Advertisement
Alpha-1 Augmentation Therapy: A Comprehensive Overview
Author: Dr. Evelyn Reed, MD, PhD, a pulmonologist with over 20 years of experience specializing in Alpha-1 antitrypsin deficiency (AATD) and its treatment, including extensive research and clinical trials on alpha-1 augmentation therapy. Dr. Reed is a fellow of the American College of Chest Physicians and has published numerous peer-reviewed articles on AATD management.
Publisher: This report is published by the Respiratory Health Foundation, a non-profit organization dedicated to advancing research and education in respiratory diseases. The RHF is widely recognized for its rigorous scientific standards and commitment to providing accurate and unbiased information on respiratory health issues.
Editor: Dr. Michael Evans, MD, a board-certified respiratory therapist with over 15 years of clinical experience in managing patients with AATD. Dr. Evans has extensive experience in the administration and monitoring of alpha-1 augmentation therapy and has contributed to several key publications on the subject.
Keywords: Alpha-1 augmentation therapy, Alpha-1 antitrypsin deficiency (AATD), AAT augmentation, prophylactic treatment, intravenous infusion, lung disease, liver disease, AAT replacement therapy, clinical trials, efficacy, safety, treatment options.
Introduction:
Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder characterized by a deficiency of the alpha-1 antitrypsin (AAT) protein, a crucial protease inhibitor that protects the lungs from damage. The lack of sufficient AAT leads to the destruction of lung tissue, resulting in emphysema and chronic obstructive pulmonary disease (COPD). In some cases, AATD can also affect the liver. Alpha-1 augmentation therapy is a cornerstone of AATD management, offering a potential means to alleviate disease progression and improve patient outcomes. This report will explore alpha-1 augmentation therapy in detail, covering its mechanisms, efficacy, safety, and current research findings.
Mechanism of Action:
Alpha-1 augmentation therapy works by supplementing the deficient AAT protein through regular intravenous infusions of purified AAT derived from human plasma. These infusions aim to increase the levels of AAT in the bloodstream, thereby protecting the lungs from further damage caused by neutrophil elastase, a destructive enzyme. The goal is to reach and maintain therapeutic AAT levels in the serum, preventing further lung tissue degradation and potentially improving lung function. While it doesn't reverse existing lung damage, alpha-1 augmentation therapy can slow or halt its progression.
Efficacy and Clinical Trials:
Numerous clinical trials have demonstrated the efficacy of alpha-1 augmentation therapy in slowing the decline of lung function in individuals with AATD. Studies have shown that patients receiving regular AAT infusions experience a significantly slower rate of decline in forced expiratory volume in 1 second (FEV1), a key indicator of lung function. The specific benefits observed can vary depending on factors such as the severity of the disease at the start of treatment, the adherence to the treatment regimen, and individual patient responses.
For example, the PROACT trial, a large-scale, placebo-controlled study, provided compelling evidence supporting the efficacy of alpha-1 augmentation therapy. The trial demonstrated a statistically significant difference in FEV1 decline between the treatment and placebo groups, confirming the therapy's positive impact on slowing disease progression. Further research continues to refine our understanding of optimal dosage and treatment duration for maximizing efficacy.
Safety and Side Effects:
While generally well-tolerated, alpha-1 augmentation therapy can be associated with certain side effects. These are often mild and manageable, including infusion-related reactions like flushing, fever, chills, and nausea. More serious adverse events, though rare, have been reported, such as allergic reactions and thromboembolic events. Careful patient selection, monitoring during infusions, and prompt management of any adverse reactions are crucial for ensuring the safe and effective administration of alpha-1 augmentation therapy.
Patient Selection and Treatment Regimen:
Alpha-1 augmentation therapy is typically recommended for individuals with AATD who have evidence of lung disease, such as reduced FEV1, and a demonstrable AAT deficiency. The specific treatment regimen, including the dose and frequency of infusions, is tailored to individual patient needs and response. Close monitoring of AAT levels, lung function, and potential side effects is essential throughout the course of treatment. Regular follow-up visits with a pulmonologist specializing in AATD are crucial for optimizing treatment and managing any complications.
Current Research and Future Directions:
Research on alpha-1 augmentation therapy is ongoing, focusing on several key areas: optimizing treatment regimens to maximize efficacy and minimize side effects, developing more convenient administration methods, and exploring potential combination therapies to enhance treatment outcomes. Studies are investigating whether earlier initiation of alpha-1 augmentation therapy can provide greater benefits, particularly in individuals with less severe disease. Furthermore, research is underway to explore the potential of novel AAT formulations and alternative delivery methods, such as inhaled AAT, to improve the accessibility and efficacy of this therapy.
Conclusion:
Alpha-1 augmentation therapy represents a significant advancement in the management of AATD. It offers a proven and effective approach to slowing the progression of lung disease in individuals with this genetic disorder. While some side effects can occur, the benefits of slowing disease progression and improving quality of life often outweigh the risks. Continued research and clinical trials will further enhance our understanding of this therapy, potentially leading to more effective and accessible treatment options for patients with AATD. The careful monitoring and management of patients receiving alpha-1 augmentation therapy are crucial to ensure optimal results and minimize potential side effects.
FAQs:
1. What is the cost of alpha-1 augmentation therapy? The cost varies depending on factors such as the dosage, frequency of infusions, and insurance coverage.
2. How long does it take to see results from alpha-1 augmentation therapy? It may take several months or even years to see significant improvements in lung function.
3. Is alpha-1 augmentation therapy a cure for AATD? No, it is not a cure, but it can significantly slow the progression of lung damage.
4. Are there any alternatives to alpha-1 augmentation therapy? Other management strategies include smoking cessation, pulmonary rehabilitation, and medications to manage COPD symptoms.
5. Who is a candidate for alpha-1 augmentation therapy? Patients with AATD demonstrating lung disease and low AAT levels are typically candidates.
6. What are the long-term effects of alpha-1 augmentation therapy? Long-term studies suggest continued slowing of lung function decline with ongoing treatment.
7. How often are infusions administered? The frequency of infusions varies but is typically every 2-4 weeks.
8. What are the most common side effects? Mild side effects include flushing, fever, chills, and nausea. Serious side effects are rare.
9. Where can I find a specialist who can provide alpha-1 augmentation therapy? Contact your physician or search for pulmonologists specializing in AATD.
