tbuckley/cabot-search huggingface dataset:
Global services and support for children with developmental delays and disabilities: Bridging research and policy gaps
Authors: Pamela Y. Collins, Beverly Pringle, Charlee Alexander, Gary L. Darmstadt, Jody Heymann, Gillian Huebner, Vesna Kutlesic, Cheryl Polk, Lorraine Sherr, Andy Shih, Dragana Sretenov, Mariana Zindel
Journal: PLoS Medicine (2017)

Pamela Collins and colleagues explain the research and policy approaches needed globally to ensure children with developmental delays and disabilities are fully included in health and education services.
Whole blood gene expression and white matter Hyperintensities
Authors: Honghuang Lin, Claudia Satizabal, Zhijun Xie, Qiong Yang, Tianxiao Huan, Roby Joehanes, Chengping Wen, Peter J. Munson, Alexa Beiser, Daniel Levy, Sudha Seshadri
Journal: Molecular Neurodegeneration (2017)

White matter hyperintensities (WMH) are an important biomarker of cumulative vascular brain injury and have been associated with cognitive decline and an increased risk of dementia, stroke, depression, and gait impairments. The pathogenesis of white matter lesions however, remains uncertain. The characterization of gene expression profiles associated with WMH might help uncover molecular mechanisms underlying WMH.We performed a transcriptome-wide association study of gene expression profiles with WMH in 3248 participants from the Framingham Heart Study using the Affymetrix Human Exon 1.0 ST Array.We identified 13 genes that were significantly associated with WMH (FDR < 0.05) after adjusting for age, sex and blood cell components. Many of these genes are involved in inflammation-related pathways.Thirteen genes were significantly associated with WMH. Our study confirms the hypothesis that inflammation might be an important factor contributing to white matter lesions. Future work is needed to explore if these gene products might serve as potential therapeutic targets.
ENESTgoal Treatment-Free Remission Study: Updated Preliminary Results and Digital Polymerase Chain Reaction Analysis in Patients with Chronic Myeloid Leukemia in Chronic Phase Who Switched from Imatinib to Nilotinib
Authors: Ellen K. Ritchie, Rose Catchatourian, Rebecca B Klisovic, Javier Pinilla-Ibarz, Michael W. Deininger, Harry P. Erba, Jerald P. Radich, Michael R. Savona, Ilva Dautaj, Johannes Wolff, Michael J. Mauro
Journal: Blood (2016)
Investigating a biomarker‐driven approach to target collagen turnover in diabetic heart failure with preserved ejection fraction patients. Effect of torasemide versus furosemide on serum C‐terminal propeptide of procollagen type I (DROP‐PIP trial)
Authors: Tobias Daniel Trippel, Sophie Van Linthout, Dirk Westermann, Ruhdja Lindhorst, Anja Sandek, Stefanie Ernst, Anna Bobenko, Mario Kasner, Frank Spillmann, Arantxa González, Begoña López, Susana Ravassa, Burkert Pieske, Walter J. Paulus, Javier Díez, Frank Edelmann, Carsten Tschöpe
Journal: European Journal of Heart Failure (2017)
SAT0743-HPR Risk factors for fear of falling in patients with knee osteoarthritis
Authors: O Aydoğdu, Z Sarı, US Yurdalan, GM Polat
Journal: Annals of the Rheumatic Diseases (2017)

