tbuckley/cabot-search huggingface dataset:
AB0360D ADHERENCE TO THERAPY IN RHEUMATIC PATIENTS TREATED WITH SUBCUTANEOUS BIOLOGICS
Authors: Michele Colaci, Tiziana Puglisi, Elena Calaciura
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background</h3> Patients' adherence to treatments is undoubtedly a fundamental part of a successful therapeutic strategy, even for rheumatic patients. In fact, the lack of adherence to treatment should be considered in all cases of "supposed" inefficacy of therapy prescribed. <h3>Objectives</h3> We aimed to evaluate the adherence to treatment in a series of rheumatic patients from a single Centre (Catania, Sicily, Italy). <h3>Methods</h3> We recruited 85 consecutive patients (M/F: 25/60; mean age 52.713.5 years) affected by chronic inflammatory arthropathies treated with subcutaneous biologics. All patients completed the 5-item version of Compliance Questionnaire for Rheumatology (CQR 5)<sup>1</sup> and the Morisky Medication Adherence Scales (MMAS-8)<sup>2</sup>, in order to estimate their adherence to therapy. Moreover, all patients completed the Patient Global Assessment (PGA), the Visual Analogue Scale (VAS) for articular pain, and the Health Assessment Questionnaire (HAQ), in order to estimate their personal judgments on the effectiveness of therapies. Finally, the eventual presence of adverse events were reported. <h3>Results</h3> Considering MMAS-8, 7/85 (8.2%) patients were low adherent to treatment (score &lt;6), and 27/85 (31.8%) presented medium-grade adherence (score 6-7). Interestingly, the CQR 5 failed to precisely identify these patients. Adherence to therapy was not related to age, gender, education level, articular disease, adverse events, association with DMARDs. Notably, the use of etanercept (originator or biosimilar) was significant more frequent in low-adherent patients (5/7 vs. 19/78; p=0.017). Finally, a trend to higher mean levels of PGA (57.1 vs. 37.6; p=0.06), VAS (68.6 vs. 42.6; p=0.06), and HAQ (1.3 vs. 0.7; p=0.052) was reported in low-adherent patients. <h3>Conclusion</h3> Our preliminary study confirmed that the low adherence to therapy (better identified with MMAS-8) is a relevant issue in the management of rheumatic patients treated with biologics. Moreover, low-adherent patients tend to perceive a worse disease condition. Further studies can clarify if low adherence is causally associated with less efficacy of therapy. <h3>References</h3> [1] Hughes, et al. BMC Musculoskeletal Disorders 2013; 14:286. 2. Morisky DE, et al. J Clin Hypertens 2008; 10:348–354. <h3>Disclosure of Interests</h3> None declared
AB0456 PERCEPTIONS OF GLUCOSTEROID SIDE EFFECTS IN RHEUMATOID ARTHRITISPATIENTS
Authors: Saoussen Miladi, Makhlouf yasmine, Alia Fazaa, Selma Chekili, Salma Kassab, Kmar Ouenniche, Leila Souebni, Kaouther Ben Abdelghani, Ahmed Laatar
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background:</h3> Most of the new recommendations proposed for the management of Rheumatoid arthritis (RA) emphasize the involvement of patients in therapeutic decision-making. This should encourage us to evaluate their level of information, fears and beliefs about the different treatments especially corticosteroids. <h3>Objectives:</h3> The aim of this study was to evaluate the fears and beliefs of Tunisian patients with RA regarding corticosteroids. <h3>Methods:</h3> A prospective study including 35 RA patients according to the ACR/EULAR 2010 criteria was performed. All of them were treated with corticosteroid therapy in addition to DMARDs and their usual long-term treatment. Every patient included in the study completed a questionnaire, containing sociodemographic component and evaluating corticosteroid modality use as well as side effects[1]. <h3>Results:</h3> The study included 35 patients with female predominance (74, 3%).The mean age was 57 years (29-72). The mean duration of the disease was 8 years (2-13 years). All RA patients were immunopositive for rheumatoid factor and/or for Anti-cyclic citrullinated peptide antibody (anti-CCP). Twenty-six patients were on Methotrexate, 6 on Infliximab, and 3 on Rituximab. Corticosteroid therapy was prescribed at a mean dose of 7.5 mg prednisone (5 to 10 mg).The level of education of our patients was generally low: 17 were illiterate, 10 had received a primary or secondary education, only 8 attended college. None of them was part of a medical or paramedical profession. Twenty patients, of whom eight diabetic, had an idea about the potentially harmful effect of corticosteroids on glycemic equilibrium (14 patients reported hyperglycemia and six patients mentioned hypoglycemia). Forty percent were aware of obesity as an effect correlated with corticotherapy. Moreover, 34.3% of patients talked about the hypertensive peak as a serious complication. Osteoporosis was mentioned in 17.1% of cases. About 10 patients had no idea about the adverse effects of steroids. When asking them about their source of information: they mentioned respectively television and radio programs with medical informative content, environment and medical or paramedical medical staff. <h3>Conclusion:</h3> This study highlights the insufficiency of the level of information provided for RA patients regarding corticosteroids side effects. New awareness-raising materials should be reinforced and adapted to make full use of their potential with less likelihood of adverse effects. <h3>References</h3> [1] Oray M, Abu Samra K, Ebrahimiadib N, Meese H, Foster CS. Long-term side effects of glucocorticoids. Expert Opin Drug Saf. 2016;15:457-65 <h3>Disclosure of Interests:</h3> None declared
THU0421 EFFECT OF GAME BASED EXERCISE PROGRAMS ON PAIN, FUNCTIONAL MOBILITY AND BALANCE IN PATIENTS WITH KNEE OSTEOARTHRITIS: RANDOMIZED CONTROLLED STUDY
Authors: DEMİRİZ SERAP YILMAZ, Ali Erdem Baki
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background</h3> Osteoarthritis is a chronic joint disease affecting the knee, hip and hand joints. Exercise is an integral component of conservative treatment for osteoarthritis. However, virtual reality applications using interactive games for rehabilitation have become a focus of interest in recent years. <h3>Objectives</h3> The aim of this study was to evaluate the effects of virtual reality games on knee pain, functional mobility and balance in patients with knee osteoarthritis. <h3>Methods</h3> Fifty patients who were complaining of knee pain, aged 40-70 years, and were diagnosed with Kellgren-Lawrence stage 2, 3, 4 idiopathic knee osteoarthritis was included. Patients were randomly assigned to two equal groups (n = 25) as the control and the study group. Age, sex, weight, height, duration of illness was recorded for all patients. In the bilateral two-way knee radiography; Kellgren-Lawrence score of the knee with the higher visual analog scale (VAS) pain score was recorded. Conventional physical therapy program, associated with knee osteoarthritis exercises, was applied routinely to all patients with knee osteoarthritis for three weeks. In the study group, all patients received virtual reality game-based exercise program by using a game console. In both groups, baseline and final values of VAS score, osteoarthritis index total score (WOMAC), and intra-community balance and mobility scale (CB&amp;M) score of each patient were recorded. Mann-Whitney U and Independent Samples T tests were used for statistical analysis, and <i>p</i> value less than 0.05 was considered significant. <h3>Results</h3> In both groups, significant change in VAS, WOMAC osteoarthritis index, and CB&amp;M score was observed (<i>p</i>&lt;0.05). In the study group, VAS, WOMAC osteoarthritis index, and CB&amp;M scores were significantly different than control group (<i>p</i>&lt;0.05). The differences between baseline and final scores of VAS and WOMAC were significantly higher (<i>p</i>&lt;0.05) in study group. <h3>Conclusion</h3> The results of the present study showed that virtual reality game-based exercise programs performed better results than conventional treatment program in patients with knee osteoarthritis. <h3>References</h3> [1] Bennell KL, Dobson F, Hinman RS. Exercise in osteoarthritis: moving from prescription to adherence. Best Pract Res Clin Rheumatol. 2014; 28(1): 93-117. [2] Ju SB, Park GD, Kim SS. Effects of proprioceptive circuit exercise on knee joint pain and muscle function in patients with knee osteoarthritis. J Phys Ther Sci. 2015; 27(8): 2439-41. [3] Molina KI, Ricci NA, de Moraes SA, Perracini MR. Virtual reality using games for improving physical functioning in older adults: a systematic review. J Neuroeng Rehabil. 2014; 11: 156. <h3>Disclosure of Interests</h3> None declared
Abstract 18206: Fatal Myocarditis is Associated With Rare Cardiomyopathy Gene Variants
Authors: Zhanna Georgievskaya, Yingying Tang, Nori Williams, Barbara Sampson, Bruce D Gelb, Amy R Kontorovich
Journal: Circulation (2017)

Introduction: Acute myocarditis (AM) is the most common cause of heart failure in otherwise healthy children. Causative viruses abound but resulting myocarditis is rare and variable. Thus, human ge...
