tbuckley/cabot-search huggingface dataset:
Abstract P275: Prescription of PCSK9 Inhibitors in the United States, by Patient, Provider, and Payer Characteristics, 2016
Authors: Matthew Ritchey, Stavros Tsipas, Angela Thompson-Paul, Kate Kirley, Fleetwood Loustalot, Gregory Wozniak
Journal: Circulation (2018)

Background: Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors were approved in 2015 for use by adults with familial hypercholesterolemia (FH) or clinical atherosclerotic cardiovascular disease (ASCVD) requiring additional low-density lipoprotein cholesterol lowering beyond use of diet modification and maximally tolerated statin therapy. This study describes the patient, provider and payer related characteristics of prescriptions filled for this new class of injectable medication during 2016. Methods: 2016 quarterly state-level prescription data were obtained from Symphony Health Solution’s PHAST 2.0. PHAST 2.0 includes data on over 90% of prescriptions filled from US retail and mail order pharmacies and, along with market purchasing data, are used to calculate state and national estimates. PCSK9 inhibitor fills among adults aged ≥18 years were characterized by quarter, patient age, provider type, and payer type. Per-capita fill rates across US states and total and patient spending per 30-day supply were calculated. Results: In 2016, 216,082 PCSK9 inhibitor prescriptions were filled. The number of fills per quarter increased from 20,348, in Q1, to 83,812, in Q4, and fill totals were greatest among those aged 65-74 years (39.3% of fills) (Figure). Cardiologists prescribed the majority of fills (60.5%). Commercial payers (43.3%) and Medicare (43.1%) were the most frequent payers. State per-capita fill rates (per 100,000) ranged from 12.1, in WY, to 191.9, in LA (median: 69.1). Total spending per 30-day supply was $951; patient spending was $58 per 30-day supply. Conclusion: Quarterly fills for PCSK9 inhibitors quadrupled during 2016, with substantial variation in per-capita fill rates across states. Cardiologists prescribed the majority of fills, which aligns with their indicated use among adults with FH or ASCVD. Total spending per 30-day supply was considerable. Tracking of prescribing trends for these medications is warranted as additional information about their efficacy becomes available.
Class II HLA Epitope Level Mismatch Can Predict Chronic Graft-Versus-Host Disease and Survival Following Haploidentical Transplant Using Post-Transplant Cyclophosphamide
Authors: Scott R Solomon, Michael T Aubrey, Cheri Anobile, Xu Zhang, Brian M Freed, Lawrence E Morris, H. Kent Holland, Melhem Solh, Asad Bashey
Journal: Blood (2018)
AB1015 Bone mineral density in patients with chronic kidney disease
Authors: V. Vasilkova, T. Mokhort, N. Filiptsova, A. Naumenka
Journal: Annals of the Rheumatic Diseases (2018)

<h3>Background</h3> Chronic kidney disease (CKD) is commonly associated with disorders of mineral and bone metabolism.<sup>1</sup> However, the relationship between renal function and bone mineral density (BMD) is controversial.<sup>2–3</sup> <h3>Objectives</h3> We aimed the relationship between markers of renal function and BMD in patients with CKD. <h3>Methods</h3> 95 patients both sexes with CKD aged 55.49±10.07 years were studied. Control group included 84 healthy subjects the same age. Standard laboratory analyses were performed in all patients. Renal function was assessed by the estimated glomerular filtration rate (eGFR), which was calculated using an equation based on creatinine (eGFRcr) and cystatin C (eGFRcys_c). Osteoporosis was defined as a femoral neck BMD T-score below −2.5. <h3>Results</h3> The serum cystatin C level was negatively correlated with BMD and T-score right and left femoral neck (p&lt;0.05), but not with BMD T-score lumber spine. The level of creatinine was negatively correlated only with BMD and T-score right femoral neck (r=−0.653, p&lt;0.05). Higher cystatin C levels were associated with a higher prevalence of osteoporosis in CKD patients (OR 3.54; 95% CI 1.63–7.85; p=0.002). In logistic regression analysis, after adjusting for age, body mass index, calcium, only cystatin C showed a negative correlation with femoral BMD. In addition, the eGFRcys_c showed a stronger positive correlation with femoral BMD than the eGFRcr. <h3>Conclusions</h3> Our findings suggest that serum cystatin C level could be a marker for femoral BMD and might help identify patients with osteoporosis who are susceptible to fractures. <h3>References</h3> [1] Relationship between bone mineral density and moderate to severe chronic kidney disease among general population in Korea. J Korean Med Sci. 2013;28(4):569–74. [2] Jorgetti V, dos Reis LM, Ott SM. Ethnic differences in bone and mineral metabolism in healthy people and patients with CKD. Kidney Int. 2014;85(6):1283–9. [3] Kong X, Tang L, Ma X, Liu W, Wang Z, Cui M, et al. Relationship between mild-to-moderate chronic kidney disease and decreased bone mineral density in Chinese adult population. Int Urol Nephrol. 