Surrogate and clinical endpoints for studies in peripheral artery occlusive disease: Are statistics the brakes?
| Autor: | Victor Breul, Jörg Tautenhahn, Matthias Waliszewski, U. Redlich |
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| Rok vydání: | 2017 |
| Předmět: |
Male
medicine.medical_specialty Peripheral artery occlusive disease Brachial Artery Endpoint Determination medicine.medical_treatment Coronary Angiography Revascularization Peripheral Arterial Disease Percutaneous Coronary Intervention Clinical endpoint medicine Humans Ankle Brachial Index Intensive care medicine Reimbursement business.industry Surrogate endpoint Confounding General Medicine Critical limb ischemia Research Design Sample size determination Female Surgery medicine.symptom business |
| Zdroj: | Polish Journal of Surgery. 89:39-48 |
| ISSN: | 2299-2847 0032-373X |
| DOI: | 10.5604/01.3001.0009.9220 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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