Safety and Efficacy of Antithrombotic Strategies in Patients With Atrial Fibrillation Undergoing Percutaneous Coronary Intervention
Authors: Renato D. Lopes, Hwanhee Hong, Ralf E. Harskamp, Deepak L. Bhatt, Roxana Mehran, Christopher P. Cannon, Christopher B. Granger, Freek W. A. Verheugt, Jianghao Li, Jurriën M. ten Berg, Nikolaus Sarafoff, C. Michael Gibson, John H. Alexander
Journal: JAMA Cardiology (2019)

The antithrombotic treatment of patients with atrial fibrillation (AF) and coronary artery disease, in particular with acute coronary syndrome (ACS) and/or percutaneous coronary intervention (PCI), poses a significant treatment dilemma in clinical practice.
AB0714 BALANCE AND FALLS IN AXIAL SPONDYLOARTHRITIS: A CROSS SECTIONAL STUDY
Authors: Kerilyn Brenda Mewes, Betania Longo, Ana Paula Beckhauser de Campos, Juliana Simioni, Thelma Laroca Skare
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background:</h3> Spondyloarthritis (SpA) patients may suffer of balance loss predisposing them to falls. <h3>Objectives:</h3> To study balance impairment and falls in SpA patients and its association with clinical and epidemiological variables, disease activity, functional and metrology indexes. <h3>Methods:</h3> Cross sectional study of 55 SpA patients with axial disease. Clinical and epidemiological were collected from the charts. Balance was accessed by Berg Balance Scale(BBS). The following instruments were applied: aSDAS(Ankylosing Spondylitis Disease activity Score)-ESR, aSDAS-CRP, BASDAI(Bath ankylosing Spondylitis Disease activity index), BASFI(Bath ankylosing Spondylitis Functional index), BASMI(Bath ankylosing Spondylitis Metrology index) and aSQoL (Ankylosing spondylitis quality of life questionnaire). The number of falls in the last year was collected. <h3>Results:</h3> In this sample, 30.9% had high risk of falls by the BBS and 25.4% recalled having at least one fall in the last years. The BBS values were lower in those with white ethnic background(p=0.01); and smokers(p=0.03) and with HLA-B27(p=0.03) and correlated inversely with BASDAI(rho=-0.28), aSDAS-ESR(rho=-0.32) and aSDAS-CRP(rho=-0.33), BASFI(rho=-0.71,p&lt;0.0001), BASMI(rho=-0.80; p&lt;0.0001), aSQoL(rho=-0.57;p&lt;0.001) and age(rho=-0.50;p&lt;0.001). Multivariated analysis showed that BASFI and BASMI were independently associated with BBS(p=0.02 and 0.0001 respectively). Patients with falls had lower BBS (p=0.03) and loss of balance correlated with impairment of the quality of life(rho=-0.56;p&lt;0.001). <h3>Conclusion:</h3> Balance is impaired in 1/3 of SpA patients and the BBS is associated mainly with functional and metrology indexes, showing that patients with severe cumulative damage are more affected. <h3>References</h3> [1] - Pompeu JE, Romano RS, Pompeu SM, Lima SM. Static and dynamic balance in subjects with ankylosing spondylitis: literature review. Rev Bras Reumatol. 2012; 52:409-16. [2] - Sawacha Z, Carraro E, Del Din S, Guiotto a, Bonaldo L, Punzi L, et al. Biomechanical assessment of balance and posture in subjects with ankylosing spondylitis. J. Neuroeng Rehabil. 2012; 9:63. <h3>Disclosure of interests:</h3> None declared
PS1044 THE PROGNOSTIC SIGNIFICANCE OF BCR‐1 AND BCR‐3 ISOFORM OF PML‐RARA AND FLT3‐ITD IN PATIENTS WITH ACUTE PROMYELOCYTIC LEUKEMIA
Authors: E. Rasekh, G. Elsayed, Y. Madney, M. El Gammal
Journal: HemaSphere (2019)

Background: Acute promyelocytic leukemia (APL) has a characteristic peculiar morphologic, genetic features and more favorable outcome. Aims: is to study the differential effect of BCR‐1 and BCR‐3 isoforms of PML‐RARA together with FLT3‐ITD mutational status on outcome of APL patients. Methods: All patients referred to the outpatient medical and pediatric oncology clinic, National cancer institute (NCI), Cairo University, Egypt in the period from May 2012 to January 2018. The diagnosis of APL was based on morphologic examination of the bone marrow and peripheral blood smears and was confirmed by the detection of the PML‐RARA transcript by FISH and RT‐PCR. Results: the study included 118 patients with newly diagnosed de novo APL (71 (60%) adults and 47 (39.8%) children). Based on the initial TLC, pediatric APL patients have higher (48.9%) risk than adults (30%) (p = 0.044). The CR rate was 91% (77/84), after