<h3>Background</h3> Severe knee osteoarthritis (OA) is characterized by stiffness, pain, and disability of the knee joint. Severe pain negatively affects muscle strength, coordination, postural stability, proprioception, mobility, and increases fall risk and possibly fear of falling. Individuals with severe knee OA experience loss of self-efficacy, activity avoidance, loss of self-confidence, and finally fear of falling without actually experiencing a fall (1). Despite the high prevalence of falls in patients with OA, few studies have investigated fear of falling in patients with knee OA (2). <h3>Objectives</h3> The present study was conducted to determine the risk factors affecting fear of falling and to investigate the relationship between fear of falling and age, gender, body mass index, pain level, range of motion, muscle strength, knee swelling, postural stability, and functional status in patients with knee OA. The purpose of our study was to evaluate the regression of fear of falling and identify its risk factors in patients with severe knee OA. <h3>Methods</h3> A total of 71 patients who were diagnosed with knee OA according to the American College of Rheumatology (ACR) and who were accepted as stage 2 and 3 based on the Kellgren-Lawrence criteria, were included in the study. Outcome measures included fear of falling, pain intensity, and range of motion, quadriceps and hamstring muscle strength, knee swelling, postural stability, and functional status. Pearson correlation and multiple logistic regression test was used to determine the risk factors of fear of falling and relationships between fear of falling and age, body mass index, pain intensity, range of motion, muscle strength, knee swelling, postural stability, and functional status. <h3>Results</h3> Multiple logistic regression analysis showed that age (odds ratio=12.5, p=0.011), body mass index (odds ratio=14.5, p=0.030), pain intensity (odds ratio=3.5, p=0.045), range of motion (odds ratio=9.8, p=0.012), and knee swelling (odds ratio=8.4, p=0.019) were independent risk factors for fear of falling among patients with knee OA. <h3>Conclusions</h3> We conclude that age, body mass index, pain intensity, range of motion and knee swelling influence the fear of falling. They are viewed as an important predictor of fear of falling in knee OA. Our results could be used to help select knee OA patients who should be enrolled in fall prevention programmes. <h3>References</h3> Tsonga T, Michalopoulou M, Kapetanakis S, et al. Risk factors for fear of falling in elderly patients with severe knee osteoarthritis before and one year after total knee arthroplasty. J Orthop Surg (Hong Kong). 2016 Dec;24(3):302–306. Levinger P, Menz HB, Wee E, Feller JA, Bartlett JR, Bergman NR. Physiological risk factors for falls in people with knee osteoarthritis before and early after knee replacement surgery. Knee Surg Sports Traumatol Arthrosc. 2011 Jul;19(7):1082–9. <h3>Disclosure of Interest</h3> None declared
AB0946 Calcinosis in children with juvenile dermatomyositis from a single-centre in north india
Authors: A Sharma, A Jindal, A Gupta, D Suri, S Singh
Journal: Annals of the Rheumatic Diseases (2017)

<h3>Background</h3> Juvenile dermatomyositis (JDM) is a rare childhood autoimmune inflammatory muscle disorder that can result in severe disability or death. Calcinosis is a unique and a poorly understood long-term complication of JDM (1). Calcinosis can present in various forms like nodular calcinosis, tumoral deposits, calcinosis universalis. <h3>Objectives</h3> We present here the images of calcinosis in children with JDM <h3>Methods</h3> All children diagnosed to have JDM and registered in Pediatric Rheumatology Clinic at Post Graduate Institute of Medical Education and Research, Chandigarh, India, were evaluated for presence of calcinosis. Consent was taken from patients or caregivers <h3>Results</h3> A total of 36 patients were evaluated. Twelve (33.33%) patients had calcinosis (Fig 1). Interestingly, 4 children had calcinosis at the time of diagnosis <h3>Conclusions</h3> Calcinosis is a distinct complication of JDM which is uncommon in inflammatory myopathies in adults (2). Calcinosis can be disabling and disfiguring. It may not be obviously visible in all patients and radiographs help reveal the extent of involvement. <h3>References</h3> Tansley SL, McHugh NJ, Wedderburn LR. Adult and juvenile dermatomyositis: are the distinct clinical features explained by our current understanding of serological subgroups and pathogenic mechanisms? Arthritis Res. Ther. 2013;15:211. Robinson AB, Reed AM. Clinical features, pathogenesis and treatment of juvenile and adult dermatomyositis. Nat. Rev. Rheumatol. 2011;7:664–75. <h3>Disclosure of Interest</h3> None declared
Altered Expression of Hematopoiesis Regulatory Genes in the Bone Marrow Mesenchymal Stem Cells of Patients of Aplastic Anemia
Authors: Soniya Nityanand, Naresh Kumar Tripathy, Chandra Prakash Chaturvedi, Ekta Minocha, Akhilesh Sharma, Khaliqur Rahman
Journal: Blood (2016)
SAT0259 High plasma concentration of mycophenolate acid in early phase of induction therapy predicts good renal outcome in lupus nephritis class iii or iv
Authors: T Kiyokawa, H Hanaoka, H Iida, K Ishimori, Y Takakuwa, T Okazaki, S Ozaki
Journal: Annals of the Rheumatic Diseases (2017)

