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Patient-Reported Outcomes in Nephrolithiasis: Can We Do Better?
Jonathan S Ellison1, Marc Williams2, Francis X Keeley2
11 Division of Pediatric Urology, Seattle Children's Hospital, University of Washington , Seattle, Washington.
Patient-reported outcome measures (PROMs) are crucial for urinary stone disease care. However, their use and reporting in clinical trials for nephrolithiasis are inconsistent, highlighting a need for better disease-specific instruments and standardized reporting.
Area of Science:
- Urology
- Health Outcomes Research
Background:
- Nephrolithiasis (urinary stone disease) poses a significant global health challenge.
- Patient-reported outcome measures (PROMs) are essential for patient-centered care in managing chronic conditions.
- Understanding PROM utilization in nephrolithiasis research is vital for improving patient outcomes.
Purpose of the Study:
- To review existing knowledge on PROMs for urinary stone disease.
- To characterize the application of PROMs in randomized controlled trials (RCTs) for nephrolithiasis.
Main Methods:
- Systematic literature search of MEDLINE and EMBASE databases for RCTs in nephrolithiasis (1990-2014).
- Assessment of PROM utilization within identified RCTs by two independent reviewers.
- Inclusion criteria focused on studies reporting health-related quality of life (HRQOL) and other patient-reported outcomes.
Main Results:
- Validated PROMs include symptom diaries, pain scales, and HRQOL instruments.
- HRQOL is significantly impacted in nephrolithiasis patients, influenced by factors like gender and treatment.
- Only one validated HRQOL instrument specific to nephrolithiasis exists; no disease-specific instrument for treatment outcomes.
- PROMs reporting in RCTs is variable, with few trials using validated HRQOL instruments and none using disease-specific tools.
Conclusions:
- Health-related quality of life is diminished in patients with urolithiasis, particularly near acute stone events.
- There is a lack of validated, disease-specific PROMs for nephrolithiasis, especially for assessing treatment outcomes.
- Future research, including RCTs for urolithiasis, should prioritize the development and standardized reporting of condition-specific PROMs to capture the disease's complex nature.
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