excluding early induction related deaths (18) and patients who did not receive treatment at NCI (no beds available) (16). All pediatric patients (both BCR‐1 and BCR‐3) and all adult patients with BCR‐3 achieved CR while 22/28 (78.5%) of patients with BCR‐1 achieved CR (p = 0.04). Adult patients with FLT3 mutation (5/8 = 62.5%) received more high‐risk regimen than patients with wild FLT3‐ITD (10/38 = 26.3%)(p = 0.047). FLT3‐ITD mutation was associated with more hemorrhage and DIC (p = 0.038), and lower 2‐year OS (96% v 44.6%)(p = 0.001) in pediatric patients. Also DIC and Hemorrhage are associated with lower 2‐year OS in pediatric patients (91.4% v 48.6%)( p &lt; 0.001). The 1‐year and 2‐year OS for pediatric patients were better than adults (56.2% v 83% and 80.7 % v 49.8% respectively) with a median of 39.4 month versus 12month respectively (p = 0.005). On multivariate analysis, Age was an independent prognostic variable affecting survival, HR 2.6 (95% CI 1.3–5.3, P = 0.007)(adults had hazards ratio of death 2.6 times higher than pediatrics). DIC &amp; FLT3‐ITD were independent prognostic variables affecting survival in the pediatric APL with HR 12.3 (95% CI 1.46–104.61, P = 0.021) and 5.2 (95% CI 1.01–26.95, P = 0.048) respectively. Summary/Conclusion: BCR‐1 isoform of PML‐RARA is associated with decreased response to ATRA‐based regimens in adult APL patients. DIC &amp; FLT3‐ITD are adverse prognostic factors in pediatric APL patients. Age is an independent prognostic factor in APL patients (whole group). Despite higher high risk groups in pediatric patients, the outcome is better than adult patients. image
Developmental Heterogeneity of Microglia and Brain Myeloid Cells Revealed by Deep Single-Cell RNA Sequencing
Authors: Qingyun Li, Zuolin Cheng, Lu Zhou, Spyros Darmanis, Norma F. Neff, Jennifer Okamoto, Gunsagar Gulati, Mariko L. Bennett, Lu O. Sun, Laura E. Clarke, Julia Marschallinger, Guoqiang Yu, Stephen R. Quake, Tony Wyss-Coray, Ben A. Barres
Journal: Neuron (2018)
Molecularly distinct models of zebrafish Myc-induced B cell leukemia
Authors: Chiara Borga, Clay A. Foster, Sowmya Iyer, Sara P. Garcia, David M. Langenau, J. Kimble Frazer
Journal: Leukemia (2018)

Zebrafish models of T cell acute lymphoblastic leukemia (T-ALL) have been studied for over a decade, but curiously, robust zebrafish B cell ALL (B-ALL) models had not been described. Recently, our laboratories reported two seemingly closely-related models of zebrafish B-ALL. In these genetic lines, the primary difference is expression of either murine or human transgenic c-MYC, each controlled by the zebrafish rag2 promoter. Here, we compare ALL gene expression in both models. Surprisingly, we find that B-ALL arise in different B cell lineages, with ighm+ vs. ighz+ B-ALL driven by murine Myc vs. human MYC, respectively. Moreover, these B-ALL types exhibit signatures of distinct molecular pathways, further unexpected dissimilarity. Thus, despite sharing analogous genetic makeup, the ALL types in each model are markedly different, proving subtle genetic changes can profoundly impact model organism phenotypes. Investigating the mechanistic differences between mouse and human c-MYC in these contexts may reveal key functional aspects governing MYC-driven oncogenesis in human malignancies.
Optimizing complex phenotypes through model-guided multiplex genome engineering
Authors: Gleb Kuznetsov, Daniel B. Goodman, Gabriel T. Filsinger, Matthieu Landon, Nadin Rohland, John Aach, Marc J. Lajoie, George M. Church
Journal: Genome biology (2017)

We present a method for identifying genomic modifications that optimize a complex phenotype through multiplex genome engineering and predictive modeling. We apply our method to identify six single nucleotide mutations that recover 59% of the fitness defect exhibited by the 63-codon E. coli strain C321.∆A. By introducing targeted combinations of changes in multiplex we generate rich genotypic and phenotypic diversity and characterize clones using whole-genome sequencing and doubling time measurements. Regularized multivariate linear regression accurately quantifies individual allelic effects and overcomes bias from hitchhiking mutations and context-dependence of genome editing efficiency that would confound other strategies.
