154 research outputs found

    'Have', 'with' and 'without'

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    The research reported in this paper is part of our attempt to get to a deeper understanding of why 'with' and 'without' are special prepositions in taking singular bare nouns more easily than other prepositions. The paper focuses on the semantics of existential and incorporation 'have', which we take to be the same and to constitute the verbal counterpart of 'with' and 'without'. We propose existential/incorporation 'have' builds relations: it selects one-place predicates and turns them into two-place predicates

    Treating juvenile idiopathic arthritis to target: What is the optimal target definition to reach all goals?

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    In 2018, an international Task Force formulated recommendations for treating Juvenile Idiopathic Arthritis (JIA) to target. The Task Force has not yet resolved three issues. The first issue is the lack of a single "best" target. The Task Force decided not to recommend the use of a specific instrument to assess inactive disease or remission. Recent studies underscore the use of a broad target definition. The second issue is the basic assumption that a treatment aggressively aimed at the target will have 'domino effects' on other treatment goals as well. Thus far, this assumption was not confirmed for pain, fatigue and stiffness. The third issue is shared decision-making, and the role of individual patient targets. Nowadays, patients and parents should have a more active role in choosing targets and their personal treatment goals. In our department the electronic medical records have been restructured in such a way that the patient's personal treatment goals with a target date appears on the front page. The visualization of their specific personal goals helps us to have meaningful discussions on the individualized treatment strategy and to share decisions. In conclusion, a joint treat to target (T2T) strategy is a promising approach for JIA. The Task Force formulated valuable overarching principles and a first version of recommendations. However, implementation of T2T needs to capture more than just inactive disease. Patients and parents should have an active role in choosing personal targets as well

    Case Report: Lessons Learned From Subsequent Autologous and Allogeneic Hematopoietic Stem Cell Transplantations in a Pediatric Patient With Relapsing Polychondritis

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    Background: Autologous hematopoietic stem cell transplantation (autoHSCT) is increasingly being recognized as a treatment option for severe refractory autoimmune diseases (AD). However, efficacy is hampered by high relapse rates. In contrast, allogeneic HSCT (alloHSCT) has high potential to cure AD, but is associated with significant morbidity and mortality, and data in AD are limited. Experience with autoHSCT in relapsing polychondritis, a rare episodic inflammatory disorder characterized by destruction of cartilage, is scarce and alloHSCT has not been described before. Case Presentation: Here, we present a case of a 9-year-old girl who was diagnosed with relapsing polychondritis, with severe airway involvement requiring a tracheostomy. The disease proved to be steroid-dependent and refractory to a wide array of disease-modifying anti-rheumatic drugs and biologicals. After an autoHSCT procedure, the disease became inactive for a short period of time, until the patient experienced a relapse after 31 days, accompanied by repopulation of effector/memory CD8+ T cells. Because of persistent inflammation and serious steroid toxicity, including severe osteoporosis, growth restriction, and excessive weight gain, the patient was offered an alloHSCT. She experienced transient antibody-mediated immune events post-alloHSCT, which subsided after rituximab. She ultimately developed a balanced immune reconstitution and is currently still in long-term disease remission, 8 years after alloHSCT. Conclusion: This case adds to the few existing reports on autoHSCT in relapsing polychondritis and gives new insights in its pathogenesis, with a possible role for CD8+ T cells. Moreover, it is the first report of successful alloHSCT as a treatment for children with this severe autoimmune disease

    The Flemish version of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR)

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    The Juvenile Arthritis Multidimensional Assessment Report (JAMAR) is a new parent/patient reported outcome measure that enables a thorough assessment of the disease status in children with juvenile idiopathic arthritis (JIA). We report the results of the cross-cultural adaptation and validation of the parent and patient versions of the JAMAR in the Flemish language. The reading comprehension of the questionnaire was tested in 10 JIA parents and patients. Each participating centre was asked to collect demographic, clinical data and the JAMAR in 100 consecutive JIA patients or all consecutive patients seen in a 6-month period and to administer the JAMAR to 100 healthy children and their parents. The statistical validation phase explored descriptive statistics and the psychometric issues of the JAMAR: the 3 Likert assumptions, floor/ceiling effects, internal consistency, Cronbach's alpha, interscale correlations, test-retest reliability, and construct validity (convergent and discriminant validity). A total of 100 JIA patients (8% systemic, 33% oligoarticular, 24% RF negative polyarthritis, 35% other categories) and 99 healthy children, were enrolled in two centres. The JAMAR components discriminated well healthy subjects from JIA patients. All JAMAR components revealed good psychometric performances. In conclusion, the Flemish version of the JAMAR is a valid tool for the assessment of children with JIA and is suitable for use both in routine clinical practice and clinical research