2015;47(9):1547–53. <h3>Acknowledgements</h3> We acknowledged the help from the Republican Research Centre for Radiation Medicine and Human Ecology for the technical assistance. <h3>Disclosure of Interest</h3> None declared
FRI0405 A novel animal model for systemic sclerosis induced by immunisation of angiotensin ii receptor 1
Authors: X. Yue, F. Petersen, X. Wang, H. Heidecke, G. Riemekasten, X. Yu
Journal: Annals of the Rheumatic Diseases (2018)

<h3>Background</h3> Systemic sclerosis (SSc) is a complex connective tissue disease which is characterised by autoimmunity, vasculopathy and fibrosis<sup>1</sup>. Our recent study showed that the progression of SSc was strongly associated with the autoantibodies against angiotensin II receptor I (AT1R), suggesting a role of autoimmunity to AT1R in the pathogenesis of the disease <sup>2</sup> <h3>Objectives</h3> In this study, we aimed to investigate the role of AT1R in the pathogenesis of SSc in mice. <h3>Methods</h3> C57BL/6J mice were immunised with membrane extract (ME) of CHO cell overexpressing human AT1R or with ME of CHO cells as control. Serum, lung and skin samples were collected and assessed 63 days after immunisation for autoantibody production, inflammation and fibrosis, which are hallmarks for SSc. <h3>Results</h3> Immunisation with hAT1R induced the production of autoantibodies against the receptor in mice, and autoantibody deposition was found in the lung. Histologically, mice immunised with hAT1R showed a SSc-like disease, including perivascular infiltrates and fibrosis in the skin as well as pulmonary inflammation. The inflammation in the skin and the lung were characterised by infiltration of T- and B-cells. Furthermore, transfer of immune cells from hAT1R-immunised mice into C57BL/6J mice induced inflammation in the lung. <h3>Conclusions</h3> This study demonstrates that immunisation with hAT1R can induce a SSc-like disease, thus showing a pathogenic role of autoimmunity to AT1R and providng a novel mosue model for the diseases. Furthermore, this study also introduces a new immunisation strategy to generate functional autoantibodies against receptors on the cell membrane. <h3>References</h3> [1] LeRoy EC, Black C, Fleischmajer R, et al. Scleroderma (systemic sclerosis): classification, subsets and pathogenesis. J Rheumatol. 1988;15(2):202–205. [2] Riemekasten G, Philippe A, Nather M, et al. Involvement of functional autoantibodies against vascular receptors in systemic sclerosis. Ann Rheum Dis. 2011;70(3):530–536. <h3>Disclosure of Interest</h3> None declared
AB0452 Biological therapy adverse events in biobadaguay registry
Authors: M. Franco, Z. Morel, M.G. Avila-Pedretti, S. Cabrera-Villalba, I. Acosta Colman, P. Babak, P. Melgarejo, G. Elizaur, P. Delgadillo, D. Cordovilla Montero, J. Losanto, G. Babak, L. Román, E. Paredes, J. Mazzoleni, P. de Abreu
Journal: Annals of the Rheumatic Diseases (2018)

<h3>Background</h3> BIOBADAGUAY is the Paraguayan/Uruguayan registry of adverse events (AE) in patients with inflammatory rheumatic conditions under biologic therapy (BT) <h3>Objectives</h3> To determine the frequency and severity of AE in patients under BT from the BIOBADAGUAY registry <h3>Methods</h3> Prospective, observational study of undetermined length to verify the efficacy, safety and survival of the BT. The methodology applied is available at https//biobadaguay.ser.es. For the present study epidemiological and clinical variables, BT, type and severity of AE were analysed. The incidence rate (IR) was calculated as the total number of adverse events per 1000 patients/year and the incidence rate ratio (IRR) was analysed using the Poisson regression model. <h3>Results</h3> 778 BT were analysed (56.6% adalimumab, 23.7% etanercept, 9.6% tocilizumab, 5.7% rituximab, 3.5%infliximabm, 0.5%golimumab, 0.38%infliximab biosimilar, 0.13% abatacept). In these, 330 AE were observed, 256 (77.6%) mild and 74 (24.4%) severe. The global IR of AE was 143.9 (95% CI,128.8–160.8) and 32.6 (95% CI,25.3–40.5), 111.6 (95% CI, 98.4–126.2) for severe and mild respectively. Infection was the most frequent AE in 175 (53.0%) and 39 (22.3%) of them were severe. Infection’s IR was 76.3 (95% CI,65.4–88.5), 59.3 (95% CI,49.76–70.2) and 17.0 (95% CI,12.1–23.3) for global, severe and mild respectively. Out of the 39 severe AE, respiratory infections were the most frequent in 43.6% of the cases. 