tbuckley/cabot-search huggingface dataset:
FRI0313 The efficacy and safety of tocilizumab therapy in patients with polymyalgia rheumatica who were resistant or intolerant to glucocorticoids and additional methotrexate
Authors: A Ueno, Y Yamamura, K Fujita, N Shibutou, M Yamamura
Journal: Annals of the Rheumatic Diseases (2017)

<h3>Background</h3> A recent trial of tocilizumab (TCZ) in patients with newly diagnosed polymyalgia rheumatica (PMR), conducted in Europe and the United States, has shown its efficacy and safety. <h3>Objectives</h3> We examined the efficacy and safety of TCZ for patients with PMR who had been primarily resistant or intolerant to glucocorticoids (GC) and additional methotrexate (MTX). <h3>Methods</h3> Sixty patients had been diagnosed with having PMR since 2011. The patients are all compatible with the 2012 EULAR/ACR provisional classification criteria for PMR, and had been treated first with GC and then, if they were resistant or intolerant to GC, were added MTX, similarly to the 2015 EULAR/ACR recommendations for the management of PMR. The disease activity were measured by PMR-AS. <h3>Results</h3> There were 17 patients with GC/MTX resistant or intolerant PMR (28%). Of them, 9 patients with PMR agreed to the proposal of TCZ addition, and their therapeutic responses to TCZ and its safety were determined. They were at the age of 68.2±10.6, including three males and six females. Before TCZ addition, the patients were treated with prednisolone (PSL) at 7.6±3.0 mg/day plus MTX at 7.1±5.1 mg/week, and serum CRP were at 1.0±1.0 mg/dL. After 8.4±5.7 months of TCZ treatment, PSL and MTX had been reduced to 1.1±1.3 mg/day and 3.3±4.5 mg/week, respectively, with CRP at 0.02±0 mg/dL. GC were able to be withdrawn in 5 patients, and MTX were further withdrawn in 4 patients. Two patients reached drug-free remission (PMR-AS=0.02). During TCZ therapy, each one patient showed the worsening of depression and occlusion of the central retinal vein. <h3>Conclusions</h3> These results indicate that TCZ may provide a therapeutic option for patients with severe PMR who were resistant or intolerant to GC and additional MTX. <h3>Disclosure of Interest</h3> None declared
FRI0732-HPR The PSS-QOL: development and first psychometric testing of a new patient-reported outcome measure for pss patients
Authors: A Lackner, J Hermann, J Unger, T Stamm, M Stradner, W Graninger, C Dejaco
Journal: Annals of the Rheumatic Diseases (2017)

<h3>Background</h3> Patients with primary Sjögren Syndrome (PSS) are affected by glandular and extraglandular manifestations leading to physical and psychological impairment. To what extent these factors affect the health related quality of life (HRQL) of these patients is largely unexplored. Disease activity scores for PSS have been developed but there is no disease-specific HRQL questionnaire available so far. <h3>Objectives</h3> To develop a questionnaire for the assessment of HRQL in PSS. <h3>Methods</h3> In a previous qualitative study, concepts related to HRQL in PSS were identified by focus-group interviews with PSS patients. Based on these concepts, a questionnaire (PSS-QoL) was developed focusing on two main topics: physical (pain and dryness) and psychosocial dimension. The first draft of this questionnaire was evaluated by semi-structured interviews with PSS patients (n=6) and rheumatologists (n=4). Based on their feedback, a revised questionnaire was constructed and re-evaluated by the patients and physicians. Subsequently, psychometric testing of PSS-QoL was performed in 75 PSS patients of the outpatient clinic of the Medical University Graz. For testing of internal consistency Crohnbach9s α was used. Convergent construct validity was tested by correlating the scores with the ESSPRI and the EQ-5D. Reliability was examined by asking patients who considered themselves to be in a stable disease to complete the questionnaire 1–2 weeks apart. In addition, an English version of PSS-QoL was was developed using a standard methodology for translation. <h3>Results</h3> Out of the 75 PSS patients, 91% were female, disease duration was 4.8±4.08 years and age of patients was 58.5±12.5 years.The internal consistency of the PSS-QoL showed a Crohnbach9s α of 0.892 and we found a moderate correlation of the PSS-QoL with the ESSPRI (Corr<sub>coeff</sub>=0.625) and the EQ-5D (EQ5D-pain/discomfort; corr<sub>coeff</sub>=0.531). A second assessment was performed after 1–2 weeks in 21 patients with stable disease. The ICC for PSS-QoL was 0.958 (95% CI 0.926 to 0.981). In comparison, the ICC for EQ-5D in this population was 0.854 (95% CI 0.735 to 0.933). Subsequently, the final German version of PSS-QoL was translated forward and back into English by native speakers. <h3>Conclusions</h3> A questionnaire to assess the HRQL in PSS patients has been developed and tested for its psychometric properties. The PSS-QoL should allow for a better and more comprehensive assessment on patients9 HRQL in PSS. Multicentre studies for further validation are needed. <h3>Disclosure of Interest</h3> None declared
AB1200-HPR The reliability of romanian roland morris disability questionnaire in people with low back pain. a preliminary study
Authors: E Ilie, L Rusu
Journal: Annals of the Rheumatic Diseases (2017)

