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1
Frost & Sullivan., ed. U.S. autoimmune disease therapeutic product markets: Disease-modifying companies heading toward regulatory approval. Mountain View, CA: Frost & Sullivan, 1994.
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2
Nie, Pei Huey, and David L. Sultzer. Treatments for Neurocognitive Disorders. Oxford University Press, 2015. http://dx.doi.org/10.1093/med:psych/9780199342211.003.0026.
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Dementia, or neurocognitive disorders, refers to a number of clinical syndromes originating in brain pathology and characterized by cognitive deficits and functional impairment. This chapter provides an update on treatment options in addition to a brief summary of dementia types and an overview of the diagnostic criteria for cognitive disorders. The diagnosis of dementia is ultimately a clinical one and includes a multidimensional perspective; as such, treatment requires a comprehensive approach. This chapter addresses two aspects of the treatment of neurocognitive disorders: pharmacological interventions that can temporarily slow the decline of cognitive deficits and the management of behavioral and psychological symptoms (neuropsychiatric symptoms) associated with dementia syndromes. The chapter also reviews disease-modifying treatments in development that may beneficially alter the course of disease, or reduce or prevent symptom expression in those at risk.
3
Tsai, Po-Heng. Cognitive Enhancers for Alzheimer’s Disease. Oxford University Press, 2017. http://dx.doi.org/10.1093/med/9780190214401.003.0003.
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Alzheimer’s disease (AD) is the most common cause of dementia. In the United States, an estimated 5.3 million people had AD dementia in 2015, including 200,000 individuals younger than age 65 years. The number of people who are affected by AD is projected to reach 16 million in 2050. There is a tremendous cost associated with caring for people with AD. In 2015, the direct costs to US society of caring for those with AD totaled an estimated $226 billion, and if no effective disease-modifying treatments become available, this could increase to $1.1 trillion in 2050. In addition to medical costs, in 2014, caregivers of people with AD and other dementias provided an estimated 17.9 billion hours of unpaid assistance, which translates to a value of $217.7 billion. Therefore, cognitive enhancers for AD by improving cognition could address symptoms associated with AD, reduce caregiver burden, and limit health care costs.
4
Paganoni, Sabrina, and Nazem Atassi. Upper Motor Neuron Disorders Hereditary Spastic Paraplegia and Primary Lateral Sclerosis. Oxford University Press, 2017. http://dx.doi.org/10.1093/med/9780199937837.003.0032.
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Upper motor neuron (UMN) syndromes are a group of rare, degenerative neurological disorders that are classified as either hereditary spastic paraplegia (HSP) or primary lateral sclerosis (PLS). Our understanding of their underlying pathophysiology is unfortunately very limited and has been a significant barrier to the development of disease-modifying treatments. Recent advances in genetics and in vitro and in vivo disease modeling have provided new insights into disease mechanisms and hold the promise to lead to the future development of mechanism-based therapies.
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Rho, Jong M. Overview. Edited by Jong M. Rho. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780190497996.003.0011.
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After nearly a century of clinical use, the ketogenic diet is firmly established as an efficacious treatment for medically intractable epilepsy. Intriguingly, there is growing experimental evidence that the ketogenic diet and its metabolites also render neuroprotective and potentially disease-modifying effects. Hence, dietary and metabolic therapies have been attempted in a variety of neurological disorders other than epilepsy, including brain cancer, cognitive disorders, autism, neurotrauma, pain, and multiple sclerosis. This section, “Ketogenic Diet: Emerging Clinical Applications and Future Potential,” explores the current preclinical and clinical evidence for metabolism-based treatments designed to counter the myriad disease processes seen in many neurological conditions. Specific attention has been given to the effects of the ketogenic diet in malignant brain cancer, autism spectrum disorder, Alzheimer’s disease, traumatic brain and spinal cord injury, pain, and multiple sclerosis.
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Hopkins, Philip M. Musculoskeletal disorders and anaesthesia. Edited by Philip M. Hopkins. Oxford University Press, 2017. http://dx.doi.org/10.1093/med/9780199642045.003.0080.
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This chapter covers the anaesthetic implications of the polyarthropathies, connective tissue diseases, and primary myopathies. There are generic considerations for management of patients with joint and muscle disease but many of these conditions have multisystem involvement, the nature of which varies between the individual members of each class. Anaesthetic management of the polyarthropathies requires knowledge of the adverse effects of disease-modifying drugs and new biological treatments. Advances in genetic diagnosis of inherited myopathies aid the identification of potential hazards of anaesthesia in these challenging patients.
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Chan, Jonathan, and Nigil Haroon. Treatment: NSAIDs. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780198734444.003.0020.
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Non-steroidal anti-inflammatory drugs (NSAIDs) constitute a diverse group of medications that inhibit prostaglandin synthesis. NSAIDs form the first-line pharmacological therapy in ankylosing spondylitis (AS). A number of randomized controlled trials (RCTs) support the efficacy of NSAIDs in reducing pain and improving patient function. Head-to-head comparisons have demonstrated equivalent effect of different NSAIDs in symptom control. The proposed disease-modifying potential of regular NSAID therapy is debatable and recent literature provides evidence to the contrary. Several safety concerns have been raised regarding long-term use of NSAIDs, especially an increase in cardiovascular risk. This chapter discusses the pharmacology, efficacy in treatment of AS, disease-modifying potential, and safety concerns of NSAIDs.
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Poddubnyy, Denis, and Hildrun Haibel. Treatment: DMARDs. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780198734444.003.0021.
