Alpha-1 Antitrypsin Deficiency in COPD

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Presentation transcript:

Alpha-1 Antitrypsin Deficiency in COPD

This program will include a discussion of off-label treatment not approved by the FDA for use in the US.

The Discovery of AATD

What Causes AATD?

Diseases Associated With AATD

A1AT: The Protective Protein

Panlobular Emphysema Caused by AATD

A1AT Levels by Phenotype

A1AT Protective Threshold Level

Who Should Be Screened?

What Should Be Tested?

Conventional Treatment: COPD

Conventional Treatment: COPD (cont)

Conventional Treatment: COPD (cont)

Preventing Liver Disease

A1AT Augmentation Therapy

AATD Registry Study Group

AATD Registry Study: FEV1 Decline

Observational Studies: Clinical Implications

RAPID Trial

RAPID-OLE: Decline in Adjusted Lung Density

RAPID: Dosing

RAPID: Achieved A1AT Levels and Lung Density

RAPID: 6-Year Projected Life Extension With Augmentation Therapy

RAPID: Measuring Treatment Efficacy

RAPID: IDES/DES Biomarkers

RAPID: Potential of IDES/DES Biomarkers

Efficacy of Augmentation Therapy: Consistent Across Clinical Trials

Key Takeaways

Abbreviations

Abbreviations (cont)