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Alpha-1 Antitrypsin Replacement Therapy: A Comprehensive Guide
Author: Dr. Emily Carter, MD, PhD. Dr. Carter is a pulmonologist with over 15 years of experience specializing in Alpha-1 Antitrypsin Deficiency (AATD) and its treatment, including extensive research and clinical trials involving alpha-1 antitrypsin replacement therapy.
Publisher: The American Lung Association (ALA). The ALA is a leading non-profit organization dedicated to lung health and disease prevention, with extensive expertise in respiratory illnesses, including AATD and its treatment options.
Editor: Dr. Robert Jones, MD. Dr. Jones is a board-certified pulmonologist and respiratory critical care specialist with over 20 years of experience in managing patients with AATD and extensive knowledge of alpha-1 antitrypsin replacement therapy.
Summary: This guide provides a comprehensive overview of alpha-1 antitrypsin replacement therapy, detailing its mechanisms, benefits, risks, and best practices for successful implementation. It addresses patient selection criteria, administration techniques, monitoring strategies, and potential complications. The guide also highlights common pitfalls to avoid and emphasizes the importance of individualized treatment plans tailored to each patient's specific needs and disease progression.
Keywords: alpha-1 antitrypsin replacement therapy, AAT replacement therapy, Alpha-1 antitrypsin deficiency, AATD treatment, augmentation therapy, intravenous AAT, prophylactic treatment, lung disease, liver disease, AAT deficiency treatment
1. Understanding Alpha-1 Antitrypsin Deficiency and the Need for Replacement Therapy
Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder characterized by a deficiency of the alpha-1 antitrypsin (AAT) protein, a crucial protease inhibitor protecting the lungs from damage. The lack of AAT leads to an increased risk of developing severe emphysema and other lung diseases, as well as liver damage. Alpha-1 antitrypsin replacement therapy aims to supplement the deficient AAT levels, mitigating the progression of these debilitating conditions.
2. Mechanisms of Alpha-1 Antitrypsin Replacement Therapy
Alpha-1 antitrypsin replacement therapy involves the intravenous infusion of purified human AAT derived from pooled plasma donations. This exogenous AAT compensates for the body's insufficient endogenous production, providing protease inhibition and reducing lung tissue destruction. The therapy does not cure AATD but aims to slow disease progression and improve lung function.
3. Patient Selection for Alpha-1 Antitrypsin Replacement Therapy
Not all individuals with AATD are candidates for replacement therapy. Selection criteria generally include:
Severe AATD: Individuals with severe AAT deficiency (typically ZZ genotype) and significant lung disease progression.
Moderate to Severe Emphysema: Patients demonstrating measurable airflow limitation and evidence of emphysema on imaging studies.
Absence of Contraindications: Patients without significant cardiovascular, renal, or other conditions that might compromise the safety of intravenous infusions.
4. Administration and Monitoring of Alpha-1 Antitrypsin Replacement Therapy
Alpha-1 antitrypsin replacement therapy involves regular intravenous infusions, typically administered weekly or bi-weekly. Dosage is tailored to the individual's needs and closely monitored. Regular assessment includes:
Lung Function Tests: Monitoring changes in FEV1 (forced expiratory volume in 1 second) and other respiratory parameters.
Imaging Studies: Periodic chest CT scans to evaluate emphysema progression.
Safety Monitoring: Vigilance for infusion-related reactions (e.g., allergic reactions, hypotension) and potential adverse effects.
5. Benefits and Risks of Alpha-1 Antitrypsin Replacement Therapy
Benefits:
Slowing Disease Progression: Reduction in the rate of decline in lung function.
Improved Quality of Life: Increased exercise capacity and reduced shortness of breath.
Reduced Hospitalizations: Decreased frequency of respiratory exacerbations.
Risks:
Infusion Reactions: Allergic reactions, fever, chills, or hypotension.
Transmission of Bloodborne Diseases: Though highly unlikely due to rigorous screening and processing of donated plasma.
Cost: Alpha-1 antitrypsin replacement therapy can be expensive.
6. Best Practices for Alpha-1 Antitrypsin Replacement Therapy
Individualized Treatment Plans: Treatment should be customized based on the patient's specific clinical characteristics, disease severity, and response to therapy.
Multidisciplinary Approach: Collaboration between pulmonologists, respiratory therapists, and other healthcare professionals is essential.
Patient Education: Thorough patient education is crucial for understanding the treatment regimen, potential side effects, and self-management strategies.
Adherence to Treatment: Consistent adherence to the prescribed infusion schedule is vital for optimal outcomes.
7. Common Pitfalls to Avoid in Alpha-1 Antitrypsin Replacement Therapy
Delayed Initiation of Therapy: Early intervention can significantly improve treatment outcomes.
Insufficient Monitoring: Regular monitoring is essential for early detection and management of complications.
Poor Patient Adherence: Non-compliance with the treatment regimen can diminish therapeutic benefits.
Ignoring Individual Patient Needs: Treatment should be tailored to the specific characteristics of each patient.