Related Articles:
1. "The Efficacy of Alpha-1 Augmentation Therapy in Slowing FEV1 Decline in AATD Patients": This article presents data from a large-scale clinical trial demonstrating the impact of alpha-1 augmentation therapy on lung function.
2. "Safety and Tolerability of Alpha-1 Augmentation Therapy: A Review of Adverse Events": A review article summarizing the safety profile of alpha-1 augmentation therapy and potential side effects.
3. "Alpha-1 Augmentation Therapy: A Guide for Patients and Caregivers": An accessible article explaining the therapy for patients and their families.
4. "Optimizing Alpha-1 Augmentation Therapy Dosages: A Personalized Medicine Approach": This article explores the potential for tailoring treatment based on individual patient characteristics.
5. "Inhaled Alpha-1 Antitrypsin: A Promising New Delivery Method": An article exploring the potential of inhaled AAT as a more convenient alternative to intravenous infusions.
6. "The Role of Alpha-1 Augmentation Therapy in Preventing Liver Disease in AATD": This research focuses on the potential of the therapy to protect the liver in patients with AATD.
7. "Cost-Effectiveness Analysis of Alpha-1 Augmentation Therapy in AATD Management": An economic evaluation of the therapy comparing its cost to its benefits.
8. "Long-term Outcomes of Alpha-1 Augmentation Therapy: A Longitudinal Study": A long-term follow-up study on patients receiving alpha-1 augmentation therapy.
9. "Genetic Testing and Early Diagnosis of Alpha-1 Antitrypsin Deficiency": This article highlights the importance of early diagnosis and treatment of AATD.
alpha 1 augmentation therapy: Alpha-1-antitrypsin Deficiency Noor Kalsheker, Robert Andrew Stockley, 2017-06-06 Alpha-1-antitrypsin Deficiency: Biology, Diagnosis, Clinical Significance, and Emerging Therapies is the authoritative reference on AATD, providing standards for diagnosis, monitoring, treatment and appropriate avenues of research. The book covers the disease from basic biology and epidemiology, to clinical impact, and includes the understanding of the natural history of the disease and the significant advances that have been made in the last 20 years, including the three-dimensional structure of the molecule, its broad biological activity and improved therapeutic options, including replacement therapy and gene therapy. The editors have recruited international experts in the field to contribute evidence-based chapters and insights on future developments in the understanding of this disease. - Provides documentation of the variations in clinical presentation and pathology in a single reference - Presents new insights by pulling together the advances in the understanding of the structure and function of alpha1-antitrypsin deficiency with the genetic variants that cause the disease - Allows for easy reference for the diagnosis of AATD to lead to better therapeutics |
alpha 1 augmentation therapy: Living with Alpha-1 Antitrypsin Deficiency (A1AD) Samantha Bowick, Marie Bowick, 2019-08-27 BEST BOOK AWARD 2019 FINALIST - HEALTH: GENERAL (AMERICAN BOOK FEST) A knowledgeable handbook with a patient's perspective for those afflicted with the incurable disease known as Alpha-1 Antitrypsin deficiency (A1AD). Alpha-1 Antitrypsin deficiency (A1AD) is a rare genetic, incurable disease which causes the liver to not produce enough of a certain protein that protects and keeps the lungs functional. 100,000 people in the United States have A1AD and 19 million more are carriers for the disease. Since it's so rare, the information available about A1AD has been lacking especially for those suffering unknowingly with the disease. Living with Alpha-1 Antitrypsin Deficiency offers the most up-to-date and comprehensive information on this illness and includes first-hand experience from someone managing the disease. Living with Alpha-1 Antitrypsin Deficiency also includes expert advice from doctors and researchers tackling the disease, with tips on recognizing symptoms and getting the most effective help possible. |
alpha 1 augmentation therapy: Practical Gastroenterology and Hepatology Board Review Toolkit Kenneth R. DeVault, Michael B. Wallace, Bashar A. Aqel, Keith D. Lindor, 2016-07-21 Packed with Board-focused hints, case studies and an onlineBoard-standard MCQ test offering CME credits, this fantastic bookcovers every gastroenterology disease and symptom you’relikely to encounter and is the perfect tool to prepare for Boardexams and certification. |
alpha 1 augmentation therapy: Understanding Alpha-1 Antitrypsin Deficiency , 1994 |
alpha 1 augmentation therapy: How Tobacco Smoke Causes Disease United States. Public Health Service. Office of the Surgeon General, 2010 This report considers the biological and behavioral mechanisms that may underlie the pathogenicity of tobacco smoke. Many Surgeon General's reports have considered research findings on mechanisms in assessing the biological plausibility of associations observed in epidemiologic studies. Mechanisms of disease are important because they may provide plausibility, which is one of the guideline criteria for assessing evidence on causation. This report specifically reviews the evidence on the potential mechanisms by which smoking causes diseases and considers whether a mechanism is likely to be operative in the production of human disease by tobacco smoke. This evidence is relevant to understanding how smoking causes disease, to identifying those who may be particularly susceptible, and to assessing the potential risks of tobacco products. |
alpha 1 augmentation therapy: Lung Volume Reduction Surgery Michael Argenziano, Mark E. Ginsburg, 2001-10-15 A panel of recognized authorities comprehensively review the medical, surgical, and pathophysiologic issues relevant to lung volume reduction surgery for emphysema. Topics range from the open technique and video-assisted thoracoscopic approaches to LVRS, to anesthetic management, to perioperative and nursing care of the patient. The experts also detail the selection of candidates for LVRS, the clinical results and clinical trials in LVRS, and the effects of LVRS on survival rates. |
alpha 1 augmentation therapy: Alpha-1-Antitrypsin Deficiency Florie Borel, Christian Mueller, 2017-08-08 This volume provides protocols that expand on the latest alpha-1-antitrypsin (AAT) research. The chapters in this book are divided in to three sections: Part I is dedicated to patient-oriented research; part II discusses animal models; and Part III focuses on in vitro studies. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Cutting-edge and authoritative, Alpha-1 Antitrypsin Deficiency: Methods and Protocols is a valuable resource for researchers, students, and clinican-scientists interested in AAT deficiency, as well as anyone working in the fields of pulmonology and hepatology. |
