databases were searched for RCTs related to the treatment of autoimmune diseases with probiotics from inception to June 2022. RevMan 5.4 software was used for meta-analysis after 2 investigators independently screened literature, extracted data, and assessed the risk of bias of included studies.
RESULTS
A total of 80 RCTs and 14 types of autoimmune disease [celiac sprue, SLE, and lupus nephritis (LN), RA, juvenile idiopathic arthritis (JIA), spondyloarthritis, psoriasis, fibromyalgia syndrome, MS, systemic sclerosis, type 1 diabetes mellitus (T1DM), oral lichen planus (OLP), Crohn's disease, ulcerative colitis] were included. The results showed that gut microbiota-based therapies may improve the symptoms and/or inflammatory factor of celiac sprue, SLE and LN, JIA, psoriasis, PSS, MS, systemic sclerosis, Crohn's disease, and ulcerative colitis. However, gut microbiota-based therapies may not improve the symptoms and/or inflammatory factor of spondyloarthritis and RA. Gut microbiota-based therapies may relieve the pain of fibromyalgia syndrome, but the effect on fibromyalgia impact questionnaire score is not significant. Gut microbiota-based therapies may improve HbA1c in T1DM, but its effect on total insulin requirement does not seem to be significant. These RCTs showed that probiotics did not increase the incidence of adverse events.
CONCLUSIONS
Gut microbiota-based therapies may improve several autoimmune diseases (celiac sprue, SLE and LN, JIA, psoriasis, fibromyalgia syndrome, PSS, MS, T1DM, Crohn's disease, and ulcerative colitis).
Topics: Humans; Colitis, Ulcerative; Crohn Disease; Celiac Disease; Diabetes Mellitus, Type 1; Fibromyalgia; Gastrointestinal Microbiome; Randomized Controlled Trials as Topic; Autoimmune Diseases; Psoriasis; Scleroderma, Systemic; Spondylarthritis; Lupus Erythematosus, Systemic
PubMed: 38475833
DOI: 10.1186/s12916-024-03303-4 -
Rheumatology (Oxford, England) Oct 2019The clinical impact of anti-drug antibodies (ADAbs) in paediatric patients with JIA remains unknown. This systematic review and meta-analysis aimed to summarize the... (Meta-Analysis)
Meta-Analysis
OBJECTIVE
The clinical impact of anti-drug antibodies (ADAbs) in paediatric patients with JIA remains unknown. This systematic review and meta-analysis aimed to summarize the prevalence of ADAbs in JIA studies; investigate the effect of ADAbs on treatment efficacy and adverse events; and explore the effect of immunosuppressive therapy on antibody formation.
METHODS
PubMed, Embase and the Cochrane Library were systematically searched to identify relevant clinical trials and observational studies that reported prevalence of ADAbs. Studies were systematically reviewed in accordance with the Preferred Reporting Items for Systematic reviews and Meta-Analyses and appropriate proportional and pairwise meta-analyses were performed.
RESULTS
A total of 5183 references were screened; 28 articles, involving 26 studies and 2354 JIA patients, met eligibility criteria. Prevalence of ADAbs ranged from 0% to 82% across nine biologic agents. Overall pooled prevalence of ADAbs was 16.9% (95% CI, 9.5, 25.9). Qualitative analysis of included studies indicated that antibodies to infliximab, adalimumab, anakinra and tocilizumab were associated with treatment failure and/or hypersensitivity reactions. Concomitant MTX uniformly reduced the risk of antibody formation during adalimumab treatment (risk ratio 0.33; 95% CI 0.21, 0.52).
CONCLUSION
The association of ADAbs with treatment failure and hypersensitivity reactions indicates their clinical relevance in paediatric patients with JIA. Based on our findings, we recommend a preliminary course of action regarding immunogenicity of biologic agents in patients with JIA. Further strategies to predict, prevent, detect and manage immunogenicity could optimize treatment outcomes and personalize treatment with biologic therapies.
Topics: Adalimumab; Antibodies, Monoclonal, Humanized; Antibody Formation; Antirheumatic Agents; Arthritis, Juvenile; Biological Factors; Child; Clinical Trials as Topic; Humans; Infliximab; Interleukin 1 Receptor Antagonist Protein; Methotrexate; Observational Studies as Topic
PubMed: 30809664
DOI: 10.1093/rheumatology/kez030 -
Bioscience Reports Jul 2019To investigate whether microRNAs genes' polymorphisms are associated with arthritis. The PubMed, Cochrane Library et al. were systematically searched to identify... (Meta-Analysis)
Meta-Analysis
To investigate whether microRNAs genes' polymorphisms are associated with arthritis. The PubMed, Cochrane Library et al. were systematically searched to identify case-control studies, systematic reviews and meta-analyses. A meta-analysis was performed to calculate odds ratios (ORs), and confidence intervals (CIs) at 95% using fixed-effect model or random-effects model. Twenty-two case-control studies involving 10489 participants fulfilled the inclusion criteria. MiR-146a rs2910164 (G/C) was not significantly associated with the risk of rheumatoid arthritis (RA) in any model. Significant associations were found between miR-146a rs2910164 (G/C) and the risk of psoriatic arthritis (PsA) in the heterozygous model and the dominant model. The heterozygous model showed a significant association between the miR-146a rs2910164 (G/C) polymorphism and ankylosing spondylitis (AS). And there was no significant association of miR-146a rs2910164 (G/C) with risk of juvenile rheumatoid arthritis (JRA) at any model. Additionally, there was a significant association of miR-499 rs3746444 (T/C) with risk of RA at two genetic models, and with a moderate heterogeneity. When subgroup analysis by ethnicity, significant associations were almost found between miR-499 rs3746444 (T/C) and the risk of RA in any model in Caucasian populations, and there is no heterogeneity. The association of miR-146a rs2910164 (G/C) with RA was not found. And there was a significant association between miR-146a rs2910164(G/C) and PsA or AS. MiR-499 rs3746444 (T/C) was associated with RA in Caucasian populations. These findings did not support the genetic association between miR-146a rs2910164 (G/C) and JRA susceptibility, as well as the association of miR-196a-2 rs11614913 (C/T), miR-146a rs2431697, miR-146a rs57095329, miR-149 rs22928323 with arthritis.
Topics: Arthritis; Arthritis, Rheumatoid; Asian People; Genetic Association Studies; Genetic Predisposition to Disease; Humans; MicroRNAs; Polymorphism, Single Nucleotide; Risk Factors; White People
PubMed: 31235484
DOI: 10.1042/BSR20190298 -
JMIR Pediatrics and Parenting Dec 2020Considering the changing landscape of internet use and rising ownership of digital technology by young people, new methods could be considered to improve the current... (Review)
Review
BACKGROUND
Considering the changing landscape of internet use and rising ownership of digital technology by young people, new methods could be considered to improve the current model of juvenile idiopathic arthritis (JIA) management.
OBJECTIVE
This systematic review aims to evaluate the usability of eHealth and mobile health (mHealth) interventions currently available for young people living with JIA.
METHODS
The PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines were used to oversee this review. We systematically searched 15 databases for 252 potential studies; 2 authors independently screened all quantitative studies reporting the use of eHealth and mHealth interventions for young people (aged 1-18 years) diagnosed with JIA. Studies were excluded if they did not report outcome measures or were reviews, commentaries, or qualitative studies. Study methodological quality was scored using the Down and Black (modified) checklist. A narrative descriptive methodology was used to quantify the data because of heterogeneity across the studies.
RESULTS
A total of 11 studies were included in this review, reporting 7 eHealth and mHealth interventions for young people (aged 4-18 years) living with JIA, targeting health issues such as pain, health-related quality of life, physical activity, and chronic disease self-management. The usability of the interventions was facilitated through training and ongoing support. The engagement was promoted by a combination of persuasive influences, and barriers preventing adherence were removed through personal reminders and flexible program schedules to cater to JIA and non-JIA illnesses or other commonly seen activities in childhood. The feedback obtained was that most young people and their parents liked the interventions.
CONCLUSIONS
The results of this review need to be considered cautiously because of the lack of rigorous testing and heterogeneity, which limits the detailed descriptions of data synthesis. Further research is needed to consider gender differences, associated costs, and the effectiveness of the interventions on health outcomes to better support young people living with JIA.