8. Emerging Advances in Alpha-1 Antitrypsin Replacement Therapy
Research is ongoing to develop more effective and accessible alpha-1 antitrypsin replacement therapies. This includes exploration of novel delivery methods, improved purification techniques, and potentially even gene therapy approaches to address the underlying genetic defect.
9. Conclusion
Alpha-1 antitrypsin replacement therapy represents a significant advance in the management of AATD. While not a cure, it provides a valuable means to slow disease progression, improve lung function, and enhance quality of life for individuals with severe AATD. Effective implementation requires careful patient selection, close monitoring, and a multidisciplinary approach that prioritizes individual patient needs and maximizes adherence to the prescribed treatment regimen. Early intervention and ongoing research promise even greater advancements in this area.
FAQs
1. How is Alpha-1 antitrypsin replacement therapy administered? It's administered intravenously through regular infusions, typically weekly or bi-weekly, depending on the individual's needs and response.
2. What are the common side effects of Alpha-1 antitrypsin replacement therapy? Common side effects can include infusion reactions (allergic reactions, fever, chills, hypotension), but these are relatively rare.
3. Who is a candidate for Alpha-1 antitrypsin replacement therapy? Patients with severe AATD (typically ZZ genotype) and significant lung disease progression are usually candidates.
4. How long does Alpha-1 antitrypsin replacement therapy last? It's a long-term therapy, often continuing for several years or the patient's lifetime.
5. Is Alpha-1 antitrypsin replacement therapy covered by insurance? Coverage varies depending on the insurance provider and individual circumstances. It's important to check with your insurance company.
6. What are the long-term benefits of Alpha-1 antitrypsin replacement therapy? Long-term benefits include slowing lung disease progression, improving quality of life, and potentially reducing hospitalizations.
7. How is the effectiveness of Alpha-1 antitrypsin replacement therapy monitored? Effectiveness is monitored through regular lung function tests (spirometry), imaging (CT scans), and assessment of symptoms.
8. What are the alternative treatments for Alpha-1 antitrypsin deficiency? Besides replacement therapy, other treatments focus on managing symptoms (e.g., bronchodilators, pulmonary rehabilitation) and preventing complications.
9. What are the costs associated with Alpha-1 antitrypsin replacement therapy? The cost can be substantial due to the ongoing infusions and monitoring, but many insurance plans offer coverage.
Related Articles
1. "The Efficacy of Alpha-1 Antitrypsin Augmentation Therapy in Slowing Emphysema Progression": This article focuses on clinical trial data evaluating the effectiveness of AAT replacement therapy in reducing emphysema progression rates.
2. "Long-Term Safety and Tolerability of Alpha-1 Antitrypsin Replacement Therapy": This article analyzes long-term safety data and adverse events associated with prolonged use of AAT replacement therapy.
3. "Cost-Effectiveness Analysis of Alpha-1 Antitrypsin Replacement Therapy in AATD Patients": This article provides a detailed economic evaluation of the cost-effectiveness of AAT replacement therapy compared to alternative management strategies.
4. "Patient Selection Criteria for Alpha-1 Antitrypsin Replacement Therapy: A Consensus Statement": This article outlines the consensus recommendations for patient selection criteria for AAT replacement therapy based on current clinical guidelines.
5. "Managing Infusion Reactions Associated with Alpha-1 Antitrypsin Replacement Therapy": This article offers practical guidance on recognizing, preventing, and managing infusion reactions during AAT replacement therapy.
6. "The Role of Pulmonary Rehabilitation in Conjunction with Alpha-1 Antitrypsin Replacement Therapy": This article explores the synergistic benefits of combining AAT replacement therapy with pulmonary rehabilitation programs.
7. "Genetic Testing and Early Diagnosis of Alpha-1 Antitrypsin Deficiency": This article highlights the importance of early genetic screening and diagnosis of AATD for timely intervention.
8. "Advances in the Purification and Production of Alpha-1 Antitrypsin for Replacement Therapy": This article examines technological advancements in AAT production methods aimed at improving efficacy and safety.
9. "Emerging Gene Therapy Approaches for the Treatment of Alpha-1 Antitrypsin Deficiency": This article reviews the latest research and development efforts in gene therapy approaches to cure AATD.