    Genomic Health Literacy Interventions in Pediatrics:Scoping Review

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    BACKGROUND: The emergence of genetic and genomic sequencing approaches for pediatric patients has raised questions about the genomic health literacy levels, attitudes toward receiving genomic information, and use of this information to inform treatment decisions by pediatric patients and their parents. However, the methods to educate pediatric patients and their parents about genomic concepts through digital health interventions have not been well-established. OBJECTIVE: The primary objective of this scoping review is to investigate the current levels of genomic health literacy and the attitudes toward receiving genomic information among pediatric patients and their parents. The secondary aim is to investigate patient education interventions that aim to measure and increase genomic health literacy among pediatric patients and their parents. The findings from this review will be used to inform future digital health interventions for patient education. METHODS: A scoping review using PRISMA-ScR (Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews) guidelines and protocols was completed using the following databases: MEDLINE, Embase, CINAHL, and Scopus. Our search strategy included genomic information inclusive of all genetic and genomic terms, pediatrics, and patient education. Inclusion criteria included the following: the study included genetic, genomic, or a combination of genetic and genomic information; the study population was pediatric (children and adolescents <18 years) and parents of patients with pediatric illnesses or only parents of patients with pediatric illnesses; the study included an assessment of the knowledge, attitudes, and intervention regarding genomic information; the study was conducted in the last 12 years between 2008 and 2020; and the study was in the English language. Descriptive data regarding study design, methodology, disease population, and key findings were extracted. All the findings were collated, categorized, and reported thematically. RESULTS: Of the 4618 studies, 14 studies (n=6, 43% qualitative, n=6, 43% mixed methods, and n=2, 14% quantitative) were included. Key findings were based on the following 6 themes: knowledge of genomic concepts, use of the internet and social media for genomic information, use of genomic information for decision-making, hopes and attitudes toward receiving genomic information, experiences with genetic counseling, and interventions to improve genomic knowledge. CONCLUSIONS: This review identified that older age is related to the capacity of understanding genomic concepts, increased genomic health literacy levels, and the perceived ability to participate in decision-making related to genomic information. In addition, internet-searching plays a major role in obtaining genomic information and filling gaps in communication with health care providers. However, little is known about the capacity of pediatric patients and their parents to understand genomic information and make informed decisions based on the genomic information obtained. More research is required to inform digital health interventions and to leverage the leading best practices to educate these genomic concepts

    The Dutch version of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR)

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    The Juvenile Arthritis Multidimensional Assessment Report (JAMAR) is a new parent/patient reported outcome measure that enables a thorough assessment of the disease status in children with juvenile idiopathic arthritis (JIA). We report the results of the cross-cultural adaptation and validation of the parent and patient versions of the JAMAR in the Dutch language. The reading comprehension of the questionnaire was tested in ten JIA parents and patients. Each participating centre was asked to collect demographic, clinical data and the JAMAR in 100 consecutive JIA patients or all consecutive patients seen in a 6-month period and to administer the JAMAR to 100 healthy children and their parents. The statistical validation phase explored descriptive statistics and the psychometric issues of the JAMAR: the three Likert assumptions, floor/ceiling effects, internal consistency, Cronbach\u2019s alpha, interscale correlations, test\u2013retest reliability, and construct validity (convergent and discriminant validity). A total of 209 JIA patients (14.3% systemic, 39.7% oligoarticular, 25.8% RF negative polyarthritis, 20.2% other categories) and 107 healthy children were enrolled in two centres. The JAMAR components discriminated well healthy subjects from JIA patients. All JAMAR components revealed good psychometric performances. In conclusion, the Dutch version of the JAMAR is a valid tool for the assessment of children with JIA and is suitable for use both in routine clinical practice and clinical research

    Randomized, double-blind, placebo-controlled, multicentre pilot study on the effects of empagliflozin on clinical outcomes in patients with acute decompensated heart failure (EMPA-RESPONSE-AHF)