<h3>Background</h3> Patient reported assessments are widely recommended by the clinical guidelines and an important part of a comprehensive assessment. Patient-reported questionnaires should be translated into local language accordingly and validated in the translated language (1). The clinicians in Romania suffer from lack of validated questionnaires in Romanian language. The Roland Morris Disability questionnaire is one of the most recommended questionnaires evaluating low back pain. Although, the Romanian translation was performed before, there is no effort regarding to its validation in Romanian language until now. <h3>Objectives</h3> To investigate the test-retest reliability Romanian version of the Roland Morris Disability Questionnaire (RMDQ-Ro) in people low back pain. <h3>Methods</h3> The permission to conduct such a study was asked to the original author Prof. Martin Roland, before starting to the study. The Romanian translated form of Roland Morris Disability Questionnaire which is provided on the web site "http://www.rmdq.org/" was used in the study. A total of 100 people with low back pain filled the RMDQ as well as Numeric Rating Scales for rest (NRS-R) and activity (NRS-A). Due to the lack of other validated measures such the Oswestry Disability Index in Romanian, these additional assessments (VAS-R and VAS-A) were performed for investigating convergent validity. For test-retest reliability 30 people filled the RMDQ-Ro after 3–14 days later as recommended. Test-rest reliability was assessed with intra-class coefficient correlation (ICC). Non-parametric tests were employed due to the heterogeneity of the data. Therefore, the Spearman correlation test was used for determining the relationship between RMDQ-Ro and VAS-R and VAS-A. <h3>Results</h3> The characteristics of the participants were shown at Table 1. The test-retest reliability of RMDQ-Ro was found at an excellent level (ICC: 0.95). Moderate positive correlations were determined between RMDQ-Ro and NRS-R (rho: 0.518, p&lt;0.001), and NRS-A (rho: 0.484, p&lt;0.001). <h3>Conclusions</h3> RMDQ-Ro was found reliable regarding test-retest reliability. This questionnaire can be performed in the repeated measures for evaluating low back pain patients. However, more psychometric characteristics of RMDQ-Ro such as internal consistency and construct validity should be investigated in further studies. <h3>References</h3> Beaton DE, Bombardier C, Guillemin F, Ferraz MB.Guidelines for the process of cross-cultural adaptation of self-report measures. Spine (Phila Pa 1976). 2000;25(24):3186–91. <h3>Disclosure of Interest</h3> None declared
Frequent Amplification of a chr19q13.41 MicroRNA Polycistron in Aggressive Primitive Neuroectodermal Brain Tumors
Authors: Meihua Li, Kyle F. Lee, Yuntao Lu, Ian Clarke, David Shih, Charles Eberhart, V. Peter Collins, Tim Van Meter, Daniel Picard, Limei Zhou, Paul Boutros, Piergiorgio Modena, Muh-Lii Liang, Steve W. Scherer, Eric Bouffet, James T. Rutka, Scott Pomeroy, Ching C. Lau, Michael D. Taylor, Amar Gajjar, Peter B. Dirks, Cynthia E. Hawkins, Annie Huang
Journal: Cancer Cell (2010)
High-dose methotrexate therapy significantly improved survival of adult acute lymphoblastic leukemia: a phase III study by JALSG
Authors: T Sakura, F Hayakawa, I Sugiura, T Murayama, K Imai, N Usui, S Fujisawa, T Yamauchi, T Yujiri, K Kakihana, Y Ito, H Kanamori, Y Ueda, Y Miyata, M Kurokawa, N Asou, K Ohnishi, S Ohtake, Y Kobayashi, K Matsuo, H Kiyoi, Y Miyazaki, T Naoe
Journal: Leukemia (2017)
SP0106 The lung as a driver of ra-associated autoimmunity
Authors: A Catrina
Journal: Annals of the Rheumatic Diseases (2017)

