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Nature rheumatology

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https://www.readbyqxmd.com/read/29048419/a-cross-sectional-quantitative-analysis-of-the-natural-history-of-farber-disease-an-ultra-orphan-condition-with-rheumatologic-and-neurological-cardinal-disease-features
#1
Matthias Zielonka, Sven F Garbade, Stefan Kölker, Georg F Hoffmann, Markus Ries
PurposeFarber disease (OMIM 22800) is an ultrarare progressive multisystemic neurodevelopmental storage disorder caused by a deficiency of the lysosomal enzyme acid ceramidase (AC). Hard clinical end points for future clinical trials remain to be defined.MethodsWe quantitatively analyzed published cases with Farber disease (N = 96). The main outcome variables were survival and diagnostic delay. As a potential predictor of survival, the influence of residual AC enzyme activity was investigated. The analysis was performed in compliance with STROBE criteria...
October 19, 2017: Genetics in Medicine: Official Journal of the American College of Medical Genetics
https://www.readbyqxmd.com/read/29042750/current-perspective-on-rituximab-in-rheumatic-diseases
#2
REVIEW
Tommaso Schioppo, Francesca Ingegnoli
The steadily increasing knowledge regarding pathogenetic mechanisms in autoimmune rheumatic diseases has paved the way to different therapeutic approaches. In particular, the market entry of biologics has dramatically modified the natural history of rheumatic chronic inflammatory diseases with a meaningful impact on patients' quality of life. Among the wide spectrum of available biological treatments, rituximab (RTX), first used in the treatment of non-Hodgkin's lymphoma, was later approved for rheumatoid arthritis and anti-neutrophil cytoplasmic antibodies-associated vasculitis...
2017: Drug Design, Development and Therapy
https://www.readbyqxmd.com/read/28983732/publishing-in-black-and-white-the-relevance-of-listing-of-scientific-journals
#3
REVIEW
Durga Prasanna Misra, Vinod Ravindran, Anupam Wakhlu, Aman Sharma, Vikas Agarwal, Vir Singh Negi
Scientific publishing, including in the field of Rheumatology, is evolving rapidly. Predatory journals are one of the major threats to contemporary publishing, especially to eager and naïve authors. In this narrative review, we discuss mechanisms that authors can employ to white list genuine scientific journals and blacklist "predatory" ones. Inclusion of a journal in reputed indices such as Medline (but not just Pubmed), Web of Science, Scopus or Embase raises the likelihood that the journal is genuine, more so if it is included in the current Journal Citation Reports...
November 2017: Rheumatology International
https://www.readbyqxmd.com/read/28977549/2016-acr-eular-adult-dermatomyositis-and-polymyositis-and-juvenile-dermatomyositis-response-criteria-methodological-aspects
#4
Lisa G Rider, Nicolino Ruperto, Angela Pistorio, Brian Erman, Nastaran Bayat, Peter A Lachenbruch, Howard Rockette, Brian M Feldman, Adam M Huber, Paul Hansen, Chester V Oddis, Ingrid E Lundberg, Anthony A Amato, Hector Chinoy, Robert G Cooper, Lorinda Chung, Katalin Danko, David Fiorentino, Ignacio García-De la Torre, Ann M Reed, Yeong Wook Song, Rolando Cimaz, Rubén J Cuttica, Clarissa A Pilkington, Alberto Martini, Janjaap van der Net, Susan Maillard, Frederick W Miller, Jiri Vencovsky, Rohit Aggarwal
Objective: The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM. Methods: Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions...
August 2, 2017: Rheumatology
https://www.readbyqxmd.com/read/28917219/reuma-pt-contribution-to-the-knowledge-of-immune-mediated-systemic-rheumatic-diseases
#5
Maria José Santos, Helena Canhão, Ana Filipa Mourão, Filipa Oliveira Ramos, Cristina Ponte, Cátia Duarte, Anabela Barcelos, Fernando Martins, José António Melo Gomes
Patient registries are key instruments aimed at a better understanding of the natural history of diseases, at assessing the effectiveness of therapeutic interventions, as well as identifying rare events or outcomes that are not captured in clinical trials. However, the potential of registries goes far beyond these aspects. For example, registries promote the standardization of clinical practice, can also provide information on domains that are not routinely collected in clinical practice and can support decision-making...