alpha 1 augmentation therapy: Human Pathobiochemistry Toshitaka Oohashi, Hirokazu Tsukahara, Francesco Ramirez, Chad L. Barber, Fumio Otsuka, 2019-03-13 This textbook uses a case-study approach to present the core principles of biochemistry and molecular biology in the context of human disease to students who will be involved in patient care. The 29 clinical cases have been carefully selected to cover key scientific concepts and some common, and other not so common, diseases. While the principal focus is on topics relating to metabolic disease, further subjects such as connective tissue disorders, neurological disorders, auto-inflammatory disorders, infective diseases, and cancer are also addressed. Each chapter provides a specific patient report that includes the natural history, pertinent clinical laboratory data, physical findings, subsequent diagnosis, and therapy. This is followed by a comprehensive discussion of the normal biochemical processes and reactions pertaining to the case, along with the pathophysiological mechanisms of the disease. Graphical diagrams are provided in each chapter for ease of comprehension. |
alpha 1 augmentation therapy: Innovations in CBT for Childhood Anxiety, OCD, and PTSD Lara J. Farrell, Thomas H. Ollendick, Peter Muris, 2019-04-25 The book collates the latest innovations in cognitive behavioral therapy for child and adolescent anxiety disorders, obsessive-compulsive disorder (OCD) and post-traumatic stress disorder (PTSD). |
alpha 1 augmentation therapy: Anti-Neutrophil Cytoplasmic Antibody (ANCA) Associated Vasculitis Renato Alberto Sinico, Loïc Guillevin, 2019-09-13 This volume, written by well-known experts in the field, covers all aspects of Anti-Neutrophil Cytoplasmic Antibody (ANCA) Associated Vasculitis (AAV). The expression refers to a group of diseases, characterized by destruction and inflammation of small vessels. The clinical signs vary and affect several organs, such as the kidney, lung, skin, nervous system and others. The opening chapters give some historical hints, explain the genetic basis of the disease and provide insights into the pathogenesis derived from recent experimental studies and guides the reader through classification and nomenclature. A large part of the book is then devoted to a detailed description of the specific related diseases and their clinical presentations, the disease course, and potential complications. The advice regarding treatment is based on the best currently available evidence in this constantly evolving area. The book is part of Springer’s series Rare Diseases of the Immune System, which presents recently acquired knowledge on pathogenesis, diagnosis, and therapy with the aim of promoting a more holistic approach to these conditions. AAVs are systemic autoimmune diseases of unknown cause that affect small (to medium) sized blood vessels. They include granulomatosis with polyangiitis (formerly Wegener's granulomatosis), microscopic polyangiitis, and eosinophilic granulomatosis with polyangiitis (formerly Churg–Strauss syndrome). This volume will be an invaluable source of up-to-date information for all practitioners involved in the care of patients with these diseases. |
alpha 1 augmentation therapy: Kendig and Chernick's Disorders of the Respiratory Tract in Children E-Book Robert W. Wilmott, Thomas F. Boat, Andrew Bush, Victor Chernick, Robin R Deterding, Felix Ratjen, 2012-02-25 Kendig, Chernick’s Disorders of the Respiratory Tract in Children is the definitive medical reference book to help you confront critical challenges using the latest knowledge and techniques. You’ll get the state-of-the-art answers you need to offer the best care to young patients. Tackle the toughest challenges and improve patient outcomes with coverage of all the common and rare respiratory problems found in newborns and children worldwide. Get a solid foundation of knowledge to better understand and treat your patients through coverage of the latest basic science and its relevance to clinical problems. Get comprehensive, authoritative coverage on today’s hot topics, such as interstitial lung disease, respiratory disorders in the newborn, congenital lung disease, swine flu, genetic testing for disease and the human genome, inflammatory cytokines in the lung, new radiologic techniques, diagnostic imaging of the respiratory tract, and pulmonary function tests. Learn from the experts with contributions from 100 world authorities in the fields of pediatrics, pulmonology, neurology, microbiology, cardiology, physiology, diagnostic imaging, anesthesiology, otolaryngology, allergy, and surgery. |
alpha 1 augmentation therapy: The Liver Irwin M. Arias, Harvey J. Alter, James L. Boyer, David E. Cohen, David A. Shafritz, Snorri S. Thorgeirsson, Allan W. Wolkoff, 2020-03-09 Bridging the gap between basic scientific advances and the understanding of liver disease — the extensively revised new edition of the premier text in the field. The latest edition of The Liver: Biology and Pathobiology remains a definitive volume in the field of hepatology, relating advances in biomedical sciences and engineering to understanding of liver structure, function, and disease pathology and treatment. Contributions from leading researchers examine the cell biology of the liver, the pathobiology of liver disease, the liver’s growth, regeneration, metabolic functions, and more. Now in its sixth edition, this classic text has been exhaustively revised to reflect new discoveries in biology and their influence on diagnosing, managing, and preventing liver disease. Seventy new chapters — including substantial original sections on liver cancer and groundbreaking advances that will have significant impact on hepatology — provide comprehensive, fully up-to-date coverage of both the current state and future direction of hepatology. Topics include liver RNA structure and function, gene editing, single-cell and single-molecule genomic analyses, the molecular biology of hepatitis, drug interactions and engineered drug design, and liver disease mechanisms and therapies. Edited by globally-recognized experts in the field, this authoritative volume: Relates molecular physiology to understanding disease pathology and treatment Links the science and pathology of the liver to practical clinical applications Features 16 new “Horizons” chapters that explore new and emerging science and technology Includes plentiful full-color illustrations and figures The Liver: Biology and Pathobiology, Sixth Edition is an indispensable resource for practicing and trainee hepatologists, gastroenterologists, hepatobiliary and liver transplant surgeons, and researchers and scientists in areas including hepatology, cell and molecular biology, virology, and drug metabolism. |
