Design of Clinical Trials in Acute Kidney Injury: Report from an NIDDK Workshop on Trial Methodology

被引：129

作者：

Palevsky, Paul M. ^{[1
]}

Molitoris, Bruce A. ^{[1
]}

Okusa, Mark D. ^{[1
]}

Levin, Adeera ^{[1
]}

Waikar, Sushrut S. ^{[1
]}

Wald, Ron ^{[1
]}

Chertow, Glenn M. ^{[1
]}

Murray, Patrick T. ^{[1
]}

Parikh, Chirag R. ^{[1
]}

Shaw, Andrew D. ^{[1
]}

Go, Alan S. ^{[1
]}

Faubel, Sarah G. ^{[1
]}

Kellum, John A. ^{[1
]}

Chinchilli, Vernon M. ^{[1
]}

Liu, Kathleen D. ^{[1
]}

Cheung, Alfred K. ^{[1
]}

Weisbord, Steven D. ^{[1
]}

Chawla, Lakhmir S. ^{[1
]}

Kaufman, James S. ^{[1
]}

Devarajan, Prasad ^{[1
]}

Toto, Robert M. ^{[1
]}

Hsu, Chi-yuan ^{[1
]}

Greene, Tom ^{[1
]}

Mehta, Ravindra L. ^{[1
]}

Stokes, John B. ^{[1
]}

Thompson, Aliza M. ^{[1
]}

Thompson, B. Taylor ^{[1
]}

Westenfelder, Christof S. ^{[1
]}

Tumlin, James A. ^{[1
]}

Warnock, David G. ^{[1
]}

Shah, Sudhir V. ^{[1
]}

Xie, Yining ^{[1
]}

Duggan, Emily G. ^{[1
]}

Kimmel, Paul L. ^{[1
]}

Star, Robert A. ^{[1
]}

机构：

[1] NIDDK, NIH, Bethesda, MD 20892 USA

来源：

CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY | 2012年 / 7卷 / 05期

关键词：

GLOMERULAR-FILTRATION-RATE; ACUTE-RENAL-FAILURE; CONSENSUS CONFERENCE; CARDIAC-SURGERY; SEVERE SEPSIS; OUTCOMES; MORTALITY; NETWORK; CARE; AKI;

D O I：

10.2215/CJN.12791211

中图分类号：

R5 [内科学]; R69 [泌尿科学（泌尿生殖系疾病）];

学科分类号：

1002 ; 100201 ;

摘要：

Acute kidney injury (AKI) remains a complex clinical problem associated with significant short-term morbidity and mortality and lacking effective pharmacologic interventions. Patients with AKI experience longer-term risks for progressive chronic ESRD, which diminish patients' health-related quality of life and create a larger burden on the healthcare system. Although experimental models have yielded numerous promising agents, translation into clinical practice has been unsuccessful, possibly because of issues in clinical trial design, such as delayed drug administration, masking of therapeutic benefit by adverse events, and inadequate sample size. To address issues of clinical trial design, the National Institute of Diabetes and Digestive and Kidney Diseases sponsored a workshop titled "Clinical Trials in Acute Kidney Injury: Current Opportunities and Barriers" in December 2010. Workshop participants included representatives from academia, industry, and government agencies whose areas of expertise spanned basic science, clinical nephrology, critical care medicine, biostatistics, pharmacology, and drug development. This document summarizes the discussions of collaborative workgroups that addressed issues related to patient selection, study endpoints, the role of novel biomarkers, sample size and power calculations, and adverse events and pilot/feasibility studies in prevention and treatment of AKI. Companion articles outline the discussions of workgroups for model trials related to prevention or treatment of established AKI in different clinical settings, such as in patients with sepsis. Clin J Am Soc Nephrol 7: 844-850, 2012. doi: 10.2215/CJN.12791211

引用

页码：844 / 850

页数：7

共 40 条

[1] Functional and survival outcomes in traumatic blunt thoracic aortic injuries: An analysis of the National Trauma Databank [J].

Arthurs, Zachary M. ;

Starnes, Benjamin W. ;

Sohn, Vance Y. ;

Singh, Niten ;

Martin, Matthew J. ;

Andersen, Charles A. .

JOURNAL OF VASCULAR SURGERY, 2009, 49 (04) :988-994

[2] Biomarkers and surrogate endpoints: Preferred definitions and conceptual framework [J].

Atkinson, AJ ;

Colburn, WA ;

DeGruttola, VG ;

DeMets, DL ;

Downing, GJ ;

Hoth, DF ;

Oates, JA ;

Peck, CC ;

Schooley, RT ;

Spilker, BA ;

Woodcock, J ;

Zeger, SL .

