Therapy for Alpha-1 Antitrypsin Deficiency: The Evidence for Efficacy

This symposium took place on 28th September 2015 as part of the European Respiratory Society International Congress 2015 in Amsterdam, Netherlands

Chairperson: Noel Gerard McElvaney1
Speakers: Emer Reeves,2 David Parr,3 Niels Seersholm,4 Kenneth R. Chapman5

1. Department of Medicine, Royal College of Surgeons in Ireland, Dublin, Ireland
2. Beaumont Hospital, Royal College of Surgeons in Ireland, Dublin, Ireland
3. University Hospitals Coventry and Warwickshire, Coventry, UK
4. Gentofte Hospital, Hellerup, Denmark
5. University of Toronto, Toronto, Ontario, Canada

Disclosure: David Parr has received honoraria for advising CSL Behring and Grifols (formerly Talecris), in addition to sponsorship from AstraZeneca and Boehringer Ingelheim. Emer Reeves and Niels Seersholm have no competing interests to disclose; however, both have received honoraria for this educational activity funded by CSL Behring. Kenneth R. Chapman has received compensation for consulting with AstraZeneca, Baxter, Boehringer Ingelheim, CSL Behring, GlaxoSmithKline, Grifols, Kamada, Novartis, Nycomed, Roche, and Telacris; has completed research funded by Amgen, AstraZeneca, Baxter, Boehringer Ingelheim, CSL Behring, Forest Labs, GlaxoSmithKline, Grifols, Novartis, Roche, and Takeda; and has participated in continuing medical education activities sponsored in whole or in part by AstraZeneca, Boehringer Ingelheim, GlaxoSmithKline, Grifols, Merck Frosst, Novartis, Pfizer, and Takeda. He is participating in research funded by the Canadian Institutes of Health Research operating grant entitled Canadian Cohort Obstructive Lung Disease. He holds the GlaxoSmithKline–Canadian Institutes of Health Research Chair in Respiratory Health Care Delivery at the University Health Network, Toronto, Ontario, Canada.
Acknowledgements: Writing assistance was provided by Ms Rachel Stevens of ApotheCom.
Support: The publication of this article was funded by CSL Behring.
Citation: EMJ Respir. 2015;3[2]:55-62.

Meeting Summary

Prof McElvaney opened the symposium with a brief overview of the disease history and available treatments to date for alpha-1 antitrypsin deficiency (AATD). He then introduced Dr Reeves, who gave a description of the physiological function of alpha-1 proteinase inhibitor (α1-PI), specifically its effect on neutrophil function in AATD. Dr Parr then discussed the limitations of using forced expiratory volume (FEV1) to observe lung disease progression, and the development and use of measurements of lung density as an alternative. Dr Seersholm followed with a comprehensive overview of recent clinical studies demonstrating the efficacy of α1-PI augmentation therapy. Dr Chapman gave the final presentation that expanded on this by describing the findings of the randomised, placebo-controlled trial of augmentation therapy in α1-PI deficiency (RAPID) study. The meeting objectives were to present the current treatment landscape for AATD-associated emphysema and the role of α1-PI therapy within this.

Click here to view the online CME activity on ‘Clinical Evidence for Augmentation Therapy in Patients with Emphysema due to Alpha-1 Antitrypsin Deficiency (AATD)’, available on Medscape.

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