<h3>Background</h3> Mychophenolate mofetil (MMF) is recommended as initial induction treatment for most cases of lupus nephritis (LN) class III-IV. Although the association between area under the concentration-versus-time curve (AUC) of myochophenolate acid (MPA) and therapeutic efficacy has been well shown in renal transplantation, it has been poorly investigated in LN. Furthermore, MMF interacts with multiple factors and its concentration may be decreased by high prednisolone (PSL) dose, low serum albumin level and low creatinine clearance. Since these factors dramatically change in induction phase of LN, the plasma concentration of MPA may also change by fixed dose of MMF administration. Here, we measured AUC<sub>0–12</sub> of MPA at different phases of induction treatment, early and middle, and prospectively investigated which concentration predicted future renal response in LN class III-IV. <h3>Objectives</h3> To investigate the relationship between the plasma concentration of MPA in early or middle phase of induction therapy and future renal response. <h3>Methods</h3> We prospectively enrolled patients with biopsy proven LN class III or IV who hospitalized from Apr to Oct 2016. As induction therapy, PSL was started at dose of 1mg/kg/day and tapered to 10mg/day by 12 weeks. Fixed dose of MMF at 2,000mg/day was continuously introduced. We measured 2 points of MPA plasma concentration depending on the phase of induction therapy, at early (week 2) and middle (week 12). We evaluated the association between these concentration and complete renal response (CR) rate at week 12. <h3>Results</h3> Six patients were enrolled. AUC<sub>0–12</sub> between the early and the middle phase was not correlated (R<sup>2</sup>=0.17, p=0.7), but that of the early phase tended to be lower (47.4±25.6 vs 58.9±19.1 mgh/L). All the patients with high AUC<sub>0–12</sub> (over 40mgh/L) at the early phase achieved CR at week 12 (Figure 1). But we could not find any association between AUC<sub>0–12</sub> at middle phase and CR rate at week 12. <h3>Conclusions</h3> High AUC<sub>0–12</sub> of MPA at the early phase of induction therapy might predict good renal response. <h3>Disclosure of Interest</h3> None declared
Incidence and predictive score for delayed hemolytic transfusion reaction in adult patients with sickle cell disease
Authors: David Narbey, Anoosha Habibi, Philippe Chadebech, Armand Mekontso‐Dessap, Mehdi Khellaf, Jean‐Daniel Lelièvre, Bertrand Godeau, Marc Michel, Frédéric Galactéros, Rachid Djoudi, Pablo Bartolucci, France Pirenne
Journal: American Journal of Hematology (2017)

Abstract Delayed hemolytic transfusion reaction (DHTR) is a life‐threatening complication of transfusion in sickle cell disease (SCD). The frequency of DHTR is underestimated because its symptoms mimic those of vaso‐occlusive crisis and antibodies (Abs) are often not detectable. No predictive factors for identifying patients likely to develop DHTR have yet been defined. We conducted a prospective single‐center observational study over 30 months in adult sickle cell patients. We included 694 transfusion episodes (TEs) in 311 patients, divided into occasional TEs (OTEs: 360) and chronic transfusion program (CTEs: 334). During follow‐up, 15 cases of DHTR were recorded, exclusively after OTEs. DHTR incidence was 4.2% per OTE (95% CI [2.6; 6.9]) and 6.8% per patient during the 30 months of the study (95% CI [4.2; 11.3]). We studied 11 additional DHTR cases, to construct a predictive score for DHTR. The DHTR mortality is high, 3 (11.5%) of the 26 DHTR patients died. The variables retained in the multivariate model were history of DHTR, number of units previously transfused and immunization status before transfusion. The resulting DHTR‐predictive score had an area under the ROC curve of 0.850 [95% CI: 0.780‐0.930], a negative‐predictive value of 98.4% and a positive‐predictive value of 50%. We report in our study population, for the first time, the incidence of DHTR, and, its occurrence exclusively in occasionally transfused patients. We also describe a simple score for predicting DHTR in patients undergoing occasional transfusion, to facilitate the management of blood transfusion in SCD patients.
AB1181 Local steiod and insulin injection in management of carpal tunnel syndrome; a comparative study
Authors: MH Abu-Zaid, S El morsy, SA Hablas
Journal: Annals of the Rheumatic Diseases (2017)