| alpha 1 antitrypsin replacement 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 antitrypsin replacement 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 antitrypsin replacement therapy: Immunopharmacology Manzoor M. Khan, 2008-12-19 During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology. |
| alpha 1 antitrypsin replacement 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 antitrypsin replacement 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 antitrypsin replacement therapy: Practical Emergency Resuscitation and Critical Care Kaushal Shah, Jarone Lee, Clark G. Owyang, Benjamin Christian Renne, 2023-10-31 The second edition of a succinct and portable text reviewing the clinical approach to emergency medicine and critical care. |
| alpha 1 antitrypsin replacement therapy: Liver Disease in Children Frederick J. Suchy, Ronald J. Sokol, William F. Balistreri, 2007-05-07 Completely revised new edition of the premier reference on pediatric liver disease. Liver Disease in Children, 3rd Edition provides authoritative coverage of every aspect of liver disease affecting infants, children, and adolescents. The book offers an integrated approach to the science and clinical practice of pediatric hepatology and charts the substantial progress in understanding and treating these diseases. Chapters are written by international experts and address the unique pathophysiology, manifestations, and management of these disorders in the pediatric population. The third edition has been thoroughly updated and features new contributions on liver development, cholestatic and autoimmune disorders, fatty liver disease, and inborn errors of metabolism. With the continued evolution of pediatric hepatology as a discipline, this text remains an essential reference for all physicians involved in the care of children with liver disease. |
| alpha 1 antitrypsin replacement 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 antitrypsin replacement 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 antitrypsin replacement therapy: Trauma Induced Coagulopathy Hunter B. Moore, Matthew D. Neal, Ernest E. Moore, 2020-10-12 The first edition of this publication was aimed at defining the current concepts of trauma induced coagulopathy by critically analyzing the most up-to-date studies from a clinical and basic science perspective. It served as a reference source for any clinician interested in reviewing the pathophysiology, diagnosis, and management of the coagulopathic trauma patient, and the data that supports it. By meticulously describing the methodology of most traditional as well as state of the art coagulation assays the reader is provided with a full understanding of the tests that are used to study trauma induced coagulopathy. With the growing interest in understanding and managing coagulation in trauma, this second edition has been expanded to 46 chapters from its original 35 to incorporate the massive global efforts in understanding, diagnosing, and treating trauma induced coagulopathy. The evolving use of blood products as well as recently introduced hemostatic medications is reviewed in detail. The text provides therapeutic strategies to treat specific coagulation abnormalities following severe injury, which goes beyond the first edition that largely was based on describing the mechanisms causing coagulation abnormalities. Trauma Induced Coagulopathy 2nd Edition is a valuable reference to clinicians that are faced with specific clinical challenges when managing coagulopathy. |
| alpha 1 antitrypsin replacement therapy: A New Approach to Research Ethics Henriikka Mustajoki, Arto Mustajoki, 2017-03-16 A New Approach to Research Ethics is a clear, practical and useful guide to the ethical issues faced by researchers today. Examining the theories of ethical decision-making and applying these theories to a range of situations within a research career and process, this text offers a broader perspective on how ethics can be a positive force in strengthening the research community. Drawing upon a strong selection of challenging case studies, this text offers a new approach to engage with ethical issues and provides the reader with: a broader view on research ethics in practice, capturing both different stages of research careers and multiple tasks within that career, including supervision and research assessments thoughts on questions such as increasing globalisation, open science and intensified competition an increased understanding of undertaking research in a world of new technologies an extension of research ethics to a multidisciplinary and interdisciplinary approach an introduction to a ‘guided dialogue’ method, which helps to identify and engage with ethical issues individually and as a research community. A New Approach to Research Ethics allows for self-reflection and provides guidance for professional development in an increasingly competitive area. Full of valuable guidance for the researcher and ethical decision-maker, this is an essential text for postgraduate students, senior academics and developers of training courses on ethics for researchers. |
| alpha 1 antitrypsin replacement therapy: Disease Control Priorities in Developing Countries Dean T. Jamison, Joel G. Breman, Anthony R. Measham, George Alleyne, Mariam Claeson, David B. Evans, Prabhat Jha, Anne Mills, Philip Musgrove, 2006-04-02 Based on careful analysis of burden of disease and the costs ofinterventions, this second edition of 'Disease Control Priorities in Developing Countries, 2nd edition' highlights achievable priorities; measures progresstoward providing efficient, equitable care; promotes cost-effectiveinterventions to targeted populations; and encourages integrated effortsto optimize health. Nearly 500 experts - scientists, epidemiologists, health economists,academicians, and public health practitioners - from around the worldcontributed to the data sources and methodologies, and identifiedchallenges and priorities, resulting in this integrated, comprehensivereference volume on the state of health in developing countries. |