5 tuberculosis, 6 malignancies and 6 deaths were observed. When analysing the IR according to diagnosis, Idiopathic Juvenile Arthritis (JIA) was associated with a higher IR of global AE when comparing to the other diagnosis (IRR=2.3 [95% CI,1.6–3.4] p=4.27 × 10–6). RA diagnosis was significantly associated with a higher risk of severe AE (IRR=2.20 [95% CI,1.2–4.1] p=1.17 × 10–2). tocilizumab was significantly associated with a higher incidence of global AE, (IRR=2.69 [95% CI, 1.90–3.82] p=3.13 × 10–8) and severe ones (IRR=3.34 [95% CI,1.81–6.1] p=1.10 × 10–4). Adalimumab was significantly associated with a lower rate of global AE(IRR=0.6 [95% CI, 0.4–0.8] p=1.86 × 10–4). <h3>Conclusions</h3> AE were mild in general and infections were the most frequent. In the present study, it was found that JIA and treatment with tocilizumab presented a higher IRR of AE while RA presented a higher rate of severe AE. <h3>Disclosure of Interest</h3> None declared
Activation and Proliferation of PD-1+ Kidney Double-Negative T Cells Is Dependent on Nonclassical MHC Proteins and IL-2
Authors: Mohanraj Sadasivam, Sanjeev Noel, Sul A. Lee, Jing Gong, Mohamad E. Allaf, Phillip Pierorazio, Hamid Rabb, Abdel Rahim A. Hamad
Journal: Journal of the American Society of Nephrology (2019)

Significance Statement Understanding how each immune cell type functions in the kidney is necessary to develop new immunotherapies for cell-mediated kidney diseases, including transplant rejection, AKI, and GN. Although CD4 − CD8 − double-negative (DN) T cells are a significant kidney T cell subpopulation that is anti-inflammatory and protective during ischemic AKI, little is known about the role of various MHC class I and II molecules in regulating their function. In addition to showing that mouse kidney DN T cells have IL-2–dependent proliferation, the authors identified two subsets of kidney DNT cells: a classical MHC class I–dependent NK1.1 + subset and an MHC-independent programmed cell death protein-1 receptor (PD-1 + ) subset that is highly responsive to ischemia-reperfusion injury. The presence of both subsets in human kidneys suggests that these findings are clinically relevant. Background CD4 − CD8 − double-negative (DN) αβ T cells with innate-like properties represent a significant component of T cells in human and mouse kidneys. They spontaneously proliferate in the steady state and protect against ischemic AKI. However, the mechanisms regulating DN T cell homeostasis and responses to external danger signals from “sterile” inflammation remain poorly understood. Methods We used knockout mice, functional assays, and an established ischemic AKI model to investigate the role of various MHC class I and II molecules in regulating kidney DN T cells. We also studied human nephrectomy samples. Results Deficiency of β 2m-dependent MHC class I (but not MHC class II) molecules led to significant reduction in frequency or absolute numbers of kidney DN T cells due to impaired activation, proliferation, increased apoptosis, and loss of an NK1.1 + subset of DN T cells. The remaining DN T cells in β 2m knockout mice mainly comprised a programmed cell death protein-1 receptor (PD-1 + ) subset that depends on IL-2 provided by conventional T cells for optimal homeostasis. However, this PD-1 + subset remained highly responsive to changes in milieu, demonstrated by responses to infused lymphocytes. It was also the major responder to ischemic AKI; the NK1.1 + subset and CD8 + T cells had minimal responses. We found both DN T cell subsets in normal and cancerous human kidneys, indicating possible clinical relevance. Conclusions DN T cells, a unique population of kidney T cells, depend on nonclassical β 2m molecules for homeostasis and use MHC-independent mechanisms to respond to external stimuli. These results have important implications for understanding the role these cells play during AKI and other immune cell–mediated kidney diseases.