July 2017: Acta Reumatológica Portuguesa
https://www.readbyqxmd.com/read/28903545/the-road-from-development-to-approval-evaluating-the-body-of-evidence-to-confirm-biosimilarity
#6
REVIEW
Paul Declerck, Mourad Farouk Rezk
Biosimilars are products that contain a similar version of the active substance of an already authorized original biologic medicinal product (reference medicinal product). Their development requires special consideration, as similarity to the reference agent needs to be established through a comprehensive comparability exercise. Given the complex nature of these agents, minor structural differences may emerge, but the process of biosimilarity determination is designed to ascertain that the nature and impact of these differences are not clinically significant...
August 1, 2017: Rheumatology
https://www.readbyqxmd.com/read/28844116/cardiometabolic-disorders-in-psoriatic-disease
#7
REVIEW
Curtis Sobchak, Lihi Eder
PURPOSE OF REVIEW: Patients with psoriasis and psoriatic arthritis, collectively termed psoriatic disease (PsD), are at an increased risk of developing cardiovascular diseases (CVD). The purpose if this manuscript is to review recent evidence about the epidemiology and underlying mechanisms of CVD in psoriatic patients and approaches to improve the management of these comorbidities. RECENT FINDINGS: Studies have shown that CVD risk is independent of traditional cardiovascular risk factors and is related to the systemic inflammatory nature of PsD...
August 26, 2017: Current Rheumatology Reports
https://www.readbyqxmd.com/read/28830409/word2vec-inversion-and-traditional-text-classifiers-for-phenotyping-lupus
#8
Clayton A Turner, Alexander D Jacobs, Cassios K Marques, James C Oates, Diane L Kamen, Paul E Anderson, Jihad S Obeid
BACKGROUND: Identifying patients with certain clinical criteria based on manual chart review of doctors' notes is a daunting task given the massive amounts of text notes in the electronic health records (EHR). This task can be automated using text classifiers based on Natural Language Processing (NLP) techniques along with pattern recognition machine learning (ML) algorithms. The aim of this research is to evaluate the performance of traditional classifiers for identifying patients with Systemic Lupus Erythematosus (SLE) in comparison with a newer Bayesian word vector method...
August 22, 2017: BMC Medical Informatics and Decision Making
https://www.readbyqxmd.com/read/28770006/categorisation-of-foot-complaints-in-systemic-lupus-erythematosus-sle-from-a-new-zealand-cohort
#9
Simon J Otter, Maheswaran Rohan, Kevin A Davies, Sunil Kumar, Peter Gow, Nicola Dalbeth, Michael Corkill, Sam Panthakalam, Keith Rome
BACKGROUND: Foot complaints have been shown to be common in systemic lupus erythematosus (SLE) and heterogeneous in nature. We aimed to categorize self-reported foot complaints in people with SLE and foot symptoms. METHODS: A self-administered validated questionnaire was posted to 406 people with SLE attending adult rheumatology clinics across three health boards in Auckland, New Zealand. In addition to foot pain, vascular complaints, dermatological lesions and neurological symptoms were included in the analysis...
2017: Journal of Foot and Ankle Research
https://www.readbyqxmd.com/read/28721016/a-review-of-the-literature-analyzing-benefits-and-concerns-of-infliximab-biosimilar-ct-p13-for-the-treatment-of-rheumatologic-diseases-focus-on-interchangeability
#10
REVIEW
Andrea Becciolini, Maria Gabriella Raimondo, Chiara Crotti, Elena Agape, Martina Biggioggero, Ennio Giulio Favalli
The introduction of biological agents drastically changed the treatment paradigm of inflammatory arthritides, ameliorating the natural history of the diseases but concomitantly increasing the drug costs due to the manufacturing process. On this concern, biosimilar drugs may represent a valid option for reducing this elevated cost and increasing the availability of these highly effective treatments. Recently, CT-P13, the first biosimilar of infliximab, has been approved with the same indications established for the reference product (RP), and its daily use is progressively increasing...
2017: Drug Design, Development and Therapy
https://www.readbyqxmd.com/read/28694777/pharmacological-properties-and-molecular-mechanisms-of-thymol-prospects-for-its-therapeutic-potential-and-pharmaceutical-development
#11
REVIEW
Mohamed Fizur Nagoor Meeran, Hayate Javed, Hasan Al Taee, Sheikh Azimullah, Shreesh K Ojha
Thymol, chemically known as 2-isopropyl-5-methylphenol is a colorless crystalline monoterpene phenol. It is one of the most important dietary constituents in thyme species. For centuries, it has been used in traditional medicine and has been shown to possess various pharmacological properties including antioxidant, free radical scavenging, anti-inflammatory, analgesic, antispasmodic, antibacterial, antifungal, antiseptic and antitumor activities. The present article presents a detailed review of the scientific literature which reveals the pharmacological properties of thymol and its multiple therapeutic actions against various cardiovascular, neurological, rheumatological, gastrointestinal, metabolic and malignant diseases at both biochemical and molecular levels...