Rheumatoid arthritis (RA) is a chronic inflammatory disease resulting from the complex interaction between genes and environment. In a large majority of patients this interaction leads to formation of disease-specific anti-citrullinated proteins antibodies (ACPA). Systemic autoimmunity may be triggered at mucosal sites (such as the lungs) long before the first signs of inflammation are starting in the joints. According to this model, smoking (and others environmental triggers) induces subclinical inflammation in the lungs, leading to increased local citrullination and formation of ACPA in genetically susceptible individuals. Lung changes on high-resolution computer tomography are present in both early-untreated ACPA positive RA and ACPA positive individuals at risk for but not yet having disease. Further, signs of subclinical inflammation and immune activation with germinal centers formation and ACPA enrichment is present in early untreated RA. Shared citrullinated targets have been described in the lungs and joints of patients with RA and more recent data unravels novel mechanisms showing how this extra-articular triggered autoimmunity progresses to joint-specific inflammation. Beside an initiating role, the lung might also be a secondary target for antibodies, especially in longstanding seropositive RA. <h3>Disclosure of Interest</h3> None declared
Abstract 3028: Preserved Cardiac Sympathetic Nerve Innervation Contributes to Left Ventricular Reverse Remodeling After Acute Myocardial Infarction: Analysis by MIBG Imaging
Authors: Kensuke Matsumoto, Teishi Kajiya, Takatoshi Hayashi, Yoshihiro Ikeda, Shinichirou Yamada, Kouhei Yamashiro, Kazuo Mizutani, Sachiyo Iwata, Katsunori Okajima, Masami Yoshida, Mitsuo Kinugasa, Naoki Murai
Journal: Circulation (2006)

Purpose : The left ventricular(LV) remodeling process, beginning soon after the onset of acute myocardial infarction(AMI) and lasting over the year, leads to progressive ventricular enlargement and dysfunction, which associates with very bad prognosis of patients with AMI. And many studies have focused on the determinants of LV remodeling after AMI, it has been implied that the cardiac sympathetic nervous system may play an important role in this process. On the other hand, although left ventricular enlargement and dysfunction has present in acute phase after AMI, there are many patients, whose LV function has improve and LV dimension has reduce at chronic phase; reverse remodeling. But the determinant factor of reverse remodeling process has not fully investigated. So we used I(123)-MIBG imaging, to investigate whether cardiac sympathetic nerve function could contribute to this processes.   Methods : We studied consecutive 44 patients with AMI who received reperfusion therapy at our hospital. We underwent sequential Tc(99m)-myocardial ECG-gated SPECT at acute and chronic phase, and I(123)-MIBG imaging at chronic phase. From the LV diastolic volume (EDV) change between acute and chronic phase, we divided patients into 2 groups; Reverse remodeling group(N=23, EDV decreased>10%)and Unchanged or remodeling group(N=21, EDV decreased ≤ 10% or EDV increased).   Results:  There were not any differences in baseline characteristics among both groups. Perfusion defect score among both groups at acute phase (16.2±12.3 vs. 22.2±16.4, respectively P   Conclusions:  Most of patients with reverse LV remodeling exhibited preserved sympathetic nervous integrity. Therefore preserved cardiac sympathetic nerve innervation (including reinnervation) may play an important role in reverse remodeling processes after AMI.
FRI0286 Serological evolution in patients with thrombotic antiphospholipid syndrome
Authors: L Riancho-Zarrabeitia, S García-Canale, M Cubería, G Daroca, M Agudo, M Lόpez-Hoyos, P Muñoz, V Martínez-Taboada
Journal: Annals of the Rheumatic Diseases (2017)

