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

Abstract

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.