Standardization of outcome measures in clinical trials of pharmacological treatment for abdominal aortic aneurysm

Abstract

An abdominal aortic aneurysm (AAA) is a common aortic wall disease with an increased prevalence in the elderly population (4–8% for those aged >65 years). Many AAAs are slow growing and remain insidious. Current standard of care for patients with small AAAs (<49 mm) is surveillance, with interventional therapy (open surgical repair or endovascular aneurysm repair) recommended for large (>50–55 mm), rapidly growing (>10 mm/year) or symptomatic AAAs. Although open surgical repair or endovascular aneurysm repair are effective, significant short- and long-term postoperative morbidity and mortality occurs. Currently, there is no pharmacological treatment specific for AAA; the need for the development of targeted pharmacological therapies based on clinically relevant and feasible outcomes acceptable to the medical community, regulatory agencies and third-party payers is high. A consensus on such end points will be critical to accelerating the development of pharmacological agents to prevent formation, arrest the expansion and reduce the rupture risk of AAA.

Keywords::

• abdominal aortic aneurysm
• clinical trial
• medical treatment
• risk of rupture
• standardized clinical end point

Acknowledgements

The authors acknowledge Guenther Mueller-Velten, Integrated Information Service, Novartis Pharmaceuticals Corporation, for his contribution to the statistical component of the hypothetical trial design of the manuscript.

Financial & competing interest disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.