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In axial spondyloarthritis (axSpA) there is little evidence to support use of classical synthetic disease-modifying antirheumatic drugs (DMARDs), with the majority of studies performed in advanced ankylosing spondylitis. Sulfasalazine is the best investigated DMARD in axSpA. Its positive clinical effect, if any, seems to be more prominent in the presence of peripheral arthritis, although a certain proportion of patients with axial disease might benefit from sulfasalazine therapy. Available data indicate that there is no evidence that methotrexate might be effective in axial disease, and only marginal evidence exists in support of methotrexate use in case of peripheral involvement. No true disease-modifying properties (e.g. retardation of structural damage progression in the spine) have been demonstrated for DMARDs in axSpA to date. Efficacy of a combination therapy (e.g. methotrexate plus sulfasalazine) as well as benefits of methotrexate (or other DMARDs) in addition to tumour necrosis factor α inhibitors in axSpA remain uncertain.
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Morgan, David. Immunotherapy for Neurodegenerative Disorders. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780190233563.003.0017.
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Neurodegenerative diseases are a growing health concern through the world as gains in longevity result in an increased population at risk of these age-related disorders. Unfortunately no disease modifying treatments exist for these disorders. Over the last three decades enormous insights have been gained into the causes of these disorders. One approach to treating these diseases is to direct immunotherapy against the misfolded proteins that accumulate within the brains of those with neurodegenerative disease in an attempt to clear the accumulating proteins and slow or prevent expression of the disease. This chapter summarizes the recent (and frustrating) experience with anti-Aβ immunotherapy to treat mild to moderate Alzheimer’s disease, and holds hope that newer generation antibodies and treating presymptomatic disease will have greater impact. In addition, it reviews the preclinical data regarding approaches to treating tau, synuclein, and prion disorders, all of which demonstrate consistent effects in mice and cultured neurons.
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Cummings, Jeffrey L., and Jagan A. Pillai. Neurodegenerative Diseases. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780190233563.003.0001.
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Neurodegenerative diseases (NDDs) are growing in frequency and represent a major threat to public health. Advances in scientific progress have made it clear that NDDs share many underlying processes, including shared intracellular mechanisms such as protein misfolding and aggregation, cell-to-cell prion-like spread, growth factor signaling abnormalities, RNA and DNA disturbances, glial cell changes, and neuronal loss. Transmitter deficits are shared across many types of disorders. Means of studying NDDs with human iPS cells and transgenic models are similar. The progression of NDDs through asymptomatic, prodromal, and manifest stages is shared across disorders. Clinical features of NDDs, including cognitive impairment, disease progression, age-related effects, terminal stages, neuropsychiatric manifestations, and functional disorders and disability, have many common elements. Clinical trials, biomarkers, brain imaging, and regulatory aspects of NDD can share information across NDDs. Disease-modifying and transmitter-based therapeutic interventions, clinical trials, and regulatory approaches to treatments for NDDs are also similar.
11
Braun, Juergen, and Irene E. van der Horst-Bruinsma. Treatment: biologics. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780198734444.003.0022.
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According to classification criteria, the spectrum of spondyloarthritis (SpA) covers axial SpA (axSpA), which includes non-radiographic axSpA and ankylosing spondylitis, and peripheral SpA, which overlaps with psoriatic arthritis. Management recommendations for many forms of SpA have been recently published. Treatment of patients with axSpA with active disease starts with a sufficient dose of non-steroidal anti-inflammatory drugs (NSAIDs) for at least 4 weeks and preferably longer, in combination with exercise. In case of peripheral SpA, several disease-modifying antirheumatic drugs can be given, but these are not efficacious in axial disease. In case of insufficient response to NSAIDs for axSpA, biologic treatment can be added. The biologics most commonly used are TNF-blocking agents, but some other biologic agents seem to be beneficial in axial diseases as well, such as secukinumab (an IL-17 blocker). However, these new drugs have not yet been approved for axSpA at the time of writing this chapter.
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Ghaemi, Nassir. Clinical Psychopharmacology. Oxford University Press, 2018. http://dx.doi.org/10.1093/med/9780199995486.001.0001.
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Clinical Psychopharmacology offers a comprehensive guide to clinical practice that explores the science and art of clinical research and its individualized application. Content is primarily based on clinical research and pharmacological studies, unlike most texts that rely on inferences from biological mechanisms. The text consists of 49 chapters, organized into 6 sections, focusing on disease-modifying versus symptomatic effects of available treatments, careful differential diagnosis including non-DSM diagnostic concepts, key clinical research studies, essential facts about the most common drugs, and more. Four appendices address key diagnostic controversies. This innovative book advocates a scientific and humanistic approach to practice and examines not only the benefits, but also the harms of psychotropic drugs. Providing a solid foundation of knowledge and a great deal of practical information, this book is a valuable resource for psychiatrists, nurse practitioners, medical students, trainees in psychiatry, pharmacists, and other mental health professionals.
13
Scolding, Neil. Paraneoplastic disorders and neuroimmunology. Oxford University Press, 2011. http://dx.doi.org/10.1093/med/9780198569381.003.0895.
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The extraordinary expansion in the field of neuroimmunology witnessed in the last decade is not just in the number of neurological disorders now considered to have an immune basis, nor the depth of understanding of disorders long known to be ‘neuroimmune’. Nor is it in the number of antibodies discovered and now testable, nor in the range of new immune suppressant or modifying treatments now emerging or already available. It is of course all of these things, but it is also more than the sum of these parts. What we are currently privileged to witness is the coming together of immunological understanding, the neurobiology of disease, and rational immune therapy, or at least the beginning of this process. To take one isolated example, neurogenetics and neurophysiology taught us about the clinical consequences of channel disruption; laboratory-based neuroimmunology showed antibodies to be capable of producing comparable acquired disease; and it seems likely that specific anti-B-cell humanized monoclonal antibodies offer the therapeutic potential to remove these channel-disrupting antibodies. Neither of these steps could be described in the last edition, and one can imagine similar dramatic changes will emerge before the next.
