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CME: Alpha-1 antitrypsin deficiency (AATD): Optimizing pharmacotherapeutic management strategies


AN ACCREDITED UP-TO-DATE VERSION OF THIS TOPIC CAN BE FOUND AT: Addressing unmet needs and updates in the pharmacotherapeutic management of alpha-1 antitrypsin deficiency (AATD) and its complications

Activity Description / Statement of Need:

In this online CME self-learning activity:

Alpha 1-antitrypsin deficiency (AATD), characterized by low serum levels of the serine protease inhibitor alpha-1 antitrypsin (AAT), is a genetic disorder resulting in destruction of lung structures. Reduced levels of AAT result in overactivity of neutrophil elastase, which destroys connective tissue within the lung and causes degradation of alveoli, reduced pulmonary elastic recoil, and airflow. Breakdown of the alveoli eventually manifest as emphysema or other forms of chronic lung disease, including chronic obstructive lung disease (COPD). Other complications associated with AATD include liver disease, panniculitis, and vasculitis. The most common cause of death in patients with severe AATD is respiratory failure, which accounts for 45 to 72% of deaths. Smoking, occupational hazards such as firefighting, and high levels of cumulative exposure to pollution accelerate the rate of lung function decline in people with AATD. AATD is estimated to affect one out of every 2,000 to 5,000 individuals, mainly of North European or Iberian ancestry, with a global prevalence of over 3.4 million affected individuals.

Target Audience:

The following healthcare professionals: pulmonologists and primary care physicians; physician assistants, nurse practitioners, nurses, and pharmacists who practice in pulmonology and internal medicine; and any other healthcare professionals with an interest in or who clinically encounter patients with AATD.

Commercial Support Disclosure: This activity is supported by an educational grant from Takeda and CSL Behring.

Learners may participate in this activity free of charge.

Release Date: July 26, 2020 -- Expiration Date: July 26, 2022

Faculty: Igor Barjaktarevic, MD


Faculty introduction, disclosures

Introduction content: review of AATD pathophysiology, diagnosis

  • History of AATD: Identification in 1963
  • Pathophysiology: Hepatic manufacture of AAT protein whose principle function is in lung; lack of AAT ->  excess protease -> lung degradation
  • Risk factors: Smoking, genetics
  • Age of clinical presentation, who is at risk, and who should be tested, including family members
  • Differential diagnosis: AATD, COPD, Idiopathic giant bullous emphysema (vanishing lung syndrome), congenital lobar emphysema, “Ritalin” (methylphenidate) Lung, Swyer James Syndrome (post infectious obliterative bronchiolitis)
  • Natural history of disease progression
  • General understanding of AATD among health care professionals
  • Importance of early diagnosis
  • Patient case

Treatment of AATD

  • Present maintenance pharmacotherapy: AAT augmentation therapy
  • AAT augmentation: Considerations for use, dosage, risks in special populations
  • AAT augmentation: Outcomes over time
  • AAT augmentation: Results when combined with disease state management
  • Defining the role of AAT augmentation therapies and their appropriate use in selected patient populations
  • The role of lung transplant and International Society Guidelines on recipient selection
  • Clinical controversy: AAT augmentation post-transplant
  • Patient case

Barriers to care

  • Genetic testing: Informed consent, and financial and psychosocial well-being
  • Facilitating access to patient assistance programs

Summary, conclusions, and best practice recap

Learning Objectives

By the end of the session the participant will be able to:

  • Summarize the present state of awareness of AATD among healthcare professionals and its attendant impact on patient care.
  • Describe the best time to test a patient for AATD and whether to conduct a family screening.
  • Apply best treatment practices to patients with AATD in various clinical scenarios.
  • Describe the challenges associated with the optimal diagnosis and treatment of AATD.


ACCME Activity #201806113


Faculty Disclosure and Resolution of COI

As a provider of continuing medical education, it is the policy of ScientiaCME to ensure balance, independence, objectivity, and scientific rigor in all of its educational activities. In accordance with this policy, faculty and educational planners must disclose any significant relationships with commercial interests whose products or devices may be mentioned in faculty presentations, and any relationships with the commercial supporter of the activity. The intent of this disclosure is to provide the intended audience with information on which they can make their own judgments. Additionally, in the event a conflict of interest (COI) does exist, it is the policy of ScientiaCME to ensure that the COI is resolved in order to ensure the integrity of the CME activity. For this CME activity, any COI has been resolved thru content review ScientiaCME.

Faculty Disclosure: Igor Barjaktarevic, MD, PhD, Assistant Professor, Medical Director of COPD program, Pulmonary and Critical Care Medicine, David Geffen School of Medicine at UCLA,  has received financial support (consulting and/or research) from PCORI, COPD Foundation, AMGEN, GE Healthcare, Theravance, Mylan, Astra Zeneca, Boehringer, GSK, Verona Pharma, Theravance, Grifols, CSL Behring, Shire, Adverum, Vertex, Kamada, Arrowhead, and Mereo

Disclosure of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, President of ScientiaCME, has no relevant financial disclosures.

Commercial Support Disclosure: This activity is supported by an educational grant from Takeda and CSL Behring.


  • Read the learning objectives above
  • Take the Pre-Test (optional). Completion of the pre-test will help us evaluate the knowledge gained by participating in this CME activity.
  • View the online activity. You may view this is in more than one session, and may pause or repeat any portion of the presentation if you need to.
  • Minimum participation threshold: Take the post-test. A score of 70% or higher is required to pass and proceed to the activity evaluation.
  • Complete the activity evaluation and CME registration. A CE certificate will be emailed to you immediately.

Cultural/Linguistic Competence & Health Disparities

System Requirements

Windows 7 or above
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*Adobe Acrobat Reader
Mac OS 10.2.8
Safari or Chrome or Firefox
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Internet Explorer is not supported on the Macintosh

*Required to view Printable PDF Version

Perform Pre-Test (optional)

Please take a few minutes to participate in the optional pre-test. It will help us measure the knowledge gained by participating in this activity.

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