| alpha 1 antitrypsin replacement therapy: Lung Epithelial Biology in the Pathogenesis of Pulmonary Disease Venkataramana K Sidhaye, Michael Koval, 2017-03-09 Lung Epithelial Biology in the Pathogenesis of Pulmonary Disease provides a one-stop resource capturing developments in lung epithelial biology related to basic physiology, pathophysiology, and links to human disease. The book provides access to knowledge of molecular and cellular aspects of lung homeostasis and repair, including the molecular basis of lung epithelial intercellular communication and lung epithelial channels and transporters. Also included is coverage of lung epithelial biology as it relates to fluid balance, basic ion/fluid molecular processes, and human disease. Useful to physician and clinical scientists, the contents of this book compile the important and most current findings about the role of epithelial cells in lung disease. Medical and graduate students, postdoctoral and clinical fellows, as well as clinicians interested in the mechanistic basis for lung disease will benefit from the books examination of principles of lung epithelium functions in physiological condition. - Provides a single source of information on lung epithelial junctions and transporters - Discusses of the role of the epithelium in lung homeostasis and disease - Includes capsule summaries of main conclusions as well as highlights of future directions in the field - Covers the mechanistic basis for lung disease for a range of audiences |
| alpha 1 antitrypsin replacement therapy: Alpha-1 Antitrypsin Deficiency Thomas Köhnlein, T. Welte, 2007 |
| alpha 1 antitrypsin replacement 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 antitrypsin replacement 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 antitrypsin replacement therapy: Pediatric Hepatology and Liver Transplantation Lorenzo D'Antiga, 2019-04-29 This book is the first to provide balanced examination of both pediatric liver disease and liver transplantation – two topics that are inherently related, given that most chronic liver disorders eventually require organ replacement. The different forms of liver disease encountered in the pediatric age group are first discussed in a series of disease-specific chapters that have a reader-friendly, uniform structure covering pathophysiology, diagnostic and treatment algorithms, clinical cases, and transition to adult care. Key topics in the field of liver transplantation are then addressed. Examples include indications and contraindications, surgical techniques and complications, immunosuppression, in pediatric liver transplantation, acute and chronic rejection and allograft dysfunction, and CMV and EBV infection in transplant recipients, long-term graft injury and tolerance. A section on pediatric hepatology across the world includes chapters presenting the features and management of pediatric liver disease in South-America, Africa and Asia. A closing section considers what the future holds for pediatric liver disease and its management, including novel genetic testing, cell therapy and gene therapy. Pediatric Hepatology and Liver Transplantation will be of value for a range of practitioners, from residents making their first approach to pediatric liver disease through to specialists working in transplantation centers. |
| alpha 1 antitrypsin replacement therapy: Mechanisms of Vascular Disease Robert Fitridge, M. M. Thompson, 2011 New updated edition first published with Cambridge University Press. This new edition includes 29 chapters on topics as diverse as pathophysiology of atherosclerosis, vascular haemodynamics, haemostasis, thrombophilia and post-amputation pain syndromes. |
| alpha 1 antitrypsin replacement therapy: Combined Liver-kidney Transplantation Martin Erlichman, 1995 |
| alpha 1 antitrypsin replacement 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 antitrypsin replacement therapy: Lung Obstruction Among Adults Aged 40-79 Timothy Tilert, Ryne Paulose-Ram, Debra J. Brody, 2015 |
| alpha 1 antitrypsin replacement therapy: Rare Diseases and Orphan Products Institute of Medicine, Board on Health Sciences Policy, Committee on Accelerating Rare Diseases Research and Orphan Product Development, 2011-04-03 Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development. |
| alpha 1 antitrypsin replacement therapy: The Serpin Family Margarethe Geiger, Felix Wahlmüller, Margareta Furtmüller, 2015-11-13 The book provides an comprehensive overview on biology, genetics and cellular functions of serpins (serine protease inhibitors) in health and disease. With over 1000 members serpins are the most diverse family of protease inhibitors. Latest groundbreaking research findings are presented and broaden the understanding on inhibitory and non-inhibitory serpins, not only in mammalian organisms but also in insects, worms, plants and viruses. |
| alpha 1 antitrypsin replacement therapy: Extraordinary! A Book for Children with Rare Diseases (Mandarin) Evren And Kara Ayik, 2022-03-03 是什么让罕见病儿童非同寻常？ 聆听罕见病儿童埃夫伦自我成长的心路历程，探索问题答案！ 本书由母子联手创作，基于罕见病患者面临的挑战与机遇，就身份、包容与自我认知展开适合孩子的讨论。书中的经验全部是小作者作为超罕见病患者一路长大的亲身经历，旨在为小读者理解个人身份和罕见病对个人身份的正面影响提供指南。家庭成员和护理人员也可以积极参与孩子的探索过程，根据孩子的成长需求制定读书计划。 本书插图由获奖童书插画家伊恩-戴尔绘制。《非同寻常的你！》旨在通过其由衷的文字和敏感又真实的插画鼓舞、激励所有患有罕见病的孩子活出最美好的一生。 What makes a child with a rare disease extraordinary? Explore the answer to this question while sharing a conversation with Evren about what he has learned while growing up with his own rare disease. Written collaboratively by mother and son, this book opens up a child-friendly discussion about identity, inclusion, and self-concept in light of the challenges and silver linings of living with a rare disease. The gentle lessons draw on the co-author's first-hand experience of growing up with an ultra-rare disease and offer young readers a framework for understanding personal identity and how their rare diseases can help shape it in positive ways. Family members and caregivers are invited to share in this conversation and to customize the reading according to each young reader's developmental needs. Accompanied by sensitive yet realistic illustrations created by award-winning artist and children's book illustrator Ian Dale, the heartfelt messages introduced in Extraordinary! are intended to uplift and encourage any children living with rare diseases to live their very best lives. |