CLINICAL PHARMACOLOGY & THERAPEUTICS, 2001, 69 (03) :89-95

[3] Stopping Randomized Trials Early for Benefit and Estimation of Treatment Effects Systematic Review and Meta-regression Analysis [J].

Bassler, Dirk ;

Briel, Matthias ;

Montori, Victor M. ;

Lane, Melanie ;

Glasziou, Paul ;

Zhou, Qi ;

Heels-Ansdell, Diane ;

Walter, Stephen D. ;

Guyatt, Gordon H. .

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2010, 303 (12) :1180-1187

[4] Acute renal failure - definition, outcome measures, animal models, fluid therapy and information technology needs: the Second International Consensus Conference of the Acute Dialysis Quality Initiative (ADQI) Group [J].

Bellomo, R ;

Ronco, C ;

Kellum, JA ;

Mehta, RL ;

Palevsky, P .

CRITICAL CARE, 2004, 8 (04) :R204-R212

[5] REPORT OF THE AMERICAN-EUROPEAN CONSENSUS CONFERENCE ON ARDS - DEFINITIONS, MECHANISMS, RELEVANT OUTCOMES AND CLINICAL-TRIAL COORDINATION [J].

BERNARD, GR ;

ARTIGAS, A ;

BRIGHAM, KL ;

CARLET, J ;

FALKE, K ;

HUDSON, L ;

LAMY, M ;

LEGALL, JR ;

MORRIS, A ;

SPRAGG, R ;

DHAINAUT, JF ;

MATTHAY, M ;

MANCEBO, J ;

MEYRICK, B ;

PAYEN, D ;

PERRET, C ;

FOWLER, AA ;

SCHALLER, MD ;

VANASBECK, BS ;

COCHIN, B ;

LANKEN, PN ;

LEEPER, KV ;

MARINI, J ;

MURRAY, JF ;

OPPENHEIMER, L ;

PESENTI, A ;

REID, L ;

RINALDO, J ;

VILLAR, J ;

Hyers, T ;

Knaus, W ;

Matthay, R ;

Pinsky, M ;

Bone, RC ;

Bosken, C ;

Johanson, WG ;

Lewandowski, K ;

Repine, J ;

Rodriguez-Roisin, R ;

Roussos, C .

INTENSIVE CARE MEDICINE, 1994, 20 (03) :225-232

[6] In-Center Hemodialysis Six Times per Week versus Three Times per Week [J].

Chertow, Glenn M. ;

Levin, Nathan W. ;

Beck, Gerald J. ;

Depner, Thomas A. ;

Eggers, Paul W. ;

Gassman, Jennifer J. ;

Gorodetskaya, Irina ;

Greene, Tom ;

James, Sam ;

Larive, Brett ;

Lindsay, Robert M. ;

Mehta, Ravindra L. ;

Miller, Brent ;

Ornt, Daniel B. ;

Rajagopalan, Sanjay ;

Rastogi, Anjay ;

Rocco, Michael V. ;

Schiller, Brigitte ;

Sergeyeva, Olga ;

Schulman, Gerald ;

Ting, George O. ;

Unruh, Mark L. ;

Star, Robert A. ;

Kliger, Alan S. .

NEW ENGLAND JOURNAL OF MEDICINE, 2010, 363 (24) :2287-2300

[7] The duration of postoperative acute kidney injury is an additional parameter predicting long-term survival in diabetic veterans [J].

Coca, Steven G. ;

King, Joseph T., Jr. ;

Rosenthal, Ronnie A. ;

Perkal, Melissa F. ;

Parikh, Chirag R. .

KIDNEY INTERNATIONAL, 2010, 78 (09) :926-933

[8] Costs and outcomes of acute kidney injury (AKI) following cardiac surgery [J].

Dasta, Joseph F. ;

Kane-Gill, Sandra L. ;

Durtschi, Amy J. ;

Pathak, Dev S. ;

Kellum, John A. .

NEPHROLOGY DIALYSIS TRANSPLANTATION, 2008, 23 (06) :1970-1974

[9]

Dehmer Gregory J, 2004, J Cardiovasc Manag, V15, P20

[10] Exploring the benefits of adaptive sequential designs in time-to-event endpoint settings [J].

Emerson, Sarah C. ;

Rudser, Kyle D. ;

Emerson, Scott S. .

STATISTICS IN MEDICINE, 2011, 30 (11) :1199-1217

← 1 2 3 4 →