Citations

Background: VICTORION-INITIATE (NCT04929249) showed significantly greater LDL-C lowering in patients (pts) with ASCVD with an inclisiran first (IF) strategy (adding inclisiran immediately on failure to achieve LDL-C <70 mg/dL on maximally tolerated statin therapy) vs usual care (UC). Tokenization can be leveraged to link clinical trial data to pt-level real-world data (RWD) to provide a comprehensive overview of the pt journey pre- and post-trial and enhance understanding of clinical trial and long-term pt outcomes. Aim: To utilize tokenization and linked RWD to characterize the pt journey pre-VICTORION-INITIATE enrollment. Methods: Eligible pts from VICTORION-INITIATE were deidentified and tokenized with unique IDs, allowing linking to RWD (EMR, claims, hospital, and lab records) to follow the pt journey in the 3-year pre-trial and 2-year post-trial periods. Here we present demographics, clinical characteristics, and lipid-lowering therapy (LLT) use of pts with linked pre-trial RWD. Results: Of 450 pts randomized, 383 (85.1%) were linked with pre-trial RWD (IF arm, n=193; UC arm, n=190); 26.9% were female, first available recorded mean (SD) age was 64 (9.36) years. Among the 74.2% (n=284) of pts with linked, pre-trial, lipid test RWD, mean (SD) earliest and latest LDL-C values were 100.1 mg/dL (35.70) and 99.3 mg/dL (30.06), respectively, for pts in the IF arm vs 97.3 mg/dL (40.43) and 97.8 mg/dL (27.60) for pts in the UC arm ( Table ). Overall, in the pre-trial period, LLT was dispensed/prescribed to 66.1% of pts, statins to 64.5%, ezetimibe to 4.4%, and PCSK9i to 4.4%. Of pts with ≥1 recorded ASCVD events in the pre-trial period (n=141), 53.2% and 68.8% had available LLT dispensing/prescription records pre- and post-event, respectively. Conclusions: In the 3 years pre-VICTORION-INITIATE, mean LDL-C levels were above recommended goals, non-statin LLT prescription was infrequent, and ~31% of pts with ≥1 recorded ASCVD event had no evidence of post-event LLT use, highlighting a need for more aggressive LDL-C management. Full data visibility for each pt is a limitation of this approach. Tokenization post-trial will allow for long-term evaluation of LLT practices.

Repair and regeneration of a diseased lung using stem cells or bioengineered tissues is an exciting therapeutic approach for a variety of lung diseases and critical illnesses. Over the past decade, increasing evidence from preclinical models suggests that mesenchymal stromal cells, which are not normally resident in the lung, can be used to modulate immune responses after injury, but there have been challenges in translating these promising findings to the clinic. In parallel, there has been a surge in bioengineering studies investigating the use of artificial and acellular lung matrices as scaffolds for three-dimensional lung or airway regeneration, with some recent attempts of transplantation in large animal models. The combination of these studies with those involving stem cells, induced pluripotent stem cell derivatives, and/or cell therapies is a promising and rapidly developing research area. These studies have been further paralleled by significant increases in our understanding of the molecular and cellular events by which endogenous lung stem and/or progenitor cells arise during lung development and participate in normal and pathological remodeling after lung injury. For the 2023 Stem Cells, Cell Therapies, and Bioengineering in Lung Biology and Diseases Conference, scientific symposia were chosen to reflect the most cutting-edge advances in these fields. Sessions focused on the integration of “omics” technologies with function, the influence of immune cells on regeneration, and the role of the extracellular matrix in regeneration. The necessity for basic science studies to enhance fundamental understanding of lung regeneration and to design innovative translational studies was reinforced throughout the conference.