PubMed: 33258786
DOI: 10.2196/15833 -
Cancer Epidemiology Oct 2023Childhood leukemia and many autoimmune (AI) diseases are severe pediatric conditions with lifelong consequences. AI diseases form a heterogeneous disease group affecting... (Review)
Review
BACKGROUND
Childhood leukemia and many autoimmune (AI) diseases are severe pediatric conditions with lifelong consequences. AI diseases form a heterogeneous disease group affecting about 5 % of children worldwide, while leukemia is the most common malignancy among children aged 0-14 years. The timing and similarities in suggested inflammatory and infectious triggers of AI disease and leukemia have raised a question whether the diseases share common etiological origins. We conducted a systematic review to evaluate the evidence linking childhood leukemia and AI diseases.
DATA SOURCES
In the systematic literature search CINAHL (from 1970), Cochrane Library (form 1981), PubMed (from 1926) and Scopus (from 1948) were queried in June 2023.
REVIEW METHODS
We included studies covering the association between any AI disease and acute leukemia, limiting it to children and adolescents under 25 years old. The studies were reviewed independently by two researchers and the risk of bias was assessed.
RESULTS
A total of 2119 articles were screened and 253 studies were selected for detailed evaluation. Nine studies met the inclusion criteria, of which eight were cohort studies and one was a systematic review. The diseases covered were type 1 diabetes mellitus, inflammatory bowel diseases and juvenile arthritis alongside acute leukemia. Five cohort studies were suitable for more detailed analysis: a rate ratio for leukemia diagnosis after any AI disease was 2.46 (95 % CI 1.17-5.18; heterogeneity I 15 %) with a random-effects model.
CONCLUSIONS
The results of this systematic review indicate that AI diseases in childhood are associated with a moderately increased risk of leukemia. The association for individual AI diseases needs further investigation.
PubMed: 37423102
DOI: 10.1016/j.canep.2023.102411 -
Medicine Jan 2023
Topics: Humans; Arthritis, Juvenile; Synovectomy; Knee Joint
PubMed: 36607889
DOI: 10.1097/MD.0000000000032634 -
Journal of Medical Internet Research Feb 2022Juvenile idiopathic arthritis (JIA) management aims to promote remission through timely, individualized, well-coordinated interdisciplinary care using a range of... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
Juvenile idiopathic arthritis (JIA) management aims to promote remission through timely, individualized, well-coordinated interdisciplinary care using a range of pharmacological, physical, psychological, and educational interventions. However, achieving this goal is workforce-intensive. Harnessing the burgeoning eHealth and mobile health (mHealth) interventions could be a resource-efficient way of supplementing JIA management.
OBJECTIVE
This systematic review aims to identify the eHealth and mHealth interventions that have been proven to be effective in supporting health outcomes for children and young people (aged 1-18 years) living with JIA.
METHODS
We systematically searched 15 databases (2018-2021). Studies were eligible if they considered children and young people (aged 1-18 years) diagnosed with JIA, an eHealth or mHealth intervention, any comparator, and health outcomes related to the used interventions. Independently, 2 reviewers screened the studies for inclusion and appraised the study quality using the Downs and Black (modified) checklist. Study outcomes were summarized using a narrative, descriptive method and, where possible, combined for a meta-analysis using a random-effects model.
RESULTS
Of the 301 studies identified in the search strategy, 15 (5%) fair-to-good-quality studies met the inclusion criteria, which identified 10 interventions for JIA (age 4-18.6 years). Of these 10 interventions, 5 (50%) supported symptom monitoring by capturing real-time data using health applications, electronic diaries, or web-based portals to monitor pain or health-related quality of life (HRQoL). Within individual studies, a preference was demonstrated for real-time pain monitoring over recall pain assessments because of a peak-end effect, improved time efficiency (P=.002), and meeting children's and young people's HRQoL needs (P<.001) during pediatric rheumatology consultations. Furthermore, 20% (2/10) of interventions supported physical activity promotion using a web-based program or a wearable activity tracker. The web-based program exhibited a moderate effect, which increased endurance time, physical activity levels, and moderate to vigorous physical activity (standardized mean difference [SMD] 0.60, SD 0.02-1.18; I=79%; P=.04). The final 30% (3/10) of interventions supported self-management development through web-based programs, or apps, facilitating a small effect, reducing pain intensity (SMD -0.14, 95% CI -0.43 to 0.15; I=53%; P=.33), and increasing disease knowledge and self-efficacy (SMD 0.30, 95% CI 0.03-0.56; I=74%; P=.03). These results were not statistically significant. No effect was seen regarding pain interference, HRQoL, anxiety, depression, pain coping, disease activity, functional ability, or treatment adherence.