A Case of Pansclerotic Morphea Treated With Tocilizumab
Authors: April Zhang, James Nocton, Yvonne Chiu
Journal: JAMA Dermatology (2019)

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SAT0079 Treatment expectations influence both subjective and objective outcome parameters in patients with rheumatoid arthritis- a prospective cohort study
Authors: J. Mucke, R. Brinks, A. Dimitriou, J. Richter, M. Schneider
Journal: Annals of the Rheumatic Diseases (2018)

<h3>Background</h3> The prediction of individual response to treatment in rheumatoid arthritis (RA) is challenging and often limited. Here we evaluate the influence of patients' expectations and attitudes towards newly initiated disease-modifying anti-rheumatic drugs (DMARDs) on clinical outcome in RA. <h3>Methods</h3> 100 patients (74 female) with <sup>RA according to 2010</sup> ACR/EULAR classification criteria with upcoming change in DMARD treatment were included. Patients' treatment beliefs, health related quality of life, treatment expectations, and pain-related cognitions were measured using <i>the beliefs about medicines questionnaire</i> (BMQ), the SF-36, the <i>questionnaire about patient expectation</i> (PE), and the p<i>ain-related self-statement scale</i> (PRSS), respectively before treatment initiation (T0) and their DAS28-CRP was calculated at T0 and after 4 months (T4). Associations between patients' beliefs, expectations and their attitude according to the questionnaires and changes in DAS28-CRP between T0 and T4 were explored by regression analyses using the Aikaike information criterion. <h3>Results</h3> Regression analyses revealed that 42.2% of all variability in treatment response measured as a decline in DAS28-CRP (ΔDAS28) could be explained by expectations, psychological factors and laboratory parameters assessed with the applied questionnaires. Among these we identified the expected <i>improvement rate</i> with 23.4% as well as the patients' <i>fear of side effects</i> with 22.0% as the main predictors of ΔDAS28. The CRP-value at T0 accounted with 15% to the variability in ΔDAS28. Other highly influential factors were PRSS <i>catastrophizing</i> scale (10.7%), the BMQ <i>concern</i> scale (8.1%), other BMQ scales (7.9%) and medications' route of administration (8.0%). <h3>Conclusions</h3> The present study indicates a high impact of patients' expectations and their attitude towards new therapies on clinical response effecting both objective and subjective outcome parameters. Integration of individual patient's preferences and their expectations in treatment decisions and management can significantly increase treatment response. <h3>Disclosure of Interest</h3> None declared
Allogeneic Hematopoietic Cell Transplantation (HCT) Vs. Non-HCT Consolidation Therapies in Acute Myeloid Leukemia (AML) Patients 60-75 Years of Age in First Complete Remission (CR1): An Alliance (A151509), SWOG, ECOG-ACRIN and CIBMTR Study
Authors: Celalettin Ustun, Jennifer Le-Rademacher, Hai-Lin Wang, Megan Othus, Zhuoxin Sun, Brittny Major, Mei-Jie Zhang, Elizabeth Storrick, Jacqueline M Lafky, Selina Chow, Krzysztof Mrózek, Eyal C. Attar, Clara D. Bloomfield, Larry D Cripe, Richard A. Larson, Guido Marcucci, Gail J. Roboz, Geoffrey L. Uy, Richard M. Stone, Aminah Jatoi, Thomas C. Shea, Marcos de Lima, James M. Foran, Brenda M. Sandmaier, Mark R. Litzow, Harry P. Erba, Arti Hurria, Daniel J. Weisdorf, Andrew S Artz
Journal: Blood (2018)
