Evidence-based medicine and clinical trials in renal transplantation
- Rita R Alloway, PharmD, BCPS
Rita R Alloway, PharmD, BCPS
- Research Professor of Medicine
- Director, Transplant Clinical Research
- Director, Transplant Specialty Residency and Fellowship
- University of Cincinnati
- Nicole A Pilch, PharmD, MSCR, BCPS
Nicole A Pilch, PharmD, MSCR, BCPS
- Clinical Specialist
- Medical University of South Carolina
- Section Editor
- Daniel C Brennan, MD, FACP
Daniel C Brennan, MD, FACP
- Editor-in-Chief — Nephrology
- Section Editor — Renal Transplantation
- Professor of Medicine
- Medical Director and Co-Director of the Comprehensive Transplant Center, Department of Internal Medicine, Division of Nephrology
- Johns Hopkins Medical School
Since the introduction of cyclosporine [1,2], there has been a dramatic increase in the number of successful renal transplants and use of non-cyclosporine immunosuppressive agents [3-20]. As the donor and recipient selection criteria for transplantation expand, in combination with the continued introduction of new immunosuppressive agents, the ability to identify the optimal immunosuppressive regimen for a given individual has grown increasingly complex [3-21]. In addition, with improved graft and patient survival secondary to improved immunosuppression, trial outcome measures have also evolved, making individual trial results difficult to compare. To combat this difficulty, a critical, systematic, and scientific method for evaluating ongoing results from clinical trials is needed.
Evidence-based medicine is the explicit and judicious use of current and best evidence to guide clinical decisions [21,22]. Evidence-based medicine must be considered in the context of clinical expertise and with the understanding that its use is based on published literature, which is subject to reporting bias [21,23,24]. Several guidelines for the publication and design of clinical trials in solid organ transplant recipients have been developed [25-27].
This topic provides an overview of the principles of evidence-based medicine in the evaluation of immunosuppressive regimens in renal transplantation. More general issues related to evidence-based medicine and decision analysis are discussed separately. (See "Evidence-based medicine" and "Decision analysis".)
Randomized, double-blind, controlled clinical trials are the most appropriate design to prove the effectiveness of a new treatment. However, this design may not always be feasible or ethical in renal transplantation [23,28,29].
Previously, the gold standard used to assess the efficacy of a new immunosuppressant agent alone or in combination was graft survival and the incidence of acute rejection, with the latter being a predictor for long-term graft survival. However, improvements in short- and long-term allograft survival and the significant decrease in acute rejection rates have led researchers to alternative study designs and endpoints .To continue reading this article, you must log in with your personal, hospital, or group practice subscription. For more information on subscription options, click below on the option that best describes you:
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