14
Bhargava, Pavan, and Peter A. Calabresi. Multiple Sclerosis. Oxford University Press, 2017. http://dx.doi.org/10.1093/med/9780199937837.003.0087.
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Multiple sclerosis is a chronic demyelinating neurological disorder of the brain and spinal cord, with both inflammatory and degenerative components. Current treatment strategies utilize immunomodulatory and immunosuppressive agents to reduce the inflammatory disease activity and retard accumulation of disability. Future challenges for treatment include identifying agents that will promote remyelination and axonal protection to help impact progressive forms of multiple sclerosis. This chapter discusses currently available disease modifying therapies, agents currently in phase 2/3 trials, and future directions in the treatment of multiple sclerosis.
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Jicha, Gregory A., and Frederick A. Schmitt. Alzheimer’s Disease. Oxford University Press, 2017. http://dx.doi.org/10.1093/med/9780199937837.003.0017.
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Advances in the current management and treatment of Alzheimer’s disease have grown directly from our increased understanding of the neurobiology underlying this disease. Currently available pharmacologic and nonpharmacologic treatment strategies remain focused on symptomatic management of disease rather than disease modification. Despite a wealth of evidence supporting the clinical benefits of existing therapies in the management of symptomatic progression, there is limited evidence that these available therapies modify disease progression over the course of dementia progression. More recent research discoveries in the areas of genetics, molecular and cell biology, and environmental risk factors have become the focal point for an explosive growth in experimental disease-modifying strategies designed to prevent, slow, or potentially halt the progression of Alzheimer’s disease.
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Walsh, Richard A. “Are My Children at Risk, Doctor?”. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780190607555.003.0007.
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Few patients with Parkinson’s disease will be found to have a monogenic cause of their disease, even among those with early onset symptoms. The identification of a genetic etiology will in general have no implications on management but can be helpful in offering a prediction on prognosis, predicting response to treatment, and allowing genetic counseling to take place. Insights into the molecular mechanisms provided by a greater understanding of the genetics of Parkinson’s disease may offer the best hope for identifying future disease-modifying therapies. Next-generation sequencing techniques offer a more cost-effective approach to pursuing genetic testing where indicated.
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Giesser, Barbara S. Relapses, Immunosuppressive and Novel Therapies. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780199341016.003.0026.
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This chapter presents information about treatment of acute attacks of multiple sclerosis and identification of precipitating factors. Additionally, it includes a detailed discussion of immunosuppressive agents that are used (largely off label) as disease-modifying therapies and data that support their use. Many clinical trials are currently focused on testing agents for use in progressive disease as well as agents that may be neuroprotective or neurorestorative. Results of recent trials of several of these various new and experimental agents are also reviewed.
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Ellenstein, Aviva, Christina Prather, and Mikhail Kogan. Neurodegenerative Diseases: Parkinson’s and Alzheimer’s Diseases. Oxford University Press, 2018. http://dx.doi.org/10.1093/med/9780190466268.003.0020.
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Neurodegenerative diseases increase in prevalence with aging. This chapter begins with a discussion of Parkinson’s disease. Optimally individualized treatment includes dopaminergic medications, physiotherapy, and multidisciplinary care. Evidence for integrative approaches is limited. Advances in genetics and biomarkers hold promise for subtype-specific, precision treatment in the near future. The second part of this chapter focuses on Alzheimer’s disease. Standard evaluation includes assessment for possible contributing factors that may worsen cognition, and management includes optimizing factors that may improve cognitive function. No disease-modifying medical approaches yet exist, but increasing emphasis on interventions to limit chronic inflammation and optimize brain metabolism remain fundamental in the integrative approach to Alzheimer’s disease. The new metabolic approach first described by Dr. Dale Bredesen is summarized and the importance of multidisciplinary care, with emphasis on early transition to palliative care when appropriate, is reviewed.
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Stewart, Robert. Vascular and mixed dementias. Oxford University Press, 2013. http://dx.doi.org/10.1093/med/9780199644957.003.0034.
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Vascular disease is the most important environmental risk factor for dementia but this research area has been hampered by inadequate outcome definitions – in particular, a diagnostic system that attempts to separate overlapping and probably interacting pathologies. There is now substantial evidence that the well-recognised risk factors for cardiovascular disease and stroke are also risk factors for dementia, including Alzheimer’s disease. However, these risk factors frequently act over several decades, meaning that the chances of definitive randomised controlled trial evidence for risk-modifying interventions are slim. This should not obscure the wide opportunity for delaying or preventing dementia through risk factor control and uncontroversial healthy lifestyles. Care should also be taken that comorbid cerebrovascular disease is not considered as excluding a diagnosis of Alzheimer’s disease, particularly now that this determines treatment eligibility.
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Behrens, Frank, Michaela Koehm, and Michael J. Parnham. Synthetic DMARDs. Oxford University Press, 2018. http://dx.doi.org/10.1093/med/9780198737582.003.0028.
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Synthetic disease modifying anti-rheumatic drugs (sDMARDs) are first line systemic treatment options for management of active psoriatic arthritis (PsA). Most of the compounds are used based on evidence from clinical trials in rheumatoid arthritis and from experience in routine care. Methotrexate is often recommended as the first choice within among sDMARDs, despite controversial or missing evidence of efficacy, according to experience over many years. Leflunomide, sulfasalazine, and cyclosporin are additional established options to treat PsA. The recently approved PDE4 inhibitor, apremilast, has broad evidence of efficacy. All these drugs have different strengths and weaknesses and varying levels of evidence for treatment of PsA. Therefore, the best treatment choice must be based on the different individual manifestations of the disease and comorbid conditions. In this chapter, the evidence levels for the different clinical symptoms are listed and suggestions for a hierarchical order of choice are given, adapted to likely clinical scenarios.
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Coyle, Patricia K. Immune-mediated Disorders of the Central Nervous System. Edited by Emma Ciafaloni, Cheryl Bushnell, and Loralei L. Thornburg. Oxford University Press, 2018. http://dx.doi.org/10.1093/med/9780190667351.003.0010.
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This chapter reviews pregnancy in multiple sclerosis (MS), neuromyelitis optica spectrum disorder (NMOSD), and acute transverse myelitis (ATM) syndrome. MS is a major acquired disease of young adults, with a rising female predominance. MS has no direct negative consequences on fertility or pregnancy. Pregnancy has a profound effect on MS, with decrease in disease activity during the last trimester counteracted by a three-month postpartum increase in disease activity. With the development of disease-modifying therapies, important questions arise about washout periods, the feasibility and risks of treating during pregnancy and breastfeeding, and the potential of treatment-related adverse fetal effects. Fortunately, there is good information to counsel women with MS. Neuromyelitis Optica Spectrum Disorder (NMOSD) is a neuroimmune channelopathy. It is a distinct disorder from MS. NMOSD disease activity is not favorably affected by pregnancy. The postpartum period has real risk for disabling attacks. This influences recommendations about breastfeeding and how quickly to resume therapy postpartum. Acute transverse myelitis (ATM) syndrome can occur in both MS and NMOSD but can also be due to other disorders. Workup and treatment of ATM during pregnancy is reviewed, as well as implications for delivery.
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Bissell, Lesley-Anne, Dwomoa Adu, and Paul Emery. The patient with rheumatoid arthritis, mixed connective tissue disease, Sjögren syndrome, or polymyositis. Edited by Giuseppe Remuzzi. Oxford University Press, 2015. http://dx.doi.org/10.1093/med/9780199592548.003.0166.
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Renal disease is a well-recognized cause of ill health and death in rheumatoid arthritis. Three broad categories of renal disease occur. The first—and by far the most common—arises from the nephrotoxicity of the drugs used in the treatment of arthritis, particularly with non-steroidal anti-inflammatory drugs. Disease-modifying antirheumatic drugs such as gold and D-penicillamine may lead to proteinuria and a glomerulonephritis in 10–30% of patients. Ciclosporin is associated with significant nephrotoxicity and hypertension. A second major but diminishing cause of renal disease in rheumatoid arthritis is amyloidosis. Thirdly, rheumatoid arthritis may be associated with the development of glomerulonephritis. The main types described are a mesangial proliferative glomerulonephritis with or without immunoglobulin A deposits, a membranous nephropathy, and a focal segmental necrotizing glomerulonephritis of the vasculitic type.Renal disease in mixed connective tissue disease and polymyositis is infrequent, but the former can be associated with a membranous and mesangial proliferative glomerulonephritis.Sjögren syndrome is rarely associated with clinically significant renal disease, but patients can present with proteinuria, acidosis, or hyperchloraemia. Interstitial nephritis and immune complex glomerulonephritis reflect the exocrinopathy and circulating immune complex disease pathognomonic of Sjögren syndrome. Evidence for effective treatment of the renal complications is lacking. Corticosteroids and cyclophosphamide are most commonly used, with newer biological drugs, such as rituximab, showing promise.
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Robb, Jessica F., and Lawrence M. Samkoff. Immunomodulatory Agents for Relapsing-Remitting Multiple Sclerosis. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780199341016.003.0025.
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The treatment of multiple sclerosis has become much more complicated with the development of new oral and other disease-modifying therapies (DMT) that have been added to the formulary of older injectables and natalizumab. To date, twelve medications have been approved in the United States for use in cases of relapsing multiple sclerosis. Neurologists managing patients with multiple sclerosis must integrate numerous variables when selecting the appropriate agent for each individual. This chapter presents an overview of the DMTs currently approved by the U.S. Food and Drug Administration for multiple sclerosis and suggests a therapeutic algorithm to aid in choosing among these medications.
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Waje-Andreassen, Ulrike, and Nicola Logallo. Vascular imaging: Ultrasound. Oxford University Press, 2018. http://dx.doi.org/10.1093/med/9780198722366.003.0009.
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After computed tomography and computed tomography angiography or magnetic resonance imaging and magnetic resonance angiography at admission, ultrasound is the most important diagnostic tool to confirm angiographic findings and to closely follow-up patients until the clinical situation has stabilized. Thrombolysis and interventional therapy have given transcranial ultrasound a very important role in bedside monitoring of occlusions, collaterals, cerebral haemodynamics, and vasoreactivity. Detection of flow changes in sickle cell disease, circulating emboli, and right-to-left shunts may guide treatment decisions. Sonothrombolysis and targeted drug delivery are today’s research projects for acute treatment by ultrasound. Extracranial cerebrovascular ultrasound is an ‘all-round’ diagnostic tool modifying angiographic results, showing minor arterial wall disease, plaques, and plaque instability. Microembolic signals during scanning may contribute to finding the cause of stroke. In stroke prevention, ultrasound delivers the possibility for staging of arteries and improving targeted intervention. Ultrasound images may also serve as educational tools for patients to underline the need for continuous medical treatment and lifestyle changes, and may improve compliance.
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Kaplan, Tamara B., and Marcelo Matiello. Multiple Sclerosis. Edited by Angela O’Neal. Oxford University Press, 2017. http://dx.doi.org/10.1093/med/9780190609917.003.0026.
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Multiple sclerosis (MS) often affects women of childbearing age. There are many issues to consider when counseling women about their disease and treatment during this time. The Pregnancy in Multiple Sclerosis (PRIMS) study, published in 1998, is the best large-scale prospective study published to date. Based on this trial, and those that followed, it is recognized that the rate of relapse in MS decreases during pregnancy, especially during the third trimester, but there is a significant increase in relapse rate in the first three months postpartum. If relapses do occur during pregnancy, women are often treated with methylprednisolone, but this is generally avoided in the first trimester. Disease-modifying therapies (DMTs) are usually discontinued during preconception, pregnancy, and while breast-feeding. DMTs are classified under different FDA pregnancy categories based on human and animal data. Breast-feeding may influence postpartum relapse rate, but the true effect continues to be debated.
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van Assen, Sander, and Marc Bijl. Vaccination in immunocompromised adults. Oxford University Press, 2013. http://dx.doi.org/10.1093/med/9780199642489.003.0094.
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This chapter addresses all important questions regarding vaccination of patients with autoimmune inflammatory rheumatic diseases (AIIRD). First, the incidence of vaccine-preventable infections in these patients is discussed. Pulmonary infections, including influenza and pneumococcal infection, occur more often in AIIRD patients; herpes zoster and human papillomavirus are also more frequent. The efficacy of vaccination for all European registered vaccines is discussed. Treatment with disease-modifying anti-rheumatic drugs (DMARDs) and biologicals (in particular TNFα-blocking agents) do not hamper, or only slightly hamper, the immune responses to most vaccines. Rituximab is an exception, severely reducing humoral responses following influenza and pneumococcal vaccination, at least during the first 6 months after administration. Safety of vaccination is an important issue in patients with autoimmune diseases, since increased disease activity of the underlying disease as a result of vaccination is theoretically possible. The available evidence is summarized, suggesting that vaccination is safe in AIIRD patients. Live vaccines, however, are contraindicated in immunosuppressed patients with AIIRD. Finally, the European League Against Rheumatism (EULAR) recommendations are highlighted, summarizing the 'do's' and 'don'ts' of vaccination in adults with AIIRD.
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Bawa, Sandeep, Paul Wordsworth, and Inoshi Atukorala. Spondyloarthropathies. Oxford University Press, 2011. http://dx.doi.org/10.1093/med/9780199550647.003.010004.
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♦ Spondyloarthropathies are related conditions typically associated with axial skeletal involvement, absence of rheumatoid factor, familial clustering, and a variable positive association with HLA-B27♦ Ankylosing spondylitis is the prototype with sacroiliac joint involvement being a prerequisite for diagnosis♦ Diagnosis is frequently delayed for several years but the use of magnetic resonance imaging to detect sacroiliitis greatly facilitates the establishment of an early diagnosis♦ Psoriatic arthritis, reactive arthritis, and enteropathic arthritis have prominent peripheral joint involvement with variable degrees of spinal involvement♦ Non-steroidal anti-inflammatory drugs and physical therapy are the cornerstones of management but slow-acting disease-modifying antirheumatic drugs only have a role in peripheral arthritis♦ Anti-tumour necrosis factor biologic agents have revolutionized the treatment of the spondyloarthropathies.
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Graham, Andrew, and Clare Galton. Nervous system. Oxford University Press, 2013. http://dx.doi.org/10.1093/med/9780199642489.003.0018.
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Rheumatological conditions may be complicated by a variety of both central and peripheral nervous system disorder. Common complications such as entrapment neuropathies are familiar to rheumatologists but accurate diagnosis of less common neurological disorders may be challenging; careful clinical reasoning is essential, supplemented where necessary by imaging, neurophysiology, and other special investigations including cerebrospinal fluid examination. Complications vary according to the nature of background condition. In rheumatoid arthritis, neurological involvement is typically related to the mechanical consequences of advancing disease; the commonest complications are carpal tunnel syndrome and cervical myelopathy due to atlantoaxial subluxation. By contrast, neurological involvement in systemic lupus erythematosus (SLE) tends to occur earlier in the disease course, with a much wider range of manifestations. The management of stroke or seizures in SLE is not necessarily any different from that in the general population, unless complicated by the antiphospholipid syndrome. However, less common neurological syndromes may demand more specific investigation and treatment. For example, longitudinally extensive transverse myelitis and recurrent optic neuritis (neuromyelitis optica, or Devic's disease) is frequently associated with antibodies to aquaporin-4, and is highly likely to relapse unless treated vigorously with humoral immunosuppression. Nervous system involvement in vasculitis is common. Finally, not all neurological disorder in rheumatological disease is necessarily due to the underlying condition; neurological complications of disease-modifying therapy are increasingly recognized, in particular central and peripheral nervous system demyelination associated with TNF-α inhibitors.
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Marshall, Tarnya, and Rita Abdulkader. Anti-rheumatic drugs in pregnancy and lactation. Oxford University Press, 2013. http://dx.doi.org/10.1093/med/9780199642489.003.0097.
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Anti-rheumatic drugs in pregnancy and lactation are increasingly a common clinical dilemma. With the shift towards early, aggressive control of autoimmune diseases and with the advent of newer therapeutic agents, there is a need to understand the effects of these medicines in pregnancy and lactation, on fertility in both men and women, and on the process of spermatogenesis, in order to understand the risk of teratogenesis. Although there are some limited data available for the use of anti-rheumatic drugs in pregnancy and lactation, much of our knowledge is derived from animal models and from limited clinical experience in human pregnancy. The balance of therapeutic benefits and risks of harm to mother and fetus should always be carefully considered: it may vary between individuals and should be assessed on a case by case basis. Because of these issues, pregnancy should always be discussed and planned in advance, in part to reduce disease activity prior to conception but also to minimize risk to the fetus. In this chapter we use the available evidence to discuss medicines which are commonly used in the treatment of rheumatological autoimmune diseases, and cover disease-modifying anti-rheumatic drugs (DMARDS) and biological agents.
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Marshall, Tarnya, Rita Abdulkader, and Poonam Sharma. Antirheumatic drugs in pregnancy and lactation. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780199642489.003.0097_update_003.