<h3>Background</h3> Antiphospholipid syndrome (APS) is an autoinmune disease characterized by the presence of antiphospholipid antibodies (aPL) and at least one clinical event (thrombosis and /or pregnancy morbidities). The titers of aPL can fluctuate and eventually become negative. This negativization, particularly if persistent, may be associated with a lower frequency of clinical events. <h3>Objectives</h3> To describe the clinical and serological course of patients with thrombotic APS as well as the factors related with the aPL negativization <h3>Methods</h3> We performed a retrospective study including patients attended at the Rheumatology clinic from a tertiary hospital in Northern Spain. We included 94 patients with thrombotic APS according to Sidney criteria of 2006. They were classified according to the serological evolution as persistently negative aPL, transiently positive, and persistently positive aPL according to previously established criteria. <h3>Results</h3> After a mean follow-up of 145±56 months, 48.9% of patients presented a persistently negative serology, whereas in 12.8% it was transiently positive, and persistently positive in 38.3%. When analyzing potential factors related to the negativization (table 1), we found that patients with positive lupus anticoagulant tended to have a persistently negative serology during follow-up, but it did not reach statistical significance (OR 2.7; 95% CI 0.8–9.4; p=0.145). We found no association between traditional cardiovascular risk factors or previous treatments and the serological evolution. <h3>Conclusions</h3> After a mean follow-up of 12 years, 49% of thrombotic APS patients presented a persistently negative serology. We found no significant association between immunological, traditional cardiovascular risk factors or previous treatments and the persistently negative serology. <h3>Disclosure of Interest</h3> None declared
The Case for Universal Screening of Private Well Water Quality in the U.S. and Testing Requirements to Achieve It: Evidence from Arsenic
Authors: Yan Zheng, Sara V. Flanagan
Journal: Environmental Health Perspectives (2017)

Background: The 1974 Safe Drinking Water Act (SDWA) regulates &gt;170,000 public water systems to protect health, but not &gt;13 million private wells. State and local government requirements for private well water testing are rare and inconsistent; the responsibility to ensure water safety remains with individual households. Over the last two decades, geogenic arsenic has emerged as a significant public health concern due to high prevalence in many rural American communities. Objectives: We build the case for universal screening of private well water quality around arsenic, the most toxic and widespread of common private water contaminants. We argue that achieving universal screening will require policy intervention, and that testing should be made easy, accessible, and in many cases free to all private well households in the United States, considering the invisible, tasteless, odorless, and thus silent nature of arsenic. Discussion: Our research has identified behavioral, situational and financial barriers to households managing their own well water safety, resulting in far from universal screening despite traditional public health outreach efforts. We observe significant socioeconomic disparities in arsenic testing and treatment when private water is unregulated. Testing requirements can be a partial answer to these challenges. Conclusions: Universal screening, achieved through local testing requirements complemented by greater community engagement targeting biologically and socioeconomically vulnerable groups, would reduce population arsenic exposure greater than any promotional efforts to date. Universal screening of private well water will identify the dangers hidden in America’s drinking water supply and redirect attention to ensure safe water among affected households. https://doi.org/10.1289/EHP629
SP0085 Patient reported outcomes
Authors: M Schneider
Journal: Annals of the Rheumatic Diseases (2017)

In SLE as in other rheumatic diseases, the most relevant target of intervention should be a status with controlled disease process assuring no further accrual of damage. If actual expert discussions like DORIS define the frame of such a status, clinical activity measured by a validated lupus disease activity instrument, serologic activity and therapy – because of harm - are the dimensions of remission with its duration as additional factor for outcome. Patient reported outcomes (PROs) were not included. Otherwise, if payers and reimbursement system decide about relevance, patient outcomes are clear of highest importance as target. Looking on the evidence of PROs for outcome in SLE, PROs were never used as primary endpoint in clinical trials. In RCTs, PROs were often collected and mostly explorative analysed. There is no evidence that PROs can validly define the above described status of controlled disease. But from systematic analyses in RA, we know that pure PRO like VAS of general health status and semi PRO like tender joints are at least as relevant as more "objective" criteria like swollen joints or CRP as clearly exhibited by the ACR/Eular remission criteria for RA. The challenge in SLE is that the discrepancies between patients9 and physicians9 perception and perspectives are even more distinct than in RA. Sometimes, there is the expression that physicians and patients are describing different diseases. The burden of illness in lupus is better defined by pain than by organ manifestations; the overall survival in SLE is more related to lupus nephritis than to fatigue. It is obvious that physicians should analyse the actual clinical symptoms and integrate the future consequences of their actual management in their decision, and patients are more focused on release of their actual burden. Until today, these different and divers perspectives are no integrated, neither in RCTs nor in daily care. But such integration is mandatory, because no side imagines the complete picture of lupus, which may also produce to the poor results of clinical trials. In routine care, this behaviour causes frustration and mental distress, optimal results are prohibited. So, the answer to what is more relevant in the management of SLE patients - clinical targets, biological targets or PROs – is the integration of all important aspects of lupus. This implies more than the statistical evaluation of the best items of all three aspects, it is the active involvement of patients in their care: patient empowerment in SLE, a fruitful process, in which both sides have to learn a lot from and about each other. <h3>Disclosure of Interest</h3> M. Schneider Shareholder of: no, Grant/research support from: GSK; UCB, Consultant for: Astra-Zeneca; GSK; Lilly; Roche; Suppremol; UCB, Employee of: no, Paid instructor for: no, Speakers bureau: Astra-Zeneca; GSK; Roche; Pfizer; UCB
SP0134 INTERLEUKIN-2 therapy in sle
Authors: JY Humrich
Journal: Annals of the Rheumatic Diseases (2017)