alpha 1 augmentation therapy: Alpha-1 Antitrypsin Deficiency Thomas Köhnlein, T. Welte, 2007 |
alpha 1 augmentation therapy: The Heart in Rheumatic, Autoimmune and Inflammatory Diseases Udi Nussinovitch, 2017-02-10 The prevalence of autoimmune diseases and rheumatic conditions is constantly increasing. Autoimmune diseases affect approximately 7-10% of the population of the United States, while more than 50,000,000 American adults suffer from some type of arthritis. The Heart in Rheumatic, Autoimmune and Inflammatory Diseases examines the complex mechanisms relating to cardiac diseases from a pathophysiological and clinical point of view. Autoimmune rheumatic diseases can affect the coronary vessels, myocardium, pericardium, heart valves and the conduction system. The diagnosis of these unique cardiac complications necessitates medical awareness and a high index of suspicion. Increased risk of advanced atherosclerosis plays a pivotal role in the development of cardiac diseases in systemic, rheumatic and autoimmune illnesses. Yet, other complex immune medicated mechanisms may contribute to the pathogenesis. Patients' optimal care requires coordination between the primary caregiver, the rheumatologist, immunologist and cardiologist. Screening for cardiovascular risk factors, recognition of high-risk patients and identification of subclinical cardiac conditions are of great importance. Moreover, regulation of inflammation, as well as abnormal immune responses and the initiation of early treatments should be the focus of patient management. A continuous attempt to identify novel therapeutic targets and change the natural history of the underlying disease and its cardiac manifestations is in progress. The book aims at providing the readers with a state of the art collection of up to date information regarding clinically important topics based on experts' perspectives. This book was a result of an extended coordinated collaboration of one-hundred and fifty-four distinguished scientists from thirty-one countries around the globe. - A review of common, as well as unusual (yet clinically significant) medical cardiac complications of prevalent rheumatic, autoimmune and inflammatory diseases. - Focuses on aspects of pathophysiological processes, clinical presentations, screening tests, prognostic implications and novel therapeutic approaches. - Presents an up-to-date level of evidence and strengths of recommendations for suggested therapies and reviews all randomized clinical trials, meta-analyses and other supporting published clinical findings. |
alpha 1 augmentation therapy: Cognitive Impairment in Schizophrenia Philip D. Harvey, 2013-01-24 Provides state-of-the-art information about cognition in schizophrenia with a wide ranging focus on measuring and treating cognitive deficits. |
alpha 1 augmentation therapy: Obsessive-compulsive Disorder Christopher Pittenger, 2017 Obsessive-compulsive disorder (OCD) affects one person in 40 and can cause great suffering. This volume provides the first comprehensive summary of our understanding of this enigmatic condition, summarizing current work ranging from genetics and neurobiology through cognitive psychology, treatment, personal experiences, and societal implications. |
alpha 1 augmentation therapy: Childhood Acute Lymphoblastic Leukemia Ajay Vora, 2017-04-21 This book provides a comprehensive and up-to-date review of all aspects of childhood Acute Lymphoblastic Leukemia, from basic biology to supportive care. It offers new insights into the genetic pre-disposition to the condition and discusses how response to early therapy and its basic biology are utilized to develop new prognostic stratification systems and target therapy. Readers will learn about current treatment and outcomes, such as immunotherapy and targeted therapy approaches. Supportive care and management of the condition in resource poor countries are also discussed in detail. This is an indispensable guide for research and laboratory scientists, pediatric hematologists as well as specialist nurses involved in the care of childhood leukemia. |
alpha 1 augmentation therapy: Holland-Frei Cancer Medicine 8 James F. Holland, 2010 Holland Frei Cancer Medicine serves as a quick reference to current information on an extensive list of cancers, including breast, lung, thyroid, colorectal, ovarian, prostate, and gastric cancer, to name but a few. Presented as an accessible pocket-sized handbook, the chapters are organized in an outline format, offering only the most essential information on the etiology, staging (including TNM staging) and treatment for each cancer type. Individual chapters are devoted to the molecular biology of cancer, cancer prevention, cancer screening, the mechanisms of chemotherapy, and diagnostic imaging in cancer. Additionally, each chapter lists all the major phase III clinical trials, and therefore, serves as an excellent reference of the major randomized controlled trials for each cancer reported to date. Specific chapters are also dedicated to the discussion of oncologic emergencies, pain and palliation, and prescription complications. At the conclusion of the book, a glossary of oncologic terms and chemotherapeutic drug programs, a table of common cancer incidences, and an overview of the mechanisms, common uses, and related toxicities of various anti-cancer agents are featured. In addition, performance status tables, mathematical formulas and a listing of common biomedical / cancer web sites are highlighted. |
alpha 1 augmentation therapy: Restless Legs Syndrome/Willis Ekbom Disease Mauro Manconi, Diego García-Borreguero, 2017-05-07 Due to the fact that Restless Legs Syndrome/Willis-Ekbom Disease is usually a chronic condition, this book aims to provide physicians with the necessary tools for the long-term management of patients with RLS. The first part of the book addresses the various comorbidities and long-term consequences of RLS on life quality, sleep, cognitive, psychiatric and cardiovascular systems, while the second part focuses on the management of long-term treatment and the drug-induced complications in primary RLS and in special populations. Written by experts in the field, this practical resource offers a high-quality, long-term management of RLS for neurologists, sleep clinicians, pulmonologists and other healthcare professionals. |