CONCLUSIONS
Evidence that supports the inclusion of eHealth and mHealth interventions in JIA management is increasing. However, this evidence needs to be considered cautiously because of the small sample size, wide CIs, and moderate to high statistical heterogeneity. More rigorous research is needed on the longitudinal effects of real-time monitoring, web-based pediatric rheumatologist-children and young people interactions, the comparison among different self-management programs, and the use of wearable technologies as an objective measurement for monitoring physical activity before any recommendations that inform current practice can be given.
Topics: Adolescent; Arthritis, Juvenile; Child; Child, Preschool; Exercise; Humans; Infant; Quality of Life; Self-Management; Telemedicine
PubMed: 35107431
DOI: 10.2196/30457 -
BMC Medical Research Methodology May 2023There is a pressing need to improve the accuracy of rare disease clinical study endpoints. Neutral theory, first described here, can be used to assess the accuracy of...
BACKGROUND
There is a pressing need to improve the accuracy of rare disease clinical study endpoints. Neutral theory, first described here, can be used to assess the accuracy of endpoints and improve their selection in rare disease clinical studies, reducing the risk of patient misclassification.
METHODS
Neutral theory was used to assess the accuracy of rare disease clinical study endpoints and the resulting probability of false positive and false negative classifications at different disease prevalence rates. Search strings were extracted from the Orphanet Register of Rare Diseases using a proprietary algorithm to conduct a systematic review of studies published until January 2021. Overall, 11 rare diseases with one disease-specific disease severity scale (133 studies) and 12 rare diseases with more than one disease-specific disease severity scale (483 studies) were included. All indicators from clinical studies were extracted, and Neutral theory was used to calculate their match to disease-specific disease severity scales, which were used as surrogates for the disease phenotype. For those with more than one disease-severity scale, endpoints were compared with the first disease-specific disease severity scale and a composite of all later scales. A Neutrality score of > 1.50 was considered acceptable.
RESULTS
Around half the clinical studies for half the rare diseases with one disease-specific disease severity score (palmoplantar psoriasis, achalasia, systemic lupus erythematosus, systemic sclerosis and Fournier's gangrene) met the threshold for an acceptable match to the disease phenotype, one rare disease (Guillain-Barré syndrome) had one study with an acceptable match, and four diseases (Behcet's syndrome, Creutzfeldt-Jakob disease, atypical hemolytic uremic syndrome and Prader-Willi syndrome) had no studies. Clinical study endpoints in almost half the rare diseases with more than one disease-specific DSS (acromegaly, amyotrophic lateral sclerosis, cystic fibrosis, Fabry disease and juvenile rheumatoid arthritis) were a better match to the composite, while endpoints in the remaining rare diseases (Charcot Marie Tooth disease, Gaucher disease Type I, Huntington's disease, Sjogren's syndrome and Tourette syndrome) were a worse match. Misclassifications varied with increasing disease prevalence.
CONCLUSIONS
Neutral theory confirmed that disease-severity measurement needs improvement in rare disease clinical studies, especially for some diseases, and suggested that the potential for accuracy increases as the body of knowledge on a disease increases. Using Neutral theory to benchmark disease-severity measurement in rare disease clinical studies may reduce the risk of misclassification, ensuring that recruitment and treatment effect assessment optimise medicine adoption and benefit patients.
Topics: Humans; Rare Diseases; Endpoint Determination; Clinical Studies as Topic
PubMed: 37210484
DOI: 10.1186/s12874-023-01947-z