2017: Frontiers in Pharmacology
https://www.readbyqxmd.com/read/28588495/a-brief-history-of-il-1-and-il-1-ra-in-rheumatology
#12
Jean-Michel Dayer, Francesca Oliviero, Leonardo Punzi
The history of what, in 1979, was called interleukin-1 (IL-1), orchestrator of leukocyte inter-communication, began many years before then, initially by the observation of fever induction via the endogenous pyrogen (EP) (1974) and then in rheumatology on the role in tissue destruction in rheumatoid diseases via the induction of collagenase and PGE2 in human synovial cells by a mononuclear cell factor (MCF) (1977). Since then, the family has exploded to presently 11 members as well as many membrane-bound and soluble receptor forms...
2017: Frontiers in Pharmacology
https://www.readbyqxmd.com/read/28545782/autoimmune-encephalopathy-for-psychiatrists-when-to-suspect-autoimmunity-and-what-to-do-next
#13
REVIEW
Mark Oldham
OBJECTIVE: To provide a critical review of autoimmune encephalopathy-broadly defined as neuropsychiatric features directly related to an autoimmune process-relevant for psychiatric practice. METHODS: We consulted rheumatology textbooks to define the scope of autoimmune conditions and identified recent reviews of rheumatic conditions, autoimmune vasculitis, and autoimmune encephalitis. We integrated these with primary reports to provide a clinically relevant overview of autoimmune encephalopathy...
May 2017: Psychosomatics
https://www.readbyqxmd.com/read/28538014/precision-medicine-in-pediatric-rheumatology
#14
Joo Guan Yeo, Chin Teck Ng, Salvatore Albani
PURPOSE OF REVIEW: Precision medicine is the tailoring of medical care to subcategories of disease. In pediatric rheumatology, these subcategories must first be defined by their specific molecular immunological profiles, and then the effects of growth and puberty, developmental immunological changes, and differences in treatment options and adherence considered when designing therapeutic strategies. In the present review, we summarize the unmet needs in pediatric rheumatology before such precision medical care can be effectively delivered to affected patients...
September 2017: Current Opinion in Rheumatology
https://www.readbyqxmd.com/read/28516235/clinical-imaging-and-genotypical-features-of-three-deceased-and-five-surviving-cases-with-ada2-deficiency
#15
Sezgin Sahin, Amra Adrovic, Kenan Barut, Serdal Ugurlu, Eda Tahir Turanli, Huri Ozdogan, Ozgur Kasapcopur
Deficiency of adenosine deaminase type 2 (DADA2) is a rare form of autoinflammatory disorder with limited reported cases. In this paper, we have presented the clinico-immunological, radiological and genetic characteristics of five surviving and three deceased childhood-onset DADA2 patients. We aimed to compare surviving and deceased patients in terms of clinical features and treatment modalities. Moreover, we have evaluated the causes of death in our DADA2 subjects together with the previously reported cases...
May 17, 2017: Rheumatology International
https://www.readbyqxmd.com/read/28462539/correlation-of-functional-ability-of-the-hand-with-upper-limb-function-and-quality-of-life-in-patients-with-rheumatoid-arthritis
#16
Chhaya Verma, Ruchita Parikh, Milind Nadkar, Amita Mehta
BACKGROUND / OBJECTIVES: Rheumatoid arthritis (RA) is associated with significant pain and deformities, where individuals continue to perform activities with reduced abilities. Fatigue and functional disability ensues with the progressive nature of the disease. The study was designed to observe the correlation between the Functional Ability of the Hand with Upper Limb function and Quality of Life in patients with Rheumatoid Arthritis. METHODS: 75 patients diagnosed with RA who were classified in Class I, II, III as per the revised criteria for Classification of Functional Status as given by the American College of Rheumatology were asked to perform the Grip Ability Test to calculate the Hand function...