Carfilzomib Thalidomide and Dexamethasone Is Safe and Effective in the Treatment of Relapsed/Refractory Multiple Myeloma: Preliminary Outcome from the Open Label Phase II ALLGMM018/AMN003 Study
Authors: Hang Quach, Simon J. Harrison, Slavisa Ninkovic, Jane Estell, Noemi Horvath, Nick Murphy, Richard Eek, Bradley Augustson, Philip Campbell, Rajeev Rajagopal, Akash Kalro, Robyn Hemme, Belinda Butcher, Tracey Gerber, Peter Mollee, Brian GM Durie, Wee-Joo Chng
Journal: Blood (2018)
SAT0506 Ssc in older age: frequent and with a different phenotype. data of the german network for systemic sclerosis
Authors: P. Moinzadeh, G. Riemekasten, N. Blank, J. Henes, I. Koetter, E. Siegert, C. Pfeiffer, G. Zeidler, M. Schmalzing, C. Guenther, L. Susok, M. Worm, A. Kreuter, C. Sunderkoetter, U. Mueller-Ladner, A. Juche, E. Aberer, T. Schmeiser, T. Krieg, K. Kuhr, N. Hunzelmann
Journal: Annals of the Rheumatic Diseases (2018)

<h3>Background</h3> Systemic sclerosis (SSc) is a very heterogeneous multisystem connective tissue disease. The majority of affected patients develop initial clinical symptoms between the age of 30 to 50 years. It is not known whether an ageing population affects the clinical phenotype of SSc. <h3>Objectives</h3> To investigate the relationship of the age at disease onset and clinical characteristics in SSc patients using the registry of the German Network for Systemic Scleroderma. <h3>Methods</h3> Clinical data of the patient registry, currently including 4021 patients, were evaluated. Three age ranges at disease onset (&lt;40 years, 40–60 years, and &gt;60 years) were correlated with clinical characteristics. <h3>Results</h3> Among all SSc patients, 27% of patients developed first non-raynaud symptoms at the age &lt;40 years, while 44% developed SSc between 40–60 years, and 29% were older than 60 years of age. In particular, SSc patients with disease onset &gt;60 years developed significantly (p&lt;0.001) more often the lcSSc subtype (71.1%), anti-centromere antibodies (45.8%), had a significantly lower modified Rodnan Skin Score (mRSS) (7.8±8.1), more often pulmonary hypertension (PH) (17.4%), a significantly lower mean DLCO level (69.7%±21.5) and less often digital ulcerations (20.2%). However, a significant difference for lung fibrosis, heart or kidney involvement could not be observed. The more progressive subsets (dcSSc and SSc overlap syndromes) are found significantly more often at younger ages (p&lt;0.001). <h3>Conclusions</h3> In this registry, nearly one third of patients developed SSc at an age above 60 years. These are mostly of the limited cutanous subtype with frequent PH. These findings have an important influence on recommendations on diagnosis and theray of SSC. <h3>Disclosure of Interest</h3> None declared
Light Chain Escape in Multiple Myeloma
Authors: Thomas Dejoie, Guillemette Fouquet, Cyrille Hulin, Murielle Roussel, Benjamin Hebraud, Lionel Karlin, Brigitte Kolb, Carla Araujo, Nathalie Meuleman, Mourad Tiab, Jean Valère Malfuson, Pascal Bourquard, Pascal Lenain, Arnaud Jaccard, Karim Belhadj, Gérard Lepeu, Marie Lorraine Chretien, Denis Caillot, Jean Fontan, Philippe Rodon, Anna Schmitt, Laurent Voillat, Sophie Cereja, Brigitte Pegourie, Frederique Kuhnowski, Sophie Rigaudeau, Olivier Decaux, Catherine Humbrecht-Kraut, Jamilé Frayfer, Laurent Garderet, Damien Roos-Weil, Jean Claude Eisenmann, Veronique Dorvaux, Eric G. Voog, Claire Mathiot, Jean-Paul Fermand, Thierry Facon, Hervé Avet-Loiseau, Jean Luc Harousseau, Philippe Moreau, Michel Attal, Xavier Leleu
Journal: Blood (2018)