Abstract
With new patient-centric and scientific networks being created, Prof Chorostowska-Wynimko explored how these initiatives, such as the European Alpha-1 Research Collaboration (EARCO) and the European Reference Network-LUNG Alpha1 Antitrypsin Deficiency (ERN-LUNG AATD Core Network), will help to advance the management of alpha 1-antitrypsin deficiency (AATD) patients. EARCO plans to create a registry to gather information from centres across Europe and ERN-LUNG AATD plans to ensure highly specialised healthcare for AATD patients, including reliable AATD diagnostics across European laboratories.
Explaining in more detail the plans for the new EARCO registry, Dr Barrecheguren argued the case for another AATD registry to gather large-scale data that clinical trials cannot provide. She provided an overview of the new EARCO prospective follow-up registry, to be launched next year, which will integrate existing national AATD registries, enhance long-term follow-up and quality of data, and facilitate research and quality improvements across healthcare systems.
Discussing one of the first initiatives of the ERN-LUNG AATD Core Network, Dr Ferrarotti explored how to align AATD testing across Europe with the creation of European LAB-NET, an initiative first involving six European centres that will co-operate to collect, develop, verify, and make reference materials available for molecular and biochemical tests to correctly diagnose AATD and provide quality control in the laboratory diagnosis.
Dr Greulich reported on a post-hoc pooled analysis from the RAPID-randomised controlled trial (RAPID-RCT) and the RAPID-open label extension (RAPID-OLE) study, which compared the safety and tolerability of adverse event (AE) rates for two different alpha-1 antitrypsin (AAT) dosing patterns, weekly infusions of 60 mg/kg AAT, and bi-weekly infusions of 120 mg/kg AAT. Results showed there were no significant differences for exposure-adjusted event rates (p=0.850), infusion-adjusted event rates (p=0.344), and serious treatment emergent AE (TEAE) (p=1.0); TEAE occurring in the first 24 and 48 hours were comparable for both groups.
Prof Sandhaus presented the results of a telephone survey from the USA AlphaNet organisation of self-infusion practices in 555 patients with AATD. The survey found that 7.9% of respondents self-administered AAT and 92.1% who did not. Of the 44 patients who self-administered AAT, 95.4% reported being very satisfied and 4.6% were satisfied with their treatment.