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Antirheumatic drugs in pregnancy and lactation are increasingly a common clinical dilemma. With the shift towards early, aggressive control of autoimmune diseases and with the advent of newer therapeutic agents, there is a need to understand the effects of these medicines in pregnancy and lactation, on fertility in both men and women, and on the process of spermatogenesis, in order to understand the risk of teratogenesis. Although there are some limited data available for the use of antirheumatic drugs in pregnancy and lactation, much of our knowledge is derived from animal models and from limited clinical experience in human pregnancy. The balance of therapeutic benefits and risks of harm to mother and fetus should always be carefully considered: it may vary between individuals and should be assessed on a case by case basis. Because of these issues, pregnancy should always be discussed and planned in advance, in part to reduce disease activity prior to conception but also to minimize risk to the fetus. In this chapter we use the available evidence to discuss medicines which are commonly used in the treatment of rheumatological autoimmune diseases, and cover disease-modifying antirheumatic drugs (DMARDS) and biological agents.
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Heijstek, Marloes, Mario Abinun, and Nico Wulffraat. Vaccination in immunocompromised children. Oxford University Press, 2013. http://dx.doi.org/10.1093/med/9780199642489.003.0095.
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Can immunocompromised children be safely and effectively vaccinated? This chapter discusses the recommendations from the European League Against Rheumatism (EULAR) for the immunization of immunocompromised patients. Patients with rheumatic or autoinflammatory diseases treated with high-dose glucocorticoids, high-dose disease-modifying anti-rheumatic drugs (DMARDs), or biologicals are considered immunocompromised. Safe and effective vaccination is crucial in these patients, given their increased risk of infection. Safe vaccination implies that vaccination has no effect on disease activity and has only mild adverse effects. Effective vaccination denotes that patients are protected against infections after immunization. Particularly in severely immunosuppressed patients, concerns arise on the safety of (live-attenuated) vaccines and on the detrimental effect of immunosuppressive treatment on the immunogenicity of vaccines. Overall, vaccinations do not increase disease activity and do not cause severe adverse events. Although non-live vaccines are safe, it is recommended to withhold live-attenuated vaccines in patients on high-dose immunosuppressive drugs and biologicals. However, booster vaccinations can be considered when essential. Generally, immunogenicity of vaccines is good with some exceptions: responses are reduced in patients on high-dose glucocorticoids and rituximab; methotrexate reduces responses to (pneumococcal) polysaccharide vaccines; and anti-tumour necrosis factor alpha (TNFα) may lower vaccine-induced antibody concentrations. Offering vaccination before immunosuppressive drugs and/or measuring antibodies after immunization is recommended.
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Heijstek, Marloes, Mario Abinun, and Nico Wulffraat. Vaccination in immunocompromised children. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780199642489.003.0095_update_003.
Full textAbstract:
Can immunocompromised children be safely and effectively vaccinated? This chapter discusses the recommendations from the European League Against Rheumatism (EULAR) for the immunization of immunocompromised patients. Patients with rheumatic or autoinflammatory diseases treated with high-dose glucocorticoids, high-dose disease-modifying antirheumatic drugs (DMARDs), or biologicals are considered immunocompromised. Safe and effective vaccination is crucial in these patients, given their increased risk of infection. Safe vaccination implies that vaccination has no effect on disease activity and has only mild adverse effects. Effective vaccination denotes that patients are protected against infections after immunization. Particularly in severely immunosuppressed patients, concerns arise on the safety of (live-attenuated) vaccines and on the detrimental effect of immunosuppressive treatment on the immunogenicity of vaccines. Overall, vaccinations do not increase disease activity and do not cause severe adverse events. It is recommended to withhold live-attenuated vaccines in patients on high-dose immunosuppressive drugs and biologicals, but booster vaccinations can be considered when essential. Generally, immunogenicity of vaccines is good with some exceptions: responses are reduced in patients on high-dose glucocorticoids and rituximab; methotrexate reduces responses to (pneumococcal) polysaccharide vaccines; and tumour necrosis factor alpha (TNFα) may lower vaccine-induced antibody concentrations and may cause accelerated waning of immunity. Offering vaccination before immunosuppressive drugs and/or measuring antibodies after immunization is recommended.
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Thornton, Clare, and Justin Mason. Vascular biology. Oxford University Press, 2013. http://dx.doi.org/10.1093/med/9780199642489.003.0057.
Full textAbstract:
Vascular biology is the study of the physiology of the vasculature and how it may be the target for disease processes. An understanding of vascular biology is central to the study of rheumatic disease for three reasons: it is an integral part of a functioning immune system; it is the primary site of pathology in many conditions; and it is the site of the important secondary complications of chronic inflammation, endothelial dysfunction and atherosclerosis. Vascular biology requires a detailed knowledge of the anatomy and physiology of the vasculature and its constituent vessels. The multistep process by which leucocytes interact with endothelium lining postcapillary venules in order to leave the circulation and migrate towards a site of inflammation is central to the pathology of inflammatory disease. The vasculature is the primary site of injury in several rheumatic diseases, including the vasculitides. It may also be damaged by chronic inflammation, leading to endothelial dysfunction and accelerated atherosclerosis. Thrombosis is also a critical pathological process in several chronic inflammatory diseases, particularly the anti-phospholipid antibody syndrome and Behçet's syndrome. The vascular endothelium is central to angiogenesis, the process of new capillary outgrowth, upon which synovial proliferation in inflammatory arthritis is dependent. Angiogenesis is inhibited by current anti-rheumatic therapies and may become a target for novel anti-rheumatic drugs. An increasing area of research concerns the direct effects of drugs used in the treatment of atherosclerosis and inflammatory disease on the endothelium, and whether these agents are beneficial or harmful. Of particular interest to rheumatologists are the vascular effects of statins, disease-modifying anti-rheumatic drugs (DMARDs), immunosuppressants, and cyclooxygenase inhibitors.