<h3>Background</h3> Carpal tunnel syndrome (CTS) is the most common focal nerve entrapment; local corticosteroid (CS) injection has been widely used to treat CTS. Recently; Local insulin Injection in CTS has been studied as insulin has anti-inflammatory anti-edematous effects. <h3>Objectives</h3> To compare the effectiveness of local steroid and insulin injections in management of CTS <h3>Methods</h3> Forty patients complaining of mild or moderate idiopathic CTS (diagnosed clinically &amp; electrophysiologically and classified according to American association of neuromuscular diagnostic medicine monograph) divided into 2 equal groups. Group I received two local injections of 10 IU NPH insulin into the affected carpal tunnel 2 weeks intervals. Group II: received single injection of triamcinolone acetonide (20 mg/0.5 ml) all procedures done after informed consent. Patients with severe or secondary type of CTS were excluded from this study. Evaluation of the patients was done at baseline, 2 and 4 months later clinically by measuring visual analogue scale (VAS), Phallen and compression tests and electrophysiologically by measuring motor and sensory nerve conduction studies of median and ulnar nerves using a standardized technique. <h3>Results</h3> There was significant improvement in all clinical parameters in both groups after 2 and 4 months from injection (p&lt;0.01) including VAS, Phallen and compression tests with more improvement was noticed in group I. Also there was significant improvement in electrophysiological parameters such as distal motor latency (DML) and distal sensory velocity of median nerve 2 and 4 months after treatment (p&lt;0.01).However the improvement was more in group I but the difference between results in both groups were statistically nonsignificant. <h3>Conclusions</h3> Local insulin injection is as effective as (or even better than) local steroid injection in management of CTS <h3>References</h3> Sun Q, Li J, Gao F. New insights into insulin: The anti-inflammatory effect and its clinical relevance. World J Diabetes. 2014 Apr 15; 5(2): 89–96. Ginanneschi F, Filippou G, Bonifazi M, Frediani B, Rossi A. Effects of Local Corticosteroid Injection on Electrical Properties of Aβ-Fibers in Carpal Tunnel Syndrome. J Mol Neurosci, 2014; 52:525–530. Ashraf A, Yazdani A, Mirshams S. Evaluation of the effectiveness of local insulin injection in type 2 diabetic patients with CTS. Electromyogr Clin Neurophysiol. 2009 May-Jun;49(4):161–6. <h3>Acknowledgements</h3> Thanks a lot to Dr. Samar Abd Alhamed for her great efforts in completing statistical analysis in this study. <h3>Disclosure of Interest</h3> None declared
Bioengineering of Artificial Antigen Presenting Cells and Lymphoid Organs
Authors: Chao Wang, Wujin Sun, Yanqi Ye, Hunter N. Bomba, Zhen Gu
Journal: Theranostics (2017)