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    Aims: Inhibition of sodium–glucose co-transporter 2 (SGLT2) reduces the risk of death and heart failure (HF) admissions in patients with chronic HF. However, safety and clinical efficacy of SGLT2 inhibitors in patients with acute decompensated HF are unknown. Methods and results: In this randomized, placebo-controlled, double-blind, parallel group, multicentre pilot study, we randomized 80 acute HF patients with and without diabetes to either empagliflozin 10 mg/day or placebo for 30 days. The primary outcomes were change in visual analogue scale (VAS) dyspnoea score, diuretic response (weight change per 40 mg furosemide), change in N-terminal pro brain natriuretic peptide (NT-proBNP), and length of stay. Secondary outcomes included safety and clinical endpoints. Mean age was 76 years, 33% were female, 47% had de novo HF and median NT-proBNP was 5236 pg/mL. No difference was observed in VAS dyspnoea score, diuretic response, length of stay, or change in NT-proBNP between empagliflozin and placebo. Empagliflozin reduced a combined endpoint of in-hospital worsening HF, rehospitalization for HF or death at 60 days compared with placebo [4 (10%) vs. 13 (33%); P = 0.014]. Urinary output up until day 4 was significantly greater with empagliflozin vs. placebo [difference 3449 (95% confidence interval 578–6321) mL; P < 0.01]. Empagliflozin was safe, well tolerated, and had no adverse effects on blood pressure or renal function. Conclusions: In patients with acute HF, treatment with empagliflozin had no effect on change in VAS dyspnoea, diuretic response, NT-proBNP, and length of hospital stay, but was safe, increased urinary output and reduced a combined endpoint of worsening HF, rehospitalization for HF or death at 60 days

    Prevention of disease flares by risk-adapted stratification of therapy withdrawal in juvenile idiopathic arthritis : results from the PREVENT-JIA trial

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    Publisher Copyright: © Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.OBJECTIVES: To investigate the ability of high-sensitivity C-reactive protein (hsCRP) and S100A12 to serve as predictive biomarkers of successful drug withdrawal in children with clinical remission of juvenile idiopathic arthritis (JIA). METHODS: This multicentre trial (PREVENT-JIA) enrolled 119 patients with JIA in clinical remission, and 100 patients reached the intervention phase in which the decision whether to continue or stop treatment was based on S100A12 and hsCRP levels. Patients were monitored for 12 months after stopping medication for flares of disease. Results were compared with withdrawal of therapy without biomarker-based stratification in patients from the German Biologika in der Kinderrheumatologie (BiKeR) pharmacovigilance registry. RESULTS: In the PREVENT-JIA group, 49 patients had a flare, and 45% of patients stopping medication showed flares within the following 12 months. All patients (n=8) continuing therapy due to permanently elevated S100A12/hsCRP at more than one visit flared during the observation phase. In the BiKeR control group, the total flare rate was 62%, with 60% flaring after stopping medication. The primary outcome, time from therapy withdrawal to first flare (cumulative flare rate after therapy withdrawal), showed a significant difference in favour of the PREVENT-JIA group (p=0.046; HR 0.62, 95% CI 0.38 to 0.99). As additional finding, patients in the PREVENT-JIA trial stopped therapy significantly earlier. CONCLUSION: Biomarker-guided strategies of therapy withdrawal are feasible in clinical practice. This study demonstrates that using predictive markers of subclinical inflammation is a promising tool in the decision-making process of therapy withdrawal, which translates into direct benefit for patients. TRIAL REGISTRATION NUMBER: ISRCTN69963079.publishersversionPeer reviewe

    A diagnostic prediction model for separating juvenile idiopathic arthritis and chronic musculoskeletal pain syndrome

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    Objective: To develop and validate a diagnostic prediction model that can distinguish between juvenile idiopathic arthritis (JIA) and chronic musculoskeletal pain syndrome (CMPS) based on patient-reported outcomes. Study design: This retrospective cohort study evaluated whether the Juvenile Arthritis Multidimensional Assessment Report (JAMAR) performs well in distinguishing JIA from CMPS. We analyzed JAMARs completed by 287 patients at their first visit to the pediatric rheumatology department of Wilhelmina Children's Hospital in Utrecht, The Netherlands. Relevant JAMAR items for predicting a diagnosis of JIA were selected in a penalized multivariable model suitable for clinical application. The model was subsequently validated with new data from the same center. Results: A total of 196 JAMARs (97 JIA, 99 CMPS) were collected in the model development data, and 91 JAMARs (48 JIA, 43 CMPS) were collected in the validation data. Variables in the prediction model that were strongest associated with a diagnosis of JIA instead of CMPS were asymmetric pain/swelling in the shoulder (OR, 2.34), difficulty with self-care (OR, 2.41), skin rash (OR, 2.07), and asymmetric/pain swelling in the knee (OR, 2.29). Calibration and discrimination (area under the receiver operating characteristic curve, 0.83; 95% CI, 0.74-0.92) of the model in the validation data were good. Conclusions: Several items from the JAMAR questionnaire can potentially distinguish JIA from CMPS in patients with corresponding symptoms. We present an easy-to-use, adjusted, and validated model to separate these 2 diagnoses early at presentation based on patient-reported outcomes to facilitate proper referral and treatment
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