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Thornton, Clare, and Justin Mason. Vascular biology. Oxford University Press, 2015. http://dx.doi.org/10.1093/med/9780199642489.003.0057_update_001.
Full textAbstract:
Vascular biology is the study of the physiology of the vasculature and how it may be the target for disease processes. An understanding of vascular biology is central to the study of rheumatic disease for three reasons: it is an integral part of a functioning immune system; it is the primary site of pathology in many conditions; and it is the site of the important secondary complications of chronic inflammation, endothelial dysfunction and atherosclerosis. Vascular biology requires a detailed knowledge of the anatomy and physiology of the vasculature and its constituent vessels. The multistep process by which leucocytes interact with endothelium lining postcapillary venules in order to leave the circulation and migrate towards a site of inflammation is central to the pathology of inflammatory disease. The vasculature is the primary site of injury in several rheumatic diseases, including the vasculitides. It may also be damaged by chronic inflammation, leading to endothelial dysfunction and accelerated atherosclerosis. Thrombosis is also a critical pathological process in several chronic inflammatory diseases, particularly the anti-phospholipid antibody syndrome and Behçet’s syndrome. The vascular endothelium is central to angiogenesis, the process of new capillary outgrowth, upon which synovial proliferation in inflammatory arthritis is dependent. Angiogenesis is inhibited by current anti-rheumatic therapies and may become a target for novel anti-rheumatic drugs. An increasing area of research concerns the direct effects of drugs used in the treatment of atherosclerosis and inflammatory disease on the endothelium, and whether these agents are beneficial or harmful. Of particular interest to rheumatologists are the vascular effects of statins, disease-modifying anti-rheumatic drugs (DMARDs), immunosuppressants, and cyclooxygenase inhibitors.
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Rudwaleit, Martin. Enthesitis. Oxford University Press, 2013. http://dx.doi.org/10.1093/med/9780199642489.003.0054.
Full textAbstract:
Enthesitis is one of the key manifestations of spondyloarthritides (SpA) including ankylosing spondylitis (AS) and psoriatic arthritis. Enthesitis can occur alone or in combination with peripheral arthritis, sacroiliitis, or spondylitis. The inflammatory process is typically located at the insertion of the enthesis or ligament to bone, often resulting in osteitis as well. Because of its anatomical and functional complexity the term 'enthesis organ' has been coined. Biomechanical stress applied to the enthesis seems to play an important role for the occurrence of enthesitis in genetically predisposed individuals. Ultrasound imaging of peripheral entheses reveals enthesis abnormalities including entheseal calcification, bony erosion, or bony proliferation. Power Doppler signals demonstrating increased vascularization of inflamed entheses at the insertional site appear to be the most characteristic finding for enthesitis, yet study results are conflicting. Enthesitis-related osteitis and enthesitis at the spine is best visualized by MRI. Enthesitis may resolve spontaneously or may run a chronic course. Standard treatment includes local steroid injections, non-steroidal anti-inflammatory drugs (NSAIDs), and physical therapy. There is little evidence for the efficacy of disease-modifying anti-rheumatic drugs (DMARDs) in enthesitis. In contrast, anti-TNF agents have proven efficacy, and their use in treatment-resistant enthesitis is recommended in the Assessment of SpondyloArthritis international Society (ASAS)/European League Against Rheumatism (EULAR) recommendations for the management of AS and axial SpA and in the EULAR recommendations for psoriatic arthritis.
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Rudwaleit, Martin. Enthesitis. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780199642489.003.0054_update_002.
Full textAbstract:
Enthesitis is one of the key manifestations of spondyloarthritis (SpA) including ankylosing spondylitis (AS) and psoriatic arthritis. Enthesitis can occur alone or in combination with peripheral arthritis, sacroiliitis, or spondylitis. The inflammatory process is typically located at the insertion of the enthesis or ligament to bone, often resulting in osteitis as well. Because of its anatomical and functional complexity the term ’enthesis organ’ has been coined. Biomechanical stress applied to the enthesis seems to play an important role for the occurrence of enthesitis in genetically predisposed individuals. Ultrasound imaging of peripheral entheses reveals enthesis abnormalities including entheseal calcification, bony erosion, or bony proliferation. Power Doppler signals demonstrating increased vascularization of inflamed entheses at the insertional site appear to be the most characteristic finding for enthesitis, yet study results are conflicting. Enthesitis-related osteitis and enthesitis at the spine is best visualized by MRI. Enthesitis may resolve spontaneously or may run a chronic course. Standard treatment includes local steroid injections, non-steroidal anti-inflammatory drugs (NSAIDs), and physical therapy. There is little evidence for the efficacy of disease-modifying antirheumatic drugs (DMARDs) in enthesitis. In contrast, anti-TNF agents have proven efficacy, and their use in treatment-resistant enthesitis is recommended in the Assessment of SpondyloArthritis international Society (ASAS)/European League Against Rheumatism (EULAR) recommendations for the management of AS and axial SpA and in the EULAR recommendations for psoriatic arthritis.
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Platt, Philip, and Ismael Atchia. Injection therapy. Oxford University Press, 2013. http://dx.doi.org/10.1093/med/9780199642489.003.0087.
Full textAbstract:
Joint and soft tissue injections with glucocorticoids and other agents remain a critical aspect of the management of musculoskeletal conditions. Injection therapy has previously consisted mainly of glucocorticoid and local anaesthetic, but other agents such as hyaluronic acid, radioactive agents, plasma-rich products, and biologics have also been introduced in the practice of musculoskeletal clinicians. Overall glucocorticoid injection remains the most widely performed procedure, and is an effective treatment for an inflamed joint or soft tissue. This procedure has been widely used for at least five decades. Hydrocortisone was the initial steroid used but longer-acting steroid agents are now favoured for large joints. There is evidence of efficacy not only for inflammatory arthritis conditions, but also for osteoarthritis. There are certain contraindications for injection therapy. Hyaluronic acid (viscosupplementation) injection has become part of the management of osteoarthritis. More recently other agents such as disease-modifying anti-rheumatic drugs (DMARDs) and biologics have been injected with varying degree of success. The ability to deliver an injection accurately depends on appropriate knowledge of the anatomy and, as for any procedures, the experience and skill of the clinician. The approach to different joints and soft tissue structures is described in this chapter.