The immune system protects the body against a wide range of infectious diseases and cancer by leveraging the efficiency of immune cells and lymphoid organs.Over the past decade, immune cell/organ therapies based on the manipulation, infusion, and implantation of autologous or allogeneic immune cells/organs into patients have been widely tested and have made great progress in clinical applications.Despite these advances, therapy with natural immune cells or lymphoid organs is relatively expensive and time-consuming.Alternatively, biomimetic materials and strategies have been applied to develop artificial immune cells and lymphoid organs, which have attracted considerable attentions.In this review, we survey the latest studies on engineering biomimetic materials for immunotherapy, focusing on the perspectives of bioengineering artificial antigen presenting cells and lymphoid organs.The opportunities and challenges of this field are also discussed.
AB0880 Pharmacodynamic effects and safety of verinurad (RDEA3170) in combination with allopurinol versus allopurinol alone in adults with gout: a phase 2a, open-label study
Authors: R Fleischmann, P Winkle, JN Miner, X Yan, L Hicks, S Valdez, J Hall, M Hernandez-Illas
Journal: Annals of the Rheumatic Diseases (2017)

<h3>Background</h3> Verinurad (RDEA3170) is a high-affinity, selective URAT1 inhibitor in development for the treatment of gout and asymptomatic hyperuricemia. <h3>Objectives</h3> This Phase 2a, randomized, open-label, multicenter study investigated the multiple-dose pharmacodynamics (PD), pharmacokinetics (PK), and safety of oral verinurad in combination with allopurinol versus allopurinol alone in adults with gout (NCT02498652). <h3>Methods</h3> Patients aged ≥18 and ≤75 years with gout and serum uric acid (sUA) ≥8 mg/dL were randomized to 1 of 2 cohorts to receive allopurinol (300 mg) in combination with verinurad (dose range 2.5 mg to 20 mg) and allopurinol 300 mg or 600 mg alone (each treatment period was 7 days). Medications were administered once daily ∼30 min after breakfast (for allopurinol 300 mg b.i.d. group, the second allopurinol dose was in the evening). Colchicine 0.6 mg for gout flare prophylaxis was initiated at approximately Day -14 (start of urate-lowering therapy [ULT]) washout) or Day -7 if not on ULT. Serial blood and urine samples were measured on Days -1, 1, 7, 14, 21, 28, and 35 for PD and PK endpoints. Safety assessments included adverse events (AEs) and laboratory, electrocardiogram, and vital sign parameters. <h3>Results</h3> Forty-one patients were randomized (n=20–21 per cohort). Serum PD data pooled across cohorts demonstrated maximal % decrease in sUA from baseline (Emax)at 6–10 h after verinurad and allopurinol combination treatment. Addition of verinurad (2.5 mg to 20 mg) to allopurinol decreased sUA in dose-dependent manner (Figure). Greater sUA reductions were observed for dose combinations of verinurad ≥5 mg with allopurinol 300 mg versus allopurinol 600 mg alone, while allopurinol 600 mg once daily was equivalent to allopurinol 300 b.i.d. Emax was 46.9%, 58.9%, 59.9%, 67.1%, 68.4%, and 74.3% for verinurad at doses of 2.5, 5, 7.5, 10, 15, and 20 mg in combination with allopurinol 300 mg, versus 39.7%, 53.8%, and 54.4% with allopurinol 300 mg, allopurinol 600 mg, and allopurinol 300 mg b.i.d. alone. No drug-drug interaction on verinurad and allopurinol plasma PK parameters was observed. <h3>Conclusions</h3> Verinurad coadministered with allopurinol dose-dependently decreased sUA. All dose combinations of verinurad and allopurinol in this study were generally well tolerated with no serious AEs or renal-related events during combination treatment. <h3>Disclosure of Interest</h3> R. Fleischmann Grant/research support from: Ardea Biosciences, Inc., P. Winkle Employee of: Anaheim Clinical Trials, J. Miner Employee of: Ardea Biosciences, Inc., X. Yan Employee of: Ardea Biosciences, Inc., L. Hicks Employee of: Ardea Biosciences, Inc., S. Valdez Employee of: Ardea Biosciences, Inc., J. Hall Employee of: Ardea Biosciences, Inc., M. Hernandez-Illas: None declared
Note on the Cystine Oxidase Activity in Normal and Neoplastic Tissues of the Mouse
Authors: Jesse P. Greenstein, Florence M. Leuthardt
Journal: JNCI Journal of the National Cancer Institute (1944)