March 2017: Journal of the Association of Physicians of India
https://www.readbyqxmd.com/read/28410044/tackling-comorbidity-associated-with-rheumatic-diseases-in-standard-practice
#17
Deborah Palmer, Yasser El Miedany
In spite of the dramatic improvement of the long-term prognosis of inflammatory arthritic conditions, patients living with arthritis remain more likely to have a shorter lifespan in contrast to the age-matched population without arthritis. This high incidence of morbidity-mortality has been attributed to an increased prevalence of comorbidities, particularly cardiovascular disease, infections, and the development of malignant space-occupying lesions. In spite of the published guidelines highlighting the importance of comorbidity assessment and management, implementing these recommendations in standard clinical practice remains a challenge for the treating rheumatologists and rheumatology nurse specialists...
April 13, 2017: British Journal of Nursing: BJN
https://www.readbyqxmd.com/read/28386760/lung-transplantation-in-patients-with-systemic-sclerosis
#18
REVIEW
Rupal J Shah, Francesco Boin
PURPOSE OF REVIEW: The purpose of this study was to highlight recent findings on evaluation for lung transplantation and outcomes after transplantation in patients with systemic sclerosis (scleroderma). RECENT FINDINGS: The recognition that extra-pulmonary disease manifestations can seriously compromise post-transplant outcomes has changed the way patients are screened for lung transplant candidacy. Reluctance to transplant subjects affected by scleroderma has been driven by the complexity and multisystem nature of this disease...
May 2017: Current Rheumatology Reports
https://www.readbyqxmd.com/read/28385804/2016-american-college-of-rheumatology-european-league-against-rheumatism-criteria-for-minimal-moderate-and-major-clinical-response-in-juvenile-dermatomyositis-an-international-myositis-assessment-and-clinical-studies-group-paediatric-rheumatology-international
#19
Lisa G Rider, Rohit Aggarwal, Angela Pistorio, Nastaran Bayat, Brian Erman, Brian M Feldman, Adam M Huber, Rolando Cimaz, Rubén J Cuttica, Sheila Knupp de Oliveira, Carol B Lindsley, Clarissa A Pilkington, Marilynn Punaro, Angelo Ravelli, Ann M Reed, Kelly Rouster-Stevens, Annet van Royen-Kerkhof, Frank Dressler, Claudia Saad Magalhaes, Tamás Constantin, Joyce E Davidson, Bo Magnusson, Ricardo Russo, Luca Villa, Mariangela Rinaldi, Howard Rockette, Peter A Lachenbruch, Frederick W Miller, Jiri Vencovsky, Nicolino Ruperto
To develop response criteria for juvenile dermatomyositis (DM). We analysed the performance of 312 definitions that used core set measures from either the International Myositis Assessment and Clinical Studies Group (IMACS) or the Paediatric Rheumatology International Trials Organisation (PRINTO) and were derived from natural history data and a conjoint analysis survey. They were further validated using data from the PRINTO trial of prednisone alone compared to prednisone with methotrexate or cyclosporine and the Rituximab in Myositis (RIM) trial...
May 2017: Annals of the Rheumatic Diseases
https://www.readbyqxmd.com/read/28382778/2016-american-college-of-rheumatology-european-league-against-rheumatism-criteria-for-minimal-moderate-and-major-clinical-response-in-juvenile-dermatomyositis-an-international-myositis-assessment-and-clinical-studies-group-paediatric-rheumatology-international
#20
Lisa G Rider, Rohit Aggarwal, Angela Pistorio, Nastaran Bayat, Brian Erman, Brian M Feldman, Adam M Huber, Rolando Cimaz, Rubén J Cuttica, Sheila Knupp de Oliveira, Carol B Lindsley, Clarissa A Pilkington, Marilynn Punaro, Angelo Ravelli, Ann M Reed, Kelly Rouster-Stevens, Annet van Royen-Kerkhof, Frank Dressler, Claudia Saad Magalhaes, Tamás Constantin, Joyce E Davidson, Bo Magnusson, Ricardo Russo, Luca Villa, Mariangela Rinaldi, Howard Rockette, Peter A Lachenbruch, Frederick W Miller, Jiri Vencovsky, Nicolino Ruperto
OBJECTIVE: To develop response criteria for juvenile dermatomyositis (DM). METHODS: We analyzed the performance of 312 definitions that used core set measures from either the International Myositis Assessment and Clinical Studies Group (IMACS) or the Paediatric Rheumatology International Trials Organisation (PRINTO) and were derived from natural history data and a conjoint analysis survey. They were further validated using data from the PRINTO trial of prednisone alone compared to prednisone with methotrexate or cyclosporine and the Rituximab in Myositis (RIM) trial...
May 2017: Arthritis & Rheumatology
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