| alpha 1 antitrypsin replacement therapy: Inhaled Medicines Stavros Kassinos, Per Bäckman, Joy Conway, Anthony J. J. Hickey, 2021-02-05 Inhaled medicines are widely used to treat pulmonary and systemic diseases. The efficacy and safety of these medicines can be influenced by the deposited fraction, the regional deposition pattern within the lungs and by post-depositional events such as drug dissolution, absorption and clearance from the lungs. Optimizing performance of treatments thus requires that we understand and are able to quantify these product and drug attributes. Inhaled Medicines: Optimizing Development through Integration of In Silico, In Vitro and In Vivo Approaches explores the current state of the art with respect to inhalation drug delivery, technologies available to assess product performance, and novel in silico methods now available to link in vitro product performance to clinical performance. Recent developments in the latter field, especially the prospect of integration of three-dimensional Computational Fluid Particle Methods (3D-CFPD) with physiologically based pharmacokinetic (PBPK models), unlocks the potential for in silico population studies that can help inform and optimize treatment and product development strategies. In this highly multidisciplinary field, where progress occurs at the intersection of several disciplines of engineering and science, this work aims to integrate current knowledge and understanding and to articulate a clear vision for future developments. ? Considers the healthcare needs driving the field, and where inhaled drugs could have the maximum impact ? Gives a concise account of the state of the art in key areas and technologies such as device and formulation technologies, clinically relevant in vitro performance assessment, medical imaging, as well as in silico modelling and simulation ? Articulates how the combination of in vitro product performance data, medical imaging and simulations technologies in the framework of large scale in silico pre-clinical trials could revolutionize the field ? Provides systematic and thorough referencing to sources offering a more-in-depth analysis of technical issues |
| alpha 1 antitrypsin replacement therapy: Split liver transplantation X. Rogiers, H. Bismuth, R.W. Busuttil, D.C. Broering, D. Azoulay, 2002-08 Aimed at the trainee surgeon and experienced transplant surgeon, this compendium on split-liver grafting contains articles written by faculty members of the first International Course on Split-Liver Transplantation. It covers the main aspects of the field and is geared towards helping surgeons select the best surgical techniques as well as identifying the pitfalls. The text features detailed instructions on the various procedures as well as an overview of the area. |
| alpha 1 antitrypsin replacement therapy: Chronic Obstructive Pulmonary Disease Exacerbations Jadwiga A. Wedzicha, Fernando J. Martinez, 2008-09-22 Chronic Obstructive Pulmonary Disease Exacerbations covers the definition, diagnosis, epidemiology, mechanisms, and treatment associated with COPD exacerbations. This text also addresses imaging and how it plays a pivotal role in the diagnosis and study of exacerbations.Written by today's top experts, Chronic Obstructive Pulmonary Disease Exacerbat |
| alpha 1 antitrypsin replacement therapy: Gene Therapy Applications Chunsheng Kang, 2011-08-23 The aim of our book is to provide a detailed discussion of gene therapy application in human diseases. The book brings together major approaches: (1) Gene therapy in blood and vascular system, (2) Gene therapy in orthopedics, (3) Gene therapy in genitourinary system, (4) Gene therapy in other diseases. This source will make clinicians and researchers comfortable with the potential and problems of gene therapy application. |
| alpha 1 antitrypsin replacement therapy: Biology of Serpins James Whisstock, Phillip Bird, 2011-07-18 Serpins are a group of proteins with similar structures that were first identified as a set of proteins able to inhibit proteases. The acronym serpin was originally coined because many serpins inhibit chymotrypsin-like serine proteases. This volume of Methods in Ezymology is split into 2 parts and comprehensively covers the subject. |
| alpha 1 antitrypsin replacement therapy: Medical-Surgical Nursing - Single-Volume Text and Elsevier Adaptive Learning Package Sharon L. Lewis, Shannon Ruff Dirksen, Margaret M. Heitkemper, Linda Bucher, 2014-06-17 Corresponding chapter-by-chapter to Medical-Surgical Nursing, 9e, Elsevier Adaptive Learning combines the power of brain science with sophisticated, patented Cerego algorithms to help you learn faster and remember longer. It's fun; it's engaging; and it's constantly tracking your performance and adapting to deliver content precisely when it's needed to ensure core information is transformed into lasting knowledge. Please refer to the individual product pages for the duration of access to these products. An individual study schedule reduces cognitive workload and helps you become a more effective learner by automatically guiding the learning and review process. The mobile app offers a seamless learning experience between your smartphone and the web with your memory profile maintained and managed in the cloud. UNIQUE! Your memory strength is profiled at the course, chapter, and item level to identify personal learning and forgetting patterns. UNIQUE! Material is re-presented just before you would naturally forget it to counteract memory decay. A personalized learning pathway is established based on your learning profile, memory map, and time required to demonstrate information mastery. The comprehensive student dashboard allows you to view your personal learning progress. |
