Projects

Project Description

Patient-oriented clinical outcomes assessments (COAs) include patient-reported outcome (PRO) assessments and observer reported outcome (ObsRO) assessments. PROs are self-report measures (i.e., responses come directly from the patient without any interpretation by a clinician/observer) that are focused Review of literature consisting of previous studies done on the same area of work stating potential risks and benefits and outcome measures on evaluating symptoms or function (1). 

Nephrotic syndrome (NS), caused by rare kidney diseases, is characterized by high grade proteinuria, hypoalbuminemia, and persistent or relapsing and remitting fluid overload. Children and adults with uncontrolled NS experience manifestations of fluid overload including regional or total body edema with impaired mobility, dyspnea, pain, and fatigue. Over 5,200 pediatric hospitalizations annually are attributable to NS in the U.S. (2–4) Comparable hospitalization statistics are not available for adults. In addition, kidney diseases that cause NS are leading causes of end stage kidney disease (ESKD) in children and adults (5) and represent a significant patient and public health burden. There are currently few drugs that are approved for the treatment of NS associated kidney diseases. The failure of these drugs to control the disease is manifest by the hospitalizations, ESKD, and daily impact of the disease on patients. To promote patient centered drug development, clinical outcome assessments like patient-reported outcome (PRO) measures and observer

reported outcome (ObsRO) measures can be developed, validated, and then used to assist in understanding patient manifestations of disease activity and function. PRO concepts and subsequent developed measures require assessment for comprehension of individual items, response options, directions, and recall period in order to ensure the utility of any new measure for use as an FDA-approved measure for future drug development clinical trials (6).