Journal Article Note on the Cystine Oxidase Activity in Normal and Neoplastic Tissues of the Mouse Get access Jesse P. Greenstein, Jesse P. Greenstein senior biochemist National Cancer Institute, National Institute of Health, United States Public Health Service Search for other works by this author on: Oxford Academic PubMed Google Scholar Florence M. Leuthardt Florence M. Leuthardt laboratory technician National Cancer Institute, National Institute of Health, United States Public Health Service Search for other works by this author on: Oxford Academic PubMed Google Scholar JNCI: Journal of the National Cancer Institute, Volume 5, Issue 1, August 1944, Pages 39–40, https://doi.org/10.1093/jnci/5.1.39 Published: 01 August 1944
KIR2DS2 recognizes conserved peptides derived from viral helicases in the context of HLA-C
Authors: Mohammed M. Naiyer, Sorcha A. Cassidy, Andrea Magri, Vanessa Cowton, Kevin Chen, Salah Mansour, Hariklia Kranidioti, Berenice Mbiribindi, Pauline Rettman, Scott Harris, Liam J. Fanning, Arend Mulder, Franz H. J. Claas, Andrew D. Davidson, Arvind H. Patel, Marco A. Purbhoo, Salim I. Khakoo
Journal: Science Immunology (2017)

KIR2DS2, an activating natural killer cell receptor, recognizes conserved peptides derived from the RNA helicases of pathogenic flaviviruses.
SAT0387 “do not do” recommendations in the management of comorbidity in patients with axial spondyloarthritis. gecoax project
Authors: JC Torre-Alonso, C González, S Castañeda, R Curbelo, E Collantes, MV Hernández, A Urruticoechea-Arana, JC Nieto, J García, MΆ Abad, J Ramírez, C Suárez, R Dalmau, MD Martín-Arranz, L Leόn, JC Hermosa, JC Obaya, T Otόn, L Carmona
Journal: Annals of the Rheumatic Diseases (2017)

<h3>Background</h3> During the development of recommendations and implementation aids of the GECOAx project, the importance of avoiding certain situations was highlighted. <h3>Objectives</h3> To recognize what prescriptions, risk assessments, or preventive strategies are wrong practices and should thus be avoided in clinical practice. To establish not to do recommendations in the management of the comorbidity of AxSpA. <h3>Methods</h3> A multidisciplinary group was selected [10 rheumatologists, 1 internist, 1 cardiologist, 1 gastroenterologist, 1 psychologist and 2 family physicians]. With the support of 3 methodologists, and after interactions aimed to edit a document for the management of comorbidity launched by the same panel, a list of Not to do recommendations was issued. In a discussion meeting, evidence was provided to support the recommendations, items without sufficient basis were removed, and the final list was produced. <h3>Results</h3> A summary list of Not to do recommendations (Table 1) was issued. <h3>Conclusions</h3> These recommendations aim to avoid making common mistakes in clinical practice and to help better management of frequent comorbidity in patients with AxSpA. <h3>Acknowledgements</h3> This project was funded by Merck Sharp &amp; Dohme Spain. Merck Sharp &amp; Dohme had no influence on either the development of the project or the final content of the manuscript. <h3>Disclosure of Interest</h3> None declared
Stem Cells: Controlled Exposure of Bioactive Growth Factor in 3D Amyloid Hydrogel for Stem Cells Differentiation (Adv. Healthcare Mater. 18/2017)
Authors: Subhadeep Das, Rakesh Kumar, Narendra Nath Jha, Samir K. Maji
Journal: Advanced Healthcare Materials (2017)

Samir K. Maji and co-workers present an amyloid hydrogel for sustained growth factors delivery in tissue engineering applications in article number 1700368. The image represents stem cells (blue spheres) embedded within the amyloid hydrogel matrix (grey network) encapsulating growth factors (green dots). Within 3D culture, stem cells self-organizes into spheroids (red sphere) with up-regulated neural genes. In three-dimensional cell culture, the entrapped growth factors within hydrogel and the surface property of amyloids promoted stem cells into neuro-sphere formation and higher neuronal gene expression.