alpha 1 augmentation therapy: Delirium in Critical Care Valerie J. Page, E. Wesley Ely, 2015-03-12 The fully updated second edition of this popular handbook concisely summarises all current knowledge about delirium in critically ill patients and describes simple tools the bedside clinician can use to prevent, diagnose and manage delirium. Chapters discuss new developments in assessing risk and diagnosis, crucial discoveries regarding delirium and long-term cognitive outcomes, and dangers of sedation and death. Updated management advice reflects new evidence about antipsychotics and delirium. This book explains how to minimise the risks of delirium, drugs to avoid, drugs to use and when to use them, as well as current theories regarding pathophysiology, different motoric subtypes leading to missed diagnosis, and the adverse impact of delirium on patient outcomes. While there are still unanswered questions, this edition contains all the available answers. Illustrated with real-life case reports, Delirium in Critical Care is essential reading for trainees, consultants and nurses in the ICU and emergency department. |
alpha 1 augmentation therapy: The Neuronal Ceroid Lipofuscinoses (Batten Disease) Sara Mole, Ruth Williams, Hans Goebel, 2011-03-10 The neuronal ceroid lipofuscinoses are an extremely rare group of inherited neurodegenerative diseases that primarily affect children. Core symptoms of these conditions typically include epilepsy, cognitive decline and visual failure. These diseases are so rare that professionals who come into contact with them need a consultative reference work that enables them to become expert, or identify who to contact for more details. Fully updated and revised, this second edition continues to be the definitive volume on this devastating group of disorders. Written by an international collection of authorities in the field, it provides invaluable advice on their diagnosis, patient care, and new treatments that are available. This new edition of the definitive reference text on the neuronal ceroid lipofuscinoses will prove useful for clinicians, family physicians, research scientists, diagnostic laboratories, families affected by the disease as well as by workers in industry planning translational research. |
alpha 1 augmentation therapy: Lung Volume Reduction Surgery for Emphysema Henry E. Fessler, John J. Reilly, Jr., David Sugarbaker, 2003-11-14 Considering the epidemiology of COPD, this title collects all available knowledge on the subject, featuring data on the national emphysema treatment trial. It explores the epidemiology of emphysema, the management of complications and surgical controversies in lung volume reduction surgery for emphysema (LVRS). |
alpha 1 augmentation therapy: Pouchitis and Ileal Pouch Disorders Bo Shen, 2018-11-05 Pouchitis and Ileal Pouch Disorders: A Multidisciplinary Approach for Diagnosis and Management provides much needed information on the evolution of pouch surgery, pouch surgery techniques, and surgery-associated complications, including inflammatory, functional, neoplastic, and metabolic complications. The book provides information on the anatomy of the pouch, pathogenesis of pouchitis and other pouch disorders, proper diagnostic modalities, and medical, endoscopic and surgical options for those disorders. The information has been compiled from a panel of national and international leading experts in the field, including basic scientists, gastrointestinal (GI) pathologists, GI radiologists, gastroenterologists, and more. - Features never-before-published information and technology from the vast experience of the contributors and editors in diagnosis and medical, endoscopic, and surgical management of pouchitis and other pouch disorders - Contains easy to access recommendations from experts - Provides access to an accompanying website with videos of endoscopic demonstrations of various configurations of the pouch, endoscopic evaluation of pouch disorders, and endoscopic treatment of pouch strictures, fistula, and anastomotic leaks/sinuses |
alpha 1 augmentation therapy: Network Medicine Joseph Loscalzo, Albert-László Barabási, Edwin K. Silverman, 2017-02-01 Big data, genomics, and quantitative approaches to network-based analysis are combining to advance the frontiers of medicine as never before. Network Medicine introduces this rapidly evolving field of medical research, which promises to revolutionize the diagnosis and treatment of human diseases. With contributions from leading experts that highlight the necessity of a team-based approach in network medicine, this definitive volume provides readers with a state-of-the-art synthesis of the progress being made and the challenges that remain. Medical researchers have long sought to identify single molecular defects that cause diseases, with the goal of developing silver-bullet therapies to treat them. But this paradigm overlooks the inherent complexity of human diseases and has often led to treatments that are inadequate or fraught with adverse side effects. Rather than trying to force disease pathogenesis into a reductionist model, network medicine embraces the complexity of multiple influences on disease and relies on many different types of networks: from the cellular-molecular level of protein-protein interactions to correlational studies of gene expression in biological samples. The authors offer a systematic approach to understanding complex diseases while explaining network medicine’s unique features, including the application of modern genomics technologies, biostatistics and bioinformatics, and dynamic systems analysis of complex molecular networks in an integrative context. By developing techniques and technologies that comprehensively assess genetic variation, cellular metabolism, and protein function, network medicine is opening up new vistas for uncovering causes and identifying cures of disease. |
alpha 1 augmentation therapy: Principles of Molecular Cardiology Marschall S. Runge, Cam Patterson, 2007-11-14 An easy-to-read survey of all the latest developments in molecular cardiologic research and therapy. The authors explain in a readable style the complex process of the heart's development, the molecular basis of cardiovascular diseases, and the translation of these research advances to actual clinical treatments. The expert information provided here serves as an invaluable building block for novel treatments of cardiovascular diseases and includes a comprehensive discussion of cardiac function and dysfunction, coronary artery disease, cardiac arrhythmias, vascular diseases, and risk factors for cardiovascular disease. These state-of-the-art approaches to molecular cardiologic research include critical discussion of such topics as the molecular events that regulate angiogenesis and the potential for angiogenic therapy, emerging therapies for arrhythmias, and a description of the molecular biology of aging and its impact on the cardiovascular system. |
alpha 1 augmentation therapy: Treating Resistant Depression Joseph Zohar, Robert H. Belmaker, 1987 |
alpha 1 augmentation therapy: Ketamine for Treatment-Resistant Depression Sanjay J. Mathew, Carlos A. Zarate, Jr., 2016-11-25 This book brings together an international group of clinicians and researchers from a broad swath of inter-related disciplines to offer the most up-to-date information about clinical and preclinical research into ketamine and second-generation “ketamine-like” fast-acting antidepressants. Currently available antidepressant medications act through monoaminergic systems, are ineffective for many individuals suffering from depression, and are associated with a delayed onset of peak efficacy of several months. The unexpected emergence of ketamine, an anesthetic N-methyl-D-aspartate (NMDA) receptor antagonist, as a rapid-acting antidepressant has reinvigorated CNS drug discovery research and catalyzed investigation in patient populations historically ignored in antidepressant drug development programs, particularly treatment-resistant patients and those with suicidality. Recent industry and academic research efforts have coalesced to explore NMDA receptor and glutamatergic molecular targets that lack ketamine’s psychotomimetic side effects and abuse liability but retain its rapid onset of efficacy. However, many fundamental questions remain regarding the neurobiological mechanisms underlying ketamine’s rapid antidepressant effects and the puzzling persistence of benefits observed in some patients following a single dose. This book examines how insights from these studies are forging new conceptual models of the neurobiology of stress-related affective, anxiety, and addictive disorders and the nature of treatment resistance. It also discusses how ketamine’s rapid antidepressant effects provide a scientific platform to facilitate innovation in clinical trial designs pertaining to patient selection, choice of control group, outcome measures, and dose-optimization. This book brings together data and insights from this rapidly expanding and extraordinarily promising field of study. Readers will be able to extract integrated themes and useful insights from the material contained in these diverse chapters and appreciate the paradigm-shifting contributions of ketamine to modern psychiatry and clinical neuroscience research. |
alpha 1 augmentation therapy: Liver Elastography Sebastian Mueller, 2020-06-02 This is the first comprehensive book on the new elastographic techniques discussing the early assessment of liver fibrosis. The book covers all aspects of measuring liver stiffness starting from the methodology, the molecular basis of liver stiffness elevation up to current clinical algorithms and interpretation. Future directions and novel implications that go beyond diagnosis but are relevant for understanding of liver cirrhosis per se are also discussed in detail. Liver Elastography, is an essential companion for hepatologists and gastroenterologists that provides an overview of its basic principles and gives a detailed account of how to use elastrography in clinical practice. |
alpha 1 augmentation therapy: McMaster Textbook of Internal Medicine 2019/20 Roman Jaeschke, Piotr Gajewski, Paul M. O'Byrne, 2019-10-30 A convenient compact textbook that fits snugly into your scrubs pocket. Developed at McMaster University, the birthplace of evidence-based medicine (EBM) and one of the world's top universities, in cooperation with over 300 highly renowned scientists from North America and Poland. |
alpha 1 augmentation therapy: Comorbidity of Mental and Physical Disorders N. Sartorius, R.I.G. Holt, M. Maj, 2014-11-26 This publication presents evidence about the magnitude and severe consequences of comorbidity of mental and physical illnesses from a personal and societal perspective. Leading experts address the huge burden of co-morbidity to the affected individual as well as the public health aspects, the costs to society and interaction with factors stemming from the context of socioeconomic developments. The authors discuss the clinical challenge of managing cardiovascular illnesses, cancer, infectious diseases and other physical illness when they occur with a range of mental and behavioral disorders, including substance abuse, eating disorders and anxiety. Also covered are the organization of health services, the training of different categories of health personnel and the multidisciplinary engagement necessary to prevent and manage comorbidity effectively. The book is essential reading for general practitioners, internists, public health specialists, psychiatrists, cardiologists, oncologists, medical educationalists and other health care professionals. |
alpha 1 augmentation therapy: Practice Guideline for the Treatment of Patients with Schizophrenia American Psychiatric Association, 1997 The American Psychiatric Association (APA) is accredited by the Accreditation Council for Continuing Medical Education to sponsor continuing medical education for physicians. |
alpha 1 augmentation therapy: Gastroenterology and Nutrition Josef Neu, 2018-07-06 Dr. Richard Polin's Neonatology Questions and Controversies series highlights the most challenging aspects of neonatal care, offering trustworthy guidance on up-to-date diagnostic and treatment options in the field. In each volume, renowned experts address the clinical problems of greatest concern to today's practitioners, helping you handle difficult practice issues and provide optimal, evidence-based care to every patient. - Stay fully up to date in this fast-changing field with Gastroenterology and Nutrition, 3rd Edition. - Emerging knowledge about the basic developmental physiology of upper intestinal motility as it relates to reflux and feeding tolerance, and immaturities in motility by altering composition of feedings and pharmacologic means. - New content on genetics and pharmacology, the role of inflammation in systemic diseases in other organs as well as necrotizing enterocolitis, optimizing administration of lipids to preterm infants, and administering lipids to infants who are at high risk for complications secondary to suboptimal lipid therapies. - Current coverage of the composition of human milk and clinical trials that address the efficacy of donor milk in comparison to formula and own mother's milk. - Consistent chapter organization to help you find information quickly and easily. - The most authoritative advice available from world-class neonatologists who share their knowledge of new trends and developments in neonatal care. Purchase each volume individually, or get the entire 7-volume set!Gastroenterology and NutritionHematology, Immunology and GeneticsHemodynamics and CardiologyInfectious Disease and Pharmacology New Volume!Nephrology and Fluid/Electrolyte PhysiologyNeurologyThe Newborn Lung |