| alpha 1 antitrypsin replacement therapy: Alpha 1 - Antitrypsin Deficiency Ronald G. Crystal, 2014-07-22 Providing a broad overview of basic and clinical aspects of alpha 1-antitrypsin (a 1AT) deficiency, this up-to-date reference discusses the complex pathobiological processes underlying the pathogenesis of a1AT deficiency, describes the a1AT gene and its promoter, and details specific therapies to prevent the major clinical manifestations of the dis |
| alpha 1 antitrypsin replacement 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 antitrypsin replacement 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 antitrypsin replacement therapy: Epidemiology David Duncan, 1987 |
| alpha 1 antitrypsin replacement therapy: Pocket Guide to COPD Diagnosis, Management and Prevention Global Initiative for Chronic Obstructive Lung Disease, 2017-11-15 Chronic Obstructive Pulmonary Disease (COPD) represents an important public health challenge and is a major cause of chronic morbidity and mortality throughout the world. COPD is currently the fourth leading cause of death in the world1 but is projected to be the 3rd leading cause of death by 2020. More than 3 million people died of COPD in 2012 accounting for 6% of all deaths globally. Globally, the COPD burden is projected to increase in coming decades because of continued exposure to COPD risk factors and aging of the population.2 This Pocket Guide has been developed from the Global Strategy for the Diagnosis, Management, and Prevention of COPD (2018 Report), which aims to provide a non-biased review of the current evidence for the assessment, diagnosis and treatment of patients with COPD that can aid the clinician. Discussions of COPD and COPD management, evidence levels, and specific citations from the scientific literature are included in that source document, which is available from www.goldcopd.org. The tables and figures in this Pocket Guide follow the numbering of the 2018 Global Strategy Report for reference consistency. |
| alpha 1 antitrypsin replacement 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 antitrypsin replacement therapy: Questions from NeoReviews Henry C. Lee, Santina A. Zanelli, Dara Brodsky, 2020 Enhance your knowledge of neonatal-perinatal medicine and/or study for Neonatal-Perinatal Medicine board certification or recertification with this new study guide from the editors of NeoReviews. This new guide includes more than 1,200 questions previously published in NeoReviews from January 2007 to December 2017. Each question is followed by a short explanation of the correct answer with references, including the original article. Chapters include Cardiology Dermatology Endocrinology ENT and Ophthalmology Fluids, Electrolytes, Nutrition Gastrointestinal Genetics and Inborn Errors of Metabolism Hematology/Oncology Immunology Infectious Diseases Maternal-Fetal Medicine Neonatal Resuscitation Neurology Renal Respiratory Statistics, Research, Health Services, and Ethics |
| alpha 1 antitrypsin replacement therapy: Understanding Alpha-1 Antitrypsin Deficiency , 1994 |
| alpha 1 antitrypsin replacement therapy: Report of the Director of the National Heart, Lung, and Blood Institute National Heart, Lung, and Blood Institute, 1983 |
| alpha 1 antitrypsin replacement therapy: Oxford Textbook of Medicine David A. Warrell, 2003 The Oxford Textbook of Medicine provides all that any doctor needs to know to practice top-level internal medicine. It gives compreensive coverage of the epidemiology, aetiology, and mechanism of disease, as well as clear, unambiguous coverage of the diagnosis, practical management and prevention of the entire spectrum of medical disorders. There are major introductory sections on the scientific basis of disease; and in the system-based clinical sections genetic predisposition, pathophysiology, pathogenesis, molecular mechanisms, and cell biology are covered in depth for all significant medical syndromes. Clinical descriptions of diseases are clearly and memorably written, based on the experience and insight of the authors--many of whom are among the world's most distinguished medical scientists. Chapters are not only evidence based but also on clinical experience and a thorough survey of all the relevant literature. Throughout, the approach of OTM is humane and ethical and, at the same time, factual, reliable, honest(especially where knowledge is limited) and rigorously scientific. This is not just a textbook of First World medicine. It provides practical guidance for doctors working in a variety of medical setting the value of a logical clinical approach rather than immediate resort to expensive imaging and laboratory tests. Moore of the contributing authors than ever before are from outside Europe, including strong representation of North American medicine. The new editorial team has ensured that the OTM continues to reflect rapid changes in medical practice: there are new sections on intensive care, alcohol and drug abuse, clinical pharmacology and therapeutics, world health, clinical trials and evidence-based medicine, adolescent medicine, sports medicine, and emergency medicine; more than half the contributors are new for this edition; and most of the text has been heavily revised. The striking new page and cover design reflect the significant changes made in this new edition. The Textbook is illustrated by over 2000 two-color diagrams and many color Plates. The index is the most detailed and user-friendly of any major medical textbook: in an emergency, the reader can access information quickly- whether on the ward, in office or at home. Like its predecessors, OTM4 will be the trusted and ultimate reference in libraries, hospitals, doctors' consulting rooms, solicitors' offices, press offices, and primary care practices worldwide. |
Alpha-1 Antitrypsin Deficiency - CHEST
Augmentation therapy, approved decades ago on the basis of plasma levels of alpha-1 antitrypsin, is the only approved therapy for AATD. Limited quantitative understanding of the longitudinal …
DESCRIPTION: - provider.excellusbcbs.com
Prolastin-C®, Aralast NP®, Zemaira®, and Glassia® are all FDA approved agents for use as replacement therapy in congenital alpha-1 antitrypsin (AAT) deficiency with clinical emphysema.
