To report an adverse event or product quality complaint, call 1-877-TAKEDA-7 (1-877-825-3327)
To report an adverse event or product quality complaint, call 1-877-TAKEDA-7 (1-877-825-3327)
Not an actual patient.
AATD is a genetic condition associated with low protein levels of functional alpha-1 proteinase inhibitor (A1PI).2,3
The main clinical manifestations involve the lungs, leading to emphysema, and the liver, but it can also be associated with other skin and cardiovascular disorders.3,4,5
AATD is caused by a mutation in the SERPINA1 gene on chromosome 14 encoding for A1PI.6 Each person has two alleles (variants) of the gene, one inherited from each parent where the most common ones are:5
A1PI is primarily synthesized in the liver and secreted into the bloodstream where it diffuses into the lungs and other tissues.2,8,10 The main role of A1PI is to inhibit neutrophil elastase (NE) and keep its activity in check.2,10 NE is a proteinase responsible for the breakdown of a variety of proteins involved in modulating inflammation and therefore plays a key role in tissue repair and protection from infections.11 Therefore, a deficiency in functional A1PI results in excessive NE activity and leads to the degradation of lung tissue and blood vessels.12
The prevalence of AATD varies by genotype with an estimated 3.4 million people living with the disease worldwide (protease inhibitor [Pi] SS, PiSZ, PiZZ) and 116 million carriers (PiMS, PiMZ).2,13 In the U.S., it’s estimated that more than 500,000 people have deficient PiSS and PiSZ allele combinations, and 60,000 have the PiZZ genotype which is associated with severe deficiency, while more than 24 million people are carriers (PiMS, PiMZ).8
Variability in clinical presentations results in delayed and misdiagnosis, which means the majority of cases remain undiagnosed (>90%).1,14,15,16 A confirmed AATD diagnosis should involve both quantitative and qualitative laboratory determinations:10,17,18,19
The primary manifestations of AATD are lung disease and liver involvement, which are irreversible and progressive.20 Therefore, the consequences of delayed or misdiagnosis can be fatal in those patients with severe AATD; most commonly due to respiratory failure (58% of deaths), including pneumonia and pneumothorax; and liver diseases (12% of deaths), including liver failure and primary carcinoma.21 This leads to a high disease burden, associated with increased mortality, morbidity, quality of life impairment and cost.21,22,23,24,25,26 Nonpharmacological approaches are crucial to managing AATD and include smoking cessation, maintaining a healthy diet and moderate exercise, staying up to date with recommended immunization and pulmonary rehabilitation.17 Currently, augmentation therapy is the only A1PI pharmacological option available and can be part of the strategy in the management of patients with severe AATD-related emphysema.10,27,28 It is administered by intravenous infusion and aims to increase serum A1PI levels.2 However, the manufacturing process is complex and expensive to produce as it needs to be purified from large volumes of human plasma.29
This resource provides information on Takeda medications available in the Alpha-1 Antitrypsin Deficiency category and is not intended to represent a complete list of therapeutic options.
[Alpha1-Proteinase Inhibitor (Human)]
[Alpha1-Proteinase Inhibitor (Human)]
Watch videos focused on Alpha-1 Antitrypsin Deficiency (AATD).
Learn about Alpha-1 Antitrypsin Deficiency (AATD) mechanism of disease in this animated video.
This is not intended to be a comprehensive resource of all congresses and congress materials across therapeutic and disease areas. Congress materials may include information about investigational use(s) of compounds/products that are not approved for use by the U.S. Food and Drug Administration (FDA) and/or are inconsistent with the Prescribing Information. Takeda does not recommend the use of any Takeda product beyond the approved labeling. Any decisions regarding the usage of a Takeda product beyond the approved labeling are left to the discretion of the healthcare professional. Takeda makes no representations about whether investigational compounds or unapproved uses will be approved by the FDA.
National conference of IgNS bringing together professionals and practitioners from all disciplines and clinical specialties to advance Ig therapy practice, while providing networking and comprehensive education opportunities.
Annual meeting dedicated to the advancement of neuromuscular, musculoskeletal, and electrodiagnostic medicine.
Global meeting featuring thought leaders from around the world focused on advancing patient care in allergy and immunology.
Premier global educational event for allergists and immunologists, with thousands of attendees each year, discussing allergies, asthma, and immune deficiency disorders.
Each year, the American Academy of Neurology's Annual Meeting offers a robust lineup of diverse learning opportunities covering nearly every topic and subspecialty, helping you stay up to date on the latest trusted science and essential education.
Brings together home and alternate site infusion professionals for four days of networking, education, and exhibits. The expo features companies displaying the latest products and services supporting the industry.
This event brings together over 1200 HAE community members, including families, caregivers, healthcare professionals, and pharmaceutical representatives.
Regional annual conference discussing the most current information from top experts in allergy, asthma, and immunology.
International conference showcasing the latest advances in pulmonary disease, critical illness, and sleep disorders for scientists and clinicians at all stages of their careers.
Annual meeting with the goal of providing a stimulating forum with presentations and discussions on the latest advances in clinical immunology, including primary immunodeficiencies and immune dysregulatory diseases.
Annual meeting in neurology promoting high quality patient-centric care in a variety of topics and specialties through various learning formats.
Large assembly of pharmacy and healthcare professionals dedicated to the issues of managed care pharmacy, offering immersive education sessions and keynote presentations.
Annual conference ideal for infusion professionals or those seeking opportunities in the industry to come together and explore the latest trends in offering home-based services.
Premier global educational event for allergists and immunologists, with thousands of attendees each year, discussing allergies, asthma, and immune deficiency disorders.
Annual scientific session uniting allergists, immunologists, nurses, and physician assistants aimed at maintaining the highest standard of practice in allergy care.
This international congress offers attendees an updated view on neuromuscular disorders and networking opportunities to increase their international experience and collaborations.
Global meeting featuring thought leaders from around the world focused on advancing patient care in allergy and immunology.
National conference of IgNS bringing together professionals and practitioners from all disciplines and clinical specialties to advance Ig therapy practice, while providing networking and comprehensive education opportunities.
Biennial meeting that covers the latest discoveries in basic and clinical science covering our expanding field, bringing together internationally renowned scientists and clinicians to present and discuss major developments in diagnosis, clinical management, genetics, and immunobiology of inborn errors of immunity.
Click the button below to send us your Medical Inquiry. We are committed to responding to your inquiries, and will get back to you as soon as possible.
Contact us using the number below to: