Transitioning Clinical Data into Patient Care: Recent Real-Life Experiences with Alpha 1

This satellite symposium took place on 11th September 2017, as part of the European Respiratory Society (ERS) International Congress in Milan, Italy

Chairpersons: Jan Stolk,1 Berend Stoel1
Speakers: David Parr,2 Joachim H. Ficker,3 Charlie Strange,4 Jan Stolk1

1. Leiden University Medical Center, Leiden, Netherlands
2. University Hospitals Coventry and Warwickshire NHS Trust, Coventry, UK
3. Nuremberg General Hospital/Paracelus Medical University, Nuremberg, Germany
4. Medical University of South Carolina, Charleston, South Carolina, USA

Disclosure: Dr Stolk has acted as a consultant to CSL Behring and Kamada. Dr Stoel has acted as a consultant to BioClinica, CSL Behring, Baxalta, and Kamada. Prof Parr has acted as a consultant and is on the advisory boards for CSL Behring and Kamada, has given lecture presentations for CSL Behring and Grifols, and has attended conferences for CSL Behring. Prof Ficker is a member of the RAPID clinical trial programme study group funded by CSL Behring, has had research grants from CSL Behring, and has received personal fees and non-financial support from CSL Behring and Grifols. Prof Strange has received present and past grants from the Alpha 1 Foundation, CSL Behring, Grifols, the NIH, PneumRx, Pulmonx, and Shire; and has acted as a consultant for AstraZeneca, Baxalta, CSL Behring, Grifols, Abeona, and Pulmonx.
Acknowledgements: Writing assistance was provided by Janet Fricker.
Support: The symposium and the publication of this article was funded by CSL Behring GmbH. The views and opinions expressed are those of the authors and not necessarily CSL Behring or the European Respiratory Society.
Citation: EMJ Respir. 2017;5[1]:32-40.

Meeting Summary

The symposium explored the use of state-of-the-art technologies in building the evidence for Alpha 1 Antitrypsin (AAT) efficacy, namely the use of computed tomography (CT) and recent developments in regional lung density analysis, and current challenges and data gaps for the management of AAT deficiency (AATD). The vital importance of registries in building our knowledge and understanding of AATD and its management were also discussed. Dr Stolk opened the symposium with a brief overview of AATD and the results of the RAPID clinical trial programme, which provided evidence for the efficacy of AAT therapy in slowing the rate of lung density loss in AATD. Prof Parr then presented the rationale and methodology for assessing regional lung density changes, as measured by CT, and the potential clinical relevance of regional treatment variability in AATD. Prof Ficker addressed the clinical implications for AATD treatment, in light of data from the RAPID clinical trial programme, and provided an overview of the current challenges for treating patients with AATD, including questions surrounding when to commence AAT therapy and how the potential life-extending effect of AAT therapy can be assessed and quantified. Finally, the importance of registries was discussed; Prof Strange provided an overview of the USA Alpha 1 Foundation registry and presented key published data. In addition, he discussed current and future initiatives. Dr Stolk considered the European Alpha 1 International Registry (AIR) and presented the results of recent projects supported by this registry.

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