Surrogate and clinical endpoints for studies in peripheral artery occlusive disease: Are statistics the brakes?
Journal Title: Polish Journal of Surgery - Year 2017, Vol 89, Issue 2
Abstract
Background: The aim of this review is to present the available clinical and surrogate endpoints that may be used in future studies performed in patients with peripheral artery occlusive disease (PAOD). Importantly, we describe statistical limitations of the most commonly used endpoints and offer some guidance with respect to study design for a given sample size. The proposed endpoints may be used in studies using surgical or interventional revascularization and/or drug treatments. Methods: Considering recently published study endpoints and designs, the usefulness of these endpoints for reimbursement is evaluated. Based on these potential study endpoints and patient sample size estimates with different non-inferiority or tests for difference hypotheses, a rating relative to their corresponding reimbursement values is attempted. Results: As regards the benefit for the patients and for the payers, walking distance and the ankle brachial index (ABI) are the most feasible endpoints in a relatively small study samples given that other non-vascular impact factors can be controlled. Angiographic endpoints such as minimal lumen diameter (MLD) do not seem useful from a reimbursement standpoint despite their intuitiveness. Other surrogate endpoints, such as transcutaneous oxygen tension measurements, have yet to be established as useful endpoints in reasonably sized studies with patients with critical limb ischemia (CLI). Conclusions: From a reimbursement standpoint, WD and ABI are effective endpoints for a moderate study sample size given that non-vascular confounding factors can be controlled.<br/><br/>
Authors and Affiliations
Matthias W. Waliszewski, Ulf Redlich, Victor Breul, Jörg Tautenhahn
Keywords
Related Articles
- EP ID EP188953
- DOI 10.5604/01.3001.0009.9220
- Views 106
- Downloads 0
Own experience from the use of a substitute of an allogeneic acellular dermal matrix revitalized with in vitro cultured skin cells in clinical practice
As a result of the removal of cells from human allogeneic dermis, a collagen scaffold is obtained, which can be populated de novo with autologous/allogeneic skin cells and transplanted onto the area of skin loss. The opt...
Diagnostyka i leczenie choroby refluksowej przełyku powikłanej powstaniem przełyku Barretta
Cel pracy: Ocena efektywności klinicznego zastosowania algorytmu diagnostycznego oraz terapeutycznego w chorobie refluksowej przełyku (gastroesophageal reflux disease; GERD) powikłanej powstaniem przełyku Barretta u 46 p...
Cide-A Gene Expression in Patients with Obesity Qualified for Endovascular Treatment of Abdominal Aorta Aneurysm
CIDE-A gene and the genes of LRP group play a key role in the regulation of the body weight and lipid metabolism in mammals. CIDE-A is defined as a potential human obesity gene and the LRP1 gene is associated with the de...
Effect of complicated thyroid surgical procedures on personal and professional life of patients
Aspergillosis – Diagnostic And Therapeutic Difficulties In Critically Ill Surgical Patients
Improving the effectiveness of the treatment of critically ill and their longer survival has increased the existence of later complications rarely seen before. Among the hospital-acquired infections such complications in...