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Sieper, Joachim. Ankylosing spondylitis. Oxford University Press, 2013. http://dx.doi.org/10.1093/med/9780199642489.003.0113.
Full textAbstract:
Ankylosing spondylitis (AS) is a chronic inflammatory disease predominantly of the sacroiliac joint (SIJ) and the spine. It starts normally in the second decade of life and has a slight male predominance. The prevalence is between 0.2 and 0.8% and is strongly dependent on the prevalence of HLA B27 in a given population. For the diagnosis of AS, the presence of radiographic sacroiliitis is mandatory. However, radiographs do not detect active inflammation but only structural bony damage. Most recently new classification criteria for axial spondyloarthritis (SpA) have been developed by the Assessement of Spondylo-Arthritis international Society (ASAS) which cover AS but also the earlier form of non-radiographic axial SpA. MRI has become an important new tool for the detection of subchondral bone marrow inflammation in SIJ and spine and has become increasingly important for an early diagnosis. HLA B27 plays a central role in the pathogenesis but its exact interaction with the immune system has not yet been clarified. Besides pain and stiffness in the axial skeleton patients suffer also from periods of peripheral arthritis, enthesitis, and uveitis. New bone formation as a reaction to inflammation and subsequent ankylosis of the spine determine long-term outcome in a subgroup of patients. Currently only non-steroidal anti-inflammatory drugs (NSAIDs) and tumour necrosis factor (TNF) blockers have been proven to be effective in the medical treatment of axial SpA, and international ASAS recommendations for the structured management of axial SpA have been published based on these two types of drugs. Conventional disease-modifying anti-rheumatic drugs (DMARDs) such as methotrexate are not effective.
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Fleischmann, Roy. Signalling pathway inhibitors. Oxford University Press, 2013. http://dx.doi.org/10.1093/med/9780199642489.003.0081.
Full textAbstract:
Oral, small-molecule signalling pathway inhibitors, including ones that inhibit the JAK and SyK pathways, are currently in development for the treatment of rheumatoid arthritis (RA). Tofacitinib is an orally administered small-molecule inhibitor that targets the intracellular Janus kinase 3 and 1 (JAK1/3) molecules to a greater extent than JAK2 while baricitinib (formerly INCB028050) predominantly inhibits JAK1/2. Many of the proinflammatory cytokines implicated in the pathogenesis of RA utilize cell signalling that involves the JAK-STAT pathways and therefore inhibition of JAK-STAT signalling, by targeting multiple RA-associated cytokine pathways, has the potential to simultaneously reduce inflammation, cellular activation, and proliferation of key immune cells. Fostamatinib disodium is an orally available inhibitor of spleen tyrosine kinase (SyK), which is a cytoplasmic tyrosine kinase that is an important mediator of immunoreceptor signalling in mast cells, macrophages, neutrophils, and B cells. Interruption of SyK signalling may interrupt production of tumour necrosis factor (TNF) and metalloproteinase and therefore affect RA disease activity. Tofacitinib has been investigated in multiple phase 2 and phase 3 trials which have investigated its efficacy (clinical, functional, and radiographic) and safety in patients who have failed disease-modifying anti-inflammatory drugs (DMARDs) as monotherapy or in combination with DMARDs, compared to an inhibitor of tumour necrosis factor alpha (TNFα) and in patients who have failed TNFα inhibitors. The efficacy of fostamatinib and baricitinib has been investigated in phase 2 trials; both are in large phase 3 clinical programmes. Each of these medications has demonstrated efficacy; their safety profile has been shown to be different from each other and from currently approved biological agents. This chapter discusses what is currently known and understood about their efficacy and safety.
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Sieper, Joachim. Axial spondyloarthropathies. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780199642489.003.0113_update_003.
Full textAbstract:
Axial spondyloarthritis (axSpA) is a chronic inflammatory disease predominantly of the sacroiliac joint (SIJ) and the spine. It starts normally in the second decade of life and has a slight male predominance. The prevalence is between 0.2% and 0.8% and is strongly dependent on the prevalence of HLA-B27 in a given population. AxSpA can be split in patients with radiographic axSpA (also termed ankylosing spondylitis (AS)) and in patients with non-radiographic axSpA (nr-axSpA). For the diagnosis of AS, the presence of radiographic sacroiliitis is mandatory. However, radiographs do not detect active inflammation but only structural bony damage. Most recently new classification criteria for axSpA have been developed by the Assessment of Spondylo-Arthritis International Society (ASAS) which cover AS but also the earlier form of nr-axSpA. MRI has become an important new tool for the detection of subchondral bone marrow inflammation in SIJ and spine and has become increasingly important for an early diagnosis. HLA-B27 plays a central role in the pathogenesis but its exact interaction with the immune system has not yet been clarified. Besides pain and stiffness in the axial skeleton patients suffer also from periods of peripheral arthritis, enthesitis, and uveitis. New bone formation as a reaction to inflammation and subsequent ankylosis of the spine determine long-term outcome in a subgroup of patients. Currently only non-steroidal anti-inflammatory drugs (NSAIDs) and tumour necrosis factor (TNF) blockers have been proven to be effective in the medical treatment of axial SpA, and international ASAS recommendations for the structured management of axial SpA have been published based on these two types of drugs. Conventional disease-modifying anti-rheumatic drugs (DMARDs) such as methotrexate are not effective.