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Patient Level Outcomes - the Missing Link


Treatment of chronic kidney disease (CKD) may be life-saving, but can disrupt every aspect of a patient's life and the lives of family members. Many patients with CKD are elderly with significant comorbidities and sometimes therapies to improve survival may be less important than those that improve or maintain quality of life. In this setting, patient-level benefits become particularly important goals of therapy. Randomized controlled trials (RCT) are also essential to justify expensive therapies, such as medications used in the treatment of CKD mineral and bone disorders. Surprisingly, data to support the efficacy of these drugs for patient-level outcomes remains limited. In fact, fewer RCT are conducted in renal medicine than in any other medical specialty and reliance is often placed on association data and the assessment of intermediate and biochemical end-points. While some of these may prove to be valid surrogates for clinically important outcomes, some may not. Inclusion of patient-level outcomes in clinical research provides a missing link that can inform a more comprehensive approach to clinical practice and patient care. Incorporating measures of health-related quality of life into clinical trials can make outcomes more relevant and may be relatively simple. This paper provides examples of reliable, validated instruments to measure health-related quality of life domains and functional status, together with practical instructions for their use. Most could be incorporated into RCT of CKD mineral and bone disorder treatments. Inclusion of outcomes that are perceived by patients to be significant should become standard practice in renal medicine and in clinical renal research.

Type Journal
ISBN 1440-1797 (Electronic)
Authors O'Shaughnessy, D.; Elder, G.
Responsible Garvan Author Prof Grahame Elder
Publisher Name NEPHROLOGY
Published Date 2009-06-01
Published Volume 14
Published Issue 4
Published Pages 443-51
Status Published in-print
DOI NEP1136 [pii] 10.1111/j.1440-1797.2009.01136.x
URL link to publisher's version
OpenAccess link to author's accepted manuscript version