Modifying Alpha-1 Antitrypsin Deficiency-Related Emphysema: From Evidence to Practice - European Medical Journal

Modifying Alpha-1 Antitrypsin Deficiency-Related Emphysema: From Evidence to Practice

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Noel Gerard McElvaney,1 Ilaria Ferrarotti2
Joanna Chorostowska-Wynimko,3 Kenneth R. Chapman,4 A. Rembert Koczulla5

Prof McElvaney has received funding support from CSL Behring, Grifols, Bayer, and Vertex. Dr Ferrarotti has received travel grants and honoraria from CSL Behring and Grifols. Prof ChorostowskaWynimko has received grants, personal fees, and non-financial support from Grifols, grants from Baxalta US Inc, and grants and personal fees from CSL Behring. Prof Chapman has received consulting fees from AstraZeneca, Boehringer Ingelheim, CSL Behring, GSK, Grifols, Kamada, Merck, Novartis, Pfizer, Roche, and Takeda. Prof Chapman has also received speaker fees from AstraZeneca, Boehringer Ingelheim, Chiesi, Grifols, GSK, Merck, Novartis, and Takeda and has received research grants/contracts from Amgen, AstraZeneca, CSL Behring, Genentech, GSK, Grifols, Kamada, Novartis, Roche, and Sanofi. Prof Koczulla has received honoraria from AstraZeneca, Boehringer Ingelheim, Chiesi, CSL Behring, Grifols, GSK, Menarini, and Novartis.


Writing assistance was provided by Mia Cahill of ApotheCom.


The symposium and the publication of this article was funded by CSL Behring. The views and opinions expressed are those of the authors and not necessarily CSL Behring or the International Congress of the European Respiratory Society.

EMJ Respir. ;4[1]:35-42. DOI/10.33590/emjrespir/10310897.
ERS 2016, European Respiratory Society

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

Meeting Summary

The symposium discussed the role of disease modification in alpha-1 antitrypsin deficiency (AATD)-related emphysema. Evidence from the recent RAPID trial and its extension trial showed that treating AATD patients with intravenous alpha-1 antitrypsin (alpha-1 proteinase inhibitor; [A1-PI]) therapy slowed the rate of lung density decline and had a disease-modifying effect. By modifying the course of disease, survival can be extended by several years. Dr Ferrarotti opened the symposium by introducing the topic of AATD-related emphysema, highlighting the latest epidemiological data, and providing an overview of the treatment landscape. Prof Chorostowska-Wynimko then addressed how to determine the disease modification that occurs in AATD, focussing on the clinical trial design (classical parallel-group, placebo-controlled trial design versus a ‘late-start’ study design) and clinical outcomes (forced expiratory volume in 1 second [FEV1] versus computed tomography [CT] lung density). Prof Chapman explained the results and the post hoc analyses of the RAPID trials; a sustained reduction in lung density decline rate that proves to have a disease-modifying effect. Prof Koczulla closed the symposium by relating current evidence to the real-life management of patients, notably how patients should be monitored and the prospect of home-based care.

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