Therapy for Alpha-1 Antitrypsin Deficiency: The Evidence for Efficacy - European Medical Journal


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

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

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.


Writing assistance was provided by Ms Rachel Stevens of ApotheCom.


The publication of this article was funded by CSL Behring.

EMJ Respir. ;3[2]:55-62.

Each article is made available under the terms of the Creative Commons Attribution-Non Commercial 4.0 License.

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.

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