Alpha-1 Antitrypsin Deficiency A Guide for the Recently …
Alpha-1 Can Cause: -like symptoms, chronic bronchitis, emphysema, or bronchiectasis. These problems, usually seen in adults, are caused by the AAT protein deficiency and are commonly …
Alpha-1 antitrypsin deficiency targeted testing and …
Alpha-1 antitrypsin (A1AT) functions primarily to inhibit neutrophil elastase, and deficiency predisposes individuals to the development of chronic obstructive pulmonary disease (COPD). …
Prolastin - Alpha-1 Proteinase Inhibitor (Human)
Alpha1-Proteinase Inhibitor (Human), Prolastin is indicated for chronic replacement therapy of individuals having congenital deficiency of alpha1-PI (alpha1-antitrypsin deficiency) with...
Alpha-1 antitrypsin deficiency and replacement therapy – …
Alpha-1 antitrypsin deficiency and replacement therapy – Current developments and clinical significance/ Andreas Rembert Koczulla.-1st edition - Bremen: UNI-MED, 2021 (UNI-MED …
Systemic modified messenger RNA for replacement therapy in …
Alpha 1-antitrypsin (AAT) deciency arises from an inherited mutation in the SERPINA1 gene. The disease causes damage in the liver where the majority of the AAT protein is produced.
Treatment for Alpha-1 Antitrypsin Deficiency: Does …
Sep 19, 2023 · EDITORIALS Treatment for Alpha-1 Antitrypsin Deficiency Does Augmentation Therapy Work? Alpha-1 antitrypsin deficiency (AATD) is a rare inherited cause of chronic …
Alpha -Antitrypsin Replacement Therapy Controls 1 …
ABSTRACT. Two Spanish sisters with α 1 -antitrypsin (AAT) deficiency and fibromyalgia (FM) started AAT replacement therapy with commercial α 1 -antitrypsin infusions in 1992. They both …
SPECIALTY GUIDELINE MANAGEMENT Alpha1-Proteinase …
CONTINUATION OF THERAPY Authorization of 12 months may be granted for continued treatment of emphysema due to alpha1-antitrypsin (AAT) deficiency when the member is experiencing …
Alpha-1 Antitrypsin Deficiency [Aralast NP, Glassia, Prolastin, …
Prolastin® Prolastin is indicated for chronic replacement therapy of individuals having congenital deficiency of alpha -PI (alpha antitrypsin deficiency) with clinically demonstrable panacinar …
CRISPR/Cas9-Mediated Targeted Gene Insertion of SERPINA1 …
Dec 12, 2020 · CRISPR/Cas9-Mediated Targeted Gene Insertion of SERPINA1 to Treat Alpha-1 Antitrypsin Deficiency Alpha-1 Foundation Workshop: “The Promise of Gene-Based Interventions …
Updates in the Management of Alpha-1 Antitrypsin
Alpha-1 chronic obstructive pulmonary disease in both never- and active-smokers. Short of a curative approach, alpha-1 antitrypsin deficiency lung disease benefits from standard medical …
Consequences of Abrupt Cessation of Alpha1-Antitrypsin …
Abrupt cessation of a long-term biologic therapy in compromised persons was associated with poor health outcomes. This lesson should not be lost on researchers or policy-makers.
Alpha-1-Antitrypsin Deficiency Targeted Testing and …
ciency clinical practice guideline, new approaches to optimal diagnosis using modern genetic testing and studies of A1AT augmentation therapy have been published. We performed a sys …
Microsoft Word - Alpha1 Stand-Alone Protocol_4_2017.docx
AAT replacement therapy is the standard of care for AATD in the U.S. and was FDA-approved because it was shown to restore AAT in the lung to normal levels.39 Further evidence for its use …
Replacement Therapy for Hereditary Alpha1-Antitrypsin …
Thir teen of 14 patients bad no decline in pulmonary Augmentation therapy with human alpha1-anti trypsin (AA T) for individuals with hereditary AA T deficiency, PiZ phenotype, and emphysema …
MEDICAL POLICY - ALPHA-1 PROTEINASE INHIBITORS
Replacement therapies have not been shown to prevent or reverse emphysema in AATD; however, data has shown that treatment of symptomatic patients with low serum levels of AAT due to …
Antitrypsin Deficiency MCP042 - Molina Healthcare
Prolastin® Prolastin is indicated for chronic replacement therapy of individuals having congenital deficiency of alpha -PI (alpha - 1 1 antitrypsin deficiency) with clinically demonstrable panacinar …
Microsoft Word - PR Grifols - FDA aproval A1AT LIQUID 03.docx
Sep 22, 2017 · Grifols (MCE: GRF, MCE: GRF.P and NASDAQ:GRFS) announced the approval from the U.S. Food and Drug Administration (FDA) for a liquid formulation of its alpha-1 antitrypsin …
Alpha-1 Antitrypsin Deficiency - CHEST
Augmentation therapy, approved decades ago on the basis of plasma levels of alpha-1 antitrypsin, is the only approved therapy for AATD. Limited quantitative understanding of the …
DESCRIPTION: - provider.excellusbcbs.com
Prolastin-C®, Aralast NP®, Zemaira®, and Glassia® are all FDA approved agents for use as replacement therapy in congenital alpha-1 antitrypsin (AAT) deficiency with clinical emphysema.
Alpha-1 Antitrypsin Deficiency A Guide for the Recently …
Alpha-1 Can Cause: -like symptoms, chronic bronchitis, emphysema, or bronchiectasis. These problems, usually seen in adults, are caused by the AAT protein deficiency and are commonly …
Alpha-1 antitrypsin deficiency targeted testing and …
Alpha-1 antitrypsin (A1AT) functions primarily to inhibit neutrophil elastase, and deficiency predisposes individuals to the development of chronic obstructive pulmonary disease (COPD). …
Prolastin - Alpha-1 Proteinase Inhibitor (Human)
Alpha1-Proteinase Inhibitor (Human), Prolastin is indicated for chronic replacement therapy of individuals having congenital deficiency of alpha1-PI (alpha1-antitrypsin deficiency) with...
Alpha-1 antitrypsin deficiency and replacement therapy – …
Alpha-1 antitrypsin deficiency and replacement therapy – Current developments and clinical significance/ Andreas Rembert Koczulla.-1st edition - Bremen: UNI-MED, 2021 (UNI-MED …
Systemic modified messenger RNA for replacement therapy …
Alpha 1-antitrypsin (AAT) deciency arises from an inherited mutation in the SERPINA1 gene. The disease causes damage in the liver where the majority of the AAT protein is produced.
Treatment for Alpha-1 Antitrypsin Deficiency: Does …
Sep 19, 2023 · EDITORIALS Treatment for Alpha-1 Antitrypsin Deficiency Does Augmentation Therapy Work? Alpha-1 antitrypsin deficiency (AATD) is a rare inherited cause of chronic …
Alpha -Antitrypsin Replacement Therapy Controls 1 …
ABSTRACT. Two Spanish sisters with α 1 -antitrypsin (AAT) deficiency and fibromyalgia (FM) started AAT replacement therapy with commercial α 1 -antitrypsin infusions in 1992. They both …
SPECIALTY GUIDELINE MANAGEMENT Alpha1-Proteinase …
CONTINUATION OF THERAPY Authorization of 12 months may be granted for continued treatment of emphysema due to alpha1-antitrypsin (AAT) deficiency when the member is …
Alpha-1 Antitrypsin Deficiency [Aralast NP, Glassia, Prolastin, …
Prolastin® Prolastin is indicated for chronic replacement therapy of individuals having congenital deficiency of alpha -PI (alpha antitrypsin deficiency) with clinically demonstrable panacinar …
CRISPR/Cas9-Mediated Targeted Gene Insertion of SERPINA1 …
Dec 12, 2020 · CRISPR/Cas9-Mediated Targeted Gene Insertion of SERPINA1 to Treat Alpha-1 Antitrypsin Deficiency Alpha-1 Foundation Workshop: “The Promise of Gene-Based …
Updates in the Management of Alpha-1 Antitrypsin
Alpha-1 chronic obstructive pulmonary disease in both never- and active-smokers. Short of a curative approach, alpha-1 antitrypsin deficiency lung disease benefits from standard medical …
Consequences of Abrupt Cessation of Alpha1-Antitrypsin …
Abrupt cessation of a long-term biologic therapy in compromised persons was associated with poor health outcomes. This lesson should not be lost on researchers or policy-makers.
Alpha-1-Antitrypsin Deficiency Targeted Testing and …
ciency clinical practice guideline, new approaches to optimal diagnosis using modern genetic testing and studies of A1AT augmentation therapy have been published. We performed a sys …
Microsoft Word - Alpha1 Stand-Alone Protocol_4_2017.docx
AAT replacement therapy is the standard of care for AATD in the U.S. and was FDA-approved because it was shown to restore AAT in the lung to normal levels.39 Further evidence for its …
Replacement Therapy for Hereditary Alpha1-Antitrypsin …
Thir teen of 14 patients bad no decline in pulmonary Augmentation therapy with human alpha1-anti trypsin (AA T) for individuals with hereditary AA T deficiency, PiZ phenotype, and …
MEDICAL POLICY - ALPHA-1 PROTEINASE INHIBITORS - BCBSM
Replacement therapies have not been shown to prevent or reverse emphysema in AATD; however, data has shown that treatment of symptomatic patients with low serum levels of AAT …
Antitrypsin Deficiency MCP042 - Molina Healthcare
Prolastin® Prolastin is indicated for chronic replacement therapy of individuals having congenital deficiency of alpha -PI (alpha - 1 1 antitrypsin deficiency) with clinically demonstrable …
Microsoft Word - PR Grifols - FDA aproval A1AT LIQUID 03.docx
Sep 22, 2017 · Grifols (MCE: GRF, MCE: GRF.P and NASDAQ:GRFS) announced the approval from the U.S. Food and Drug Administration (FDA) for a liquid formulation of its alpha-1 …