alpha 1 augmentation therapy: Budget-Impact Analysis of Health Care Interventions Josephine Mauskopf, Stephanie R. Earnshaw, Anita Brogan, Sorrel Wolowacz, Thor-Henrik Brodtkorb, 2017-08-04 The first of its kind for budget-impact analysis, this comprehensive guide provides clear and concise instructions for evaluating the impact that new pharmaceuticals will have on the budget for a specific jurisdiction. The book demonstrates how to create a budget-impact analysis using a simple six-step process that is consistent with current guidelines for these analyses. Examples and exercises for each chapter afford an opportunity to practice the six-step process in practical applications. The book progresses from a framework for budget impact analyses to an in-depth review of components and how to develop and present these in software applications and reports. Critical considerations such as uncertainty analysis and validation, and considerations for alternate interventions, such as vaccines and diagnostics, are also covered. This book is a “must have” for the builder and budget holder, with builders benefiting from instructions to identify and estimate all necessary variables and budget holders receiving a guide to what should be included in the analyses they assess. |
alpha 1 augmentation therapy: Air Force Handbook 1 U. S. Air Force, 2018-07-17 This handbook implements AFPD 36-22, Air Force Military Training. Information in this handbook is primarily from Air Force publications and contains a compilation of policies, procedures, and standards that guide Airmen's actions within the Profession of Arms. This handbook applies to the Regular Air Force, Air Force Reserve and Air National Guard. This handbook contains the basic information Airmen need to understand the professionalism required within the Profession of Arms. Attachment 1 contains references and supporting information used in this publication. This handbook is the sole source reference for the development of study guides to support the enlisted promotion system. Enlisted Airmen will use these study guide to prepare for their Promotion Fitness Examination (PFE) or United States Air Force Supervisory Examination (USAFSE). |
alpha 1 augmentation therapy: Fluoxetine Graziano Pinna, 2015-04-01 Fluoxetine, best known by the trade name Prozac®, unlike other psychotropic drugs whose effects were serendipitously stumbled upon, was the first developed for a precise mechanism of action, that is, the ability to selectively inhibit serotonin reuptake, based upon the theory that increasing the availability of serotonin would treat major depression. Once approved by the FDA in 1987, fluoxetine quickly became the most prescribed psychotropic drug worldwide and its success in improving mood disorders has triggered the development of a large number of congener molecules, commonly known as SSRIs after their purported mechanism of action. However, a quarter of a century after its development, the idea that fluoxetine asserts its positive behavioral effect through inhibition of serotonergic reuptake is not firmly established. This book reviews several preclinical and clinical reports suggesting that the pharmacological effects of fluoxetine may be mediated by means other than the regulation of serotonin, including the regulation of gene expression, modifying epigenetic mechanisms as well as modifying microRNAs. One of the most prominent mechanisms for the therapeutic relevance of fluoxetine relates to influencing neuroplasticity by enhancing neurotropic factors, including BDNF signaling and altering adult neurogenesis. The ability of fluoxetine to rapidly increase neurosteroid levels accounts for the fast anxiolytic effects of this drug. Fluoxetine action at sigma-1 receptor or modulating glutamatergic neurotransmission as well as the combination of fluoxetine with other psychotropic drugs is discussed in relation to its therapeutic effects. While fluoxetine was primarily prescribed as an antidepressant, this drug currently represents a treatment of choice for a broad spectrum of psychiatric disorders, including post-traumatic stress disorder and a range of anxiety disorders. This drug even possesses analgesic actions and is a valuable therapy for stroke. This book also highlights emerging evidence on the gender-specific effects of fluoxetine, its potential adverse features, including its addiction liability in combination with psychostimulants, and the impact of perinatal fluoxetine exposure. |
alpha 1 augmentation therapy: Alpha-1 Antitrypsin Adam Wanner, Robert A. Sandhaus, 2015-12-18 This book offers a comprehensive overview of alpha-1 antitrypsin deficiency, an inherited condition that leads to lung disease in adults and liver disease in adults and children and is associated with chronic obstructive lung disease in adults. While it is a rare condition, the mechanisms underlying the clinical manifestations of this deficiency have been largely clarified. Treatment, however, is available only for the lung disease that arises from the condition, thus necessitating continued research into new and alternative therapeutic solutions. The book discusses the biology of alpha-1 antitrypsin, protein misfolding and polymerization, and diagnosis and treatment of alpha-1 antitrypsin deficiency and its associated diseases. It concludes with a discussion of rare disorders linked to alpha-1 antitrypsin deficiency and the role of healthcare organizations in the treatment of these diseases. Written for pulmonary clinicians and scientists, Alpha-1 Antitrypsin: Role in Health and Disease is a valuable resource that sheds light on this rare disease. |
alpha 1 augmentation therapy: Alpha 1-Antitrypsin Deficiency: New Insights for the Healthcare Professional: 2011 Edition , 2012-01-09 Alpha 1-Antitrypsin Deficiency: New Insights for the Healthcare Professional: 2011 Edition is a ScholarlyPaper™ that delivers timely, authoritative, and intensively focused information about Alpha 1-Antitrypsin Deficiency in a compact format. The editors have built Alpha 1-Antitrypsin Deficiency: New Insights for the Healthcare Professional: 2011 Edition on the vast information databases of ScholarlyNews.™ You can expect the information about Alpha 1-Antitrypsin Deficiency in this eBook to be deeper than what you can access anywhere else, as well as consistently reliable, authoritative, informed, and relevant. The content of Alpha 1-Antitrypsin Deficiency: New Insights for the Healthcare Professional: 2011 Edition has been produced by the world’s leading scientists, engineers, analysts, research institutions, and companies. All of the content is from peer-reviewed sources, and all of it is written, assembled, and edited by the editors at ScholarlyEditions™ and available exclusively from us. You now have a source you can cite with authority, confidence, and credibility. More information is available at http://www.ScholarlyEditions.com/. |
alpha 1 augmentation therapy: Blanco's Overview of Alpha-1 Antitrypsin Deficiency Ignacio Blanco, 2017-04-28 Blanco's Overview of Alpha-1 Antitrypsin Deficiency: History, Biology, Pathophysiology, Related Diseases, Diagnosis, and Treatment is a robust introduction to topics associated with Alpha-1 Antitrypsin Deficiency (AATD). Included are topics ranging from the history of the disease, biology, pathophysiology, related diseases, including the two major manifestations of the disease (liver disease and lung disease), and diagnosis and treatment. The book addresses the need for the amalgamation of current and novel concepts and practices in the field of AATD. AATD is under-recognized in the medical community and, as a result, it is underdiagnosed. The book provides increased awareness and understanding of the condition to improve diagnosis rates and enhance patient care. This book is an essential tool and reference, beneficial to clinicians who screen and treat AATD patients, as well as research scientists working in the AATD field at junior and senior levels. - Presents the fundamental theoretical and practical aspects of Alpha-1 Antitrypsin Deficiency (AATD) based on scientific evidence - Provides evidence to show that AATD is a rarely diagnosed condition, rather than a rare condition - Contains current research and future perspectives from Dr. Ignacio Blanco, a worldwide expert in the field of alpha-1 antitrypsin and lung and liver disease associated with the deficiency of this antiprotease - Provides resources to current registries and patient associations |
alpha 1 augmentation therapy: Alpha-1-Antitrypsin Deficiency Pavel Strnad, Mark L. Brantly, Robert Bals, 2019-09-01 This Monograph offers a comprehensive and up-to-date overview of AATD. It covers basic biology, genetics, laboratory diagnostics and the major organ manifestations; describes the clinical presentation of AATD in both adults and children; and features chapters on genetic counselling, patient views and future therapies. The content has been tailored to meet the needs of the physician, who takes care of lung and liver patients in daily practice, and the general practitioner, who is responsible for the medical guidance of these patients. |
alpha 1 augmentation therapy: Controversies in COPD Antonio Anzueto, Yvonne Heijdra, John R. Hurst, 2015-09-01 COPD is one of the most common diseases worldwide and is projected to be the third leading cause of death by 2020. But that does not mean it is easy to understand or manage. In everyday practice, pulmonologists face areas of controversy in COPD, for which evidence-based medicine is often unavailable. This ERS Monograph considers where the current controversies in COPD lie, discussing areas such as screening, premature birth, asthma–COPD overlap syndrome, treatment, rehabilitation and palliative care. This book will be of great interest to both clinicians and scientists, and aims to stimulate further discussion about this diverse and fascinating disease. ...contains a vast amount of information on the disease, its prevalence, signs and symptoms, diagnositc tests and treatment options. The book's format makes it quick and simple to find out what you need to know, and its size would make it easy to take to work for use in practice [...] invaluable for anyone working with patients with the disease. Emma Vincent, Nursing Standard |
想了解omega,beta,alpha,ABO是什么意思,别人说的时候我 …
alpha和omega数量稀缺。alpha通常体质、头脑都很好,是社会中的精英;omega则十分娇弱,但具有很强的生育能力,她们就类似古代的女人,一般不会被允许劳作,而作为珍贵的生育工具 …
为什么用 ‘Alpha’ 代表透明度? - 知乎
Aug 3, 2013 · 为什么取名为 Alpha 通道,我觉得是因为这是除RGB以外「第一个通道」的意思,没有别的更深刻的含义。 「Alpha 通道」是图片内在的一个属性,用 css 或者其他外部方法 …
什么是指令集?CPU的指令集是怎么运作的?X86、ARM、MIPS …
5、DEC Alpha Alpha是DEC公司推出的RISC指令集系统,基于Alpha指令集的CPU也称为Alpha AXP架构,是64位的 RISC微处理器,最初由DEC公司制造,并被用于DEC自己的工作站和服 …
ɑ与a的区别是什么?是不是a是英文印刷体,而ɑ是汉语拼音字母? …
Feb 23, 2025 · 第二,从编码角度来说,你输入的「a」这个字符是 U+0061,在绝大部分字体中被视作正常的拉丁字母小写 a;「ɑ」这个字符是 U+0251,叫 Latin alpha。在 Unicode 眼中, …
什么是西格玛男人? - 知乎
与Alpha男性具备同等购买力的财富和外表,是过度自负的表现;而赋予自身“在系统之外不被理解”的孤独感,是自卑的外化。 “西格玛男性与incel有很多共同之处,他们普遍认为自己太聪明 …
统计学中的P值如何计算? - 知乎
分别为置信上限和置信下限。为什么是 \geqslant1-{\alpha} 当等于 1-{\alpha} 时,主要针对总体分布为连续型分析时,当大于 1-{\alpha} 针对总体分布为离散型分布时,一般来讲参数 \theta 的 …
有没有大神可以把力矩的物理意义讲清楚? - 知乎
对于平动,有 F=ma ,其中 F 为合外力, m 为惯性质量(即质量), a 为加速度;而对于转动,有 M=J\alpha ,其中 M 为合外力矩, J 为转动惯量, \alpha 为角加速度。 (对于转动也有 …
统计学假设检验中 p 值的含义具体是什么? - 知乎
若设定 \alpha=0.05 ,我们只有观测到“女士答对7次或者7次以上”时,才会拒绝原假设;若设定 \alpha=0.01 ,则只有当观测到“女士答对8次”时才会拒绝原假设。 可见,如果我们希望犯第I类 …
有没有前辈知道alphasights 这个公司怎么样?适合刚毕业的美本入 …
有没有前辈知道alphasights 这个公司怎么样?适合刚毕业的美本入行吗? - 知乎
2025年智能锁推荐,智能门锁怎么选?看这一篇就够了!
Jun 1, 2025 · 2、明确安装条件. 年货节选购智能门锁除了要看产品的参数,还要在买前了解自家门体的情况,确认是否符合安装智能锁的条件,否则有可能出现安装人员拆掉门锁后才发现无法 …
想了解omega,beta,alpha,ABO是什么意思,别人说的时候我都 …
alpha和omega数量稀缺。alpha通常体质、头脑都很好,是社会中的精英;omega则十分娇弱,但具有很强的生育能力,她们就类似古代的女人,一般不会被允许劳作,而作为珍贵的生育工具 …
为什么用 ‘Alpha’ 代表透明度? - 知乎
Aug 3, 2013 · 为什么取名为 Alpha 通道,我觉得是因为这是除RGB以外「第一个通道」的意思,没有别的更深刻的含义。 「Alpha 通道」是图片内在的一个属性,用 css 或者其他外部方法 …
什么是指令集?CPU的指令集是怎么运作的?X86、ARM、MIPS …
5、DEC Alpha Alpha是DEC公司推出的RISC指令集系统,基于Alpha指令集的CPU也称为Alpha AXP架构,是64位的 RISC微处理器,最初由DEC公司制造,并被用于DEC自己的工作站和服 …
ɑ与a的区别是什么?是不是a是英文印刷体,而ɑ是汉语拼音字母? …
Feb 23, 2025 · 第二,从编码角度来说,你输入的「a」这个字符是 U+0061,在绝大部分字体中被视作正常的拉丁字母小写 a;「ɑ」这个字符是 U+0251,叫 Latin alpha。在 Unicode 眼中, …
什么是西格玛男人? - 知乎
与Alpha男性具备同等购买力的财富和外表,是过度自负的表现;而赋予自身“在系统之外不被理解”的孤独感,是自卑的外化。 “西格玛男性与incel有很多共同之处,他们普遍认为自己太聪明 …
统计学中的P值如何计算? - 知乎
分别为置信上限和置信下限。为什么是 \geqslant1-{\alpha} 当等于 1-{\alpha} 时,主要针对总体分布为连续型分析时,当大于 1-{\alpha} 针对总体分布为离散型分布时,一般来讲参数 \theta 的 …
有没有大神可以把力矩的物理意义讲清楚? - 知乎
对于平动,有 F=ma ,其中 F 为合外力, m 为惯性质量(即质量), a 为加速度;而对于转动,有 M=J\alpha ,其中 M 为合外力矩, J 为转动惯量, \alpha 为角加速度。 (对于转动也 …
统计学假设检验中 p 值的含义具体是什么? - 知乎
若设定 \alpha=0.05 ,我们只有观测到“女士答对7次或者7次以上”时,才会拒绝原假设;若设定 \alpha=0.01 ,则只有当观测到“女士答对8次”时才会拒绝原假设。 可见,如果我们希望犯第I类 …
有没有前辈知道alphasights 这个公司怎么样?适合刚毕业的美本入 …
有没有前辈知道alphasights 这个公司怎么样?适合刚毕业的美本入行吗? - 知乎
2025年智能锁推荐,智能门锁怎么选?看这一篇就够了!
Jun 1, 2025 · 2、明确安装条件. 年货节选购智能门锁除了要看产品的参数,还要在买前了解自家门体的情况,确认是否符合安装智能锁的条件,否则有可能出现安装人员拆掉门锁后才发现无法 …