AB0648 CORRELATIONS BETWEEN NEUTROPHIL/LYMPHOCYTE RATIO AND CLINICAL CHARACTERISTICS OF PATIENTS WITH SYSTEMIC SCLEROSIS
Authors: Giovanna Cuomo, Francesco Masini, Klodian Gjeloshi, Francesco Guarino, Fiammetta Danzo, Luigi Elio Adinolfi, Ciro Romano
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background</h3> The ratio of neutrophils to lymphocytes (NRL) is considered a novel inflammatory marker. Recently studies are ongoing for objective and easy markers (1) <h3>Objectives</h3> In this study, we aimed to identify the possible relationship between NRL and clinical characteristics inn patients with systemic sclerosis (SSc) <h3>Methods</h3> Our study is based on a retrospective analysis. We collected in a data base clinic and epidemiological characteristic of SSc patients. Fifty-two patients (46 female; 10 subset diffuse) with SSc were enrolled consecutively for analyser the correlations. We considered the correlation with NRL and skin core, ulcers, pitting scars, gastro-intestinal events, fibrosis on HTCR, respiratory parameters (FVC, DLCO), PAPs, diastolic abnormalities, capillaroscopy alterations and activity index (2). Student's t-test was used for comparison averages, correlation among variables were assessed by Sperman's correlation testing. Results: sociodemographic and clinical characteristic were reported in table 1. The correlation analysis statistically significant is summarized in table 2: NLR vs skin score, DLCO, PAPs, and activity index. The correlation analysis between NLR and presence of ulcers and pitting scars was considered not quite significant. There were no correlations between other parameters and NLR. <h3>Conclusion</h3> NLR values correlated negatively with DLCO value, and positively with PAP value, skin score and activity index NLR level may serve as inflammatory marker in patients with SSc. <h3>References</h3> [1] absenger G. et al. Br J Cancer2013; 109:395 [2] Valentini G. et al. Ann Rheum Dids2001; 60:592 <h3>Disclosure of interests</h3> None declared
AB0706 DIFFERENCES IN RHEUMATOLOGISTS’ TREATMENT PREFERENCES FOR ANKYLOSING SPONDYLITIS AND NON- RADIOGRAPHIC AXIAL SPONDYLOARTHRITIS
Authors: Phil pouliot, Lynn Price
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background:</h3> The lack of a formal indication for Non-Radiographic axial Spondyloarthritis (nr-axSpA) has meant that many US rheumatologists fundamentally treat it as an early manifestation of ankylosing Spondylitis (AS) and seldom differentiate their treatment approach when using biologics/small molecules. <h3>Objectives:</h3> This study sought to gain a better understanding of the number of aS and nr-axSpA patients rheumatologists manage, recent changes made in treatment, and how aS and nr-axSpa patients are differentiated beyond radiographic evidence. The study also evaluated the differences in use of biologics/small molecules for the treatment of aS and nr-axSpA and rheumatologist projected changes in the management of these two conditions. <h3>Methods:</h3> An independent market analytics firm administered a survey with US rheumatologists (n=104) on the treatment landscape for aS and nr-axSpA. To qualify, rheumatologists had to spend the majority of their professional time in clinical practice, manage a minimum of 50 aS or nr-axSpA patients, and have been in practice between 2 and 35 years. Respondents answered questions pertaining to their practice, the demographics of their aS/nr-axSpA patients, their treatment approaches for each type, as well as attitudinal statements. The survey was administered online in February 2018 and respondents were compensated for their participation. A similar study was also conducted in 2017 allowing for year over year comparison. The data were collected and analyzed in SPSS. <h3>Results:</h3> Three-quarters of rheumatologists reported they treated their aS and nr-axSpA patients in a similar manner, though there was a higher use of NSAIDs and local steroids in non-radiographic disease, and more biologic/JAK use in aS. More established TNF inhibitors such as adalimumab, etanercept, and infliximab dominated biologics/small molecule preference, collectively accounting for approximately three-quarters of all biologic-treated aS and nr-axSpA patients. However, use of adalimumab, etanercept, and infliximab was expected to decline or stay flat in the next three months, while use of secukinumab, tofacitinib, golimumab IV, and both infliximab biosimilars, was anticipated to increase. Although not indicated for either condition, 28% of respondents reported off-label use of tofacitinib for aS, and 25% for nr-axSpA, a significant increase from 2017. Projected off-label use of tofacitinib in the following three months is also expected to increase. <h3>Conclusion:</h3> Rheumatologists are less likely to use biologics/small molecules for nr-axSpA, but when they do, use of brands is similar to those used in aS. While TNF inhibitors continue to account for the majority of biologic/small molecule prescriptions for these conditions, rheumatologists project increased use of agents with alternative mechanisms of action in the future. <h3>Disclosure of interests:</h3> None declared