There is an unmet need for more effective and selective therapeutics in severe autoimmune diseases such as systemic lupus erythematosus (SLE). A deeper understanding of the pathogenic mechanisms in the past has led to the clinical translation of low-dose interleukin-2 (IL-2) therapy which primarily aims to restore the activity of regulatory T cells. First results from phase I/II studies are promising by proving the selective expansion of regulatory T cells <i>in vivo</i> and by providing first evidence for the clinical efficacy of low-dose IL-2 therapy in SLE. Here we will summarize key findings which led to the development of this novel therapeutic concept and will highlight the main rationales for the clinical translation of low-dose IL-2 therapy in SLE. <h3>Disclosure of Interest</h3> None declared
SAT0669 How do we use biologics in patients with a history of malignancy? an assessment of treatment patterns using scandinavian registers
Authors: K Chatzidionysiou, K Aaltonen, D Nordström, B Gudbjornsson, G Grondal, AJ Geirsson, L Steingrimsdottir, T Frisell, J Askling
Journal: Annals of the Rheumatic Diseases (2017)

<h3>Background</h3> Immune competence is of importance for the occurrence and outcome of malignancies. Robust data on risk of relapse of previous cancer following treatment with biological immune-modulators are scarce. Most treatment guidelines caution about their use in patients with a history of cancer, leaving rheumatologists with the decision whether a potential treatment benefit may offset any potential risk of cancer relapse. <h3>Objectives</h3> To assess the overall use of biologics and the relative use of different biological drugs in RA patients with a history of cancer. <h3>Methods</h3> As part of a Nordic collaboration, and using data from the ARTIS (Sweden), ROB-FIN (Finland), and ICEBIO (Iceland) biologics registers, we identified all patients with RA who initiated a first ever biological treatment 2010 through 2014. Through linkage to the national cancer registers, we identified those patients who had a history of any invasive malignancy (including squamous cell skin cancer) either within the five years preceding start of biological treatment ("recent history of malignancy") or more than five years before start of biological treatment ("non-recent history malignancy). <h3>Results</h3> The age- and gender distributions were similar across countries and drugs. Initiators of non-TNFi biologics were older than TNFI-initiators; the median age at start was the highest for rituximab. Out of a total of 8065 bio-initiations, 6% occurred in individuals with a history of cancer (2% with a cancer within 5 years, and 4% with a cancer more than 5 years before treatment start. Whereas there was little variation (around 5%) across TNFi initiators, the proportion of patients with a history of cancer at treatment start was higher among rituximab initiators, in part explained by age (Table). There were only small variations across country (not shown). <h3>Conclusions</h3> In Sweden, Finland and Iceland, one out of 20 biologics-initiators (and almost one out of five rituximab initiators) have a history of an invasive cancer, underscoring the need for more data on benefit/risks in this treatment context. The higher proportion in rituximab initiators is partly explained by differences in age at treatment start and reflects the preference for rituximab by clinicians for treatment of patients with history of cancer. <h3>Disclosure of Interest</h3> K. Chatzidionysiou Consultant for: Roche, Pfizer, Abbvie, Elli-Lilly, UCB, K. Aaltonen: None declared, D. Nordström Speakers bureau: AbbVie, BMS, Lilly, MSD, Novartis, Pfizer, Roche, UCB, B. Gudbjornsson Speakers bureau: Actavis, Celgene, MSD, Pfizer, G. Grondal: None declared, A. Geirsson: None declared, L. Steingrimsdottir: None declared, T. Frisell: None declared, J. Askling Grant/research support from: AbbVie, Eli Lilly, Janssen, Merck, Pfizer, Roche, UCB, Samsung