Outcome Measures of Clinical Trials in Pediatric Chronic Kidney Disease
Abstract
:1. Introduction
2. Clinical Outcome Measures
2.1. Survival
2.2. KRT
2.3. GFR and KRT as Composite Clinical Outcome Indicators for CKD Progression
3. Alternative Outcome Measures
3.1. Clinical Test/Measurement Value as Surrogate Outcome Indicators
3.1.1. Proteinuria
3.1.2. Blood Pressure
3.1.3. Inconclusive Indicators
4. Patient-Reported Outcome Measures (PROMs)
5. Safety Indicators
6. Gaps and Perspective
7. Conclusions
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
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Level | Definition (Need to Meet All Requirements) |
---|---|
Convincing | Be confirmed by more than one research type At least 2 independent cohort should be included among these research types No apparent unexplained heterogeneity between the studies Research is of high quality and thus is safe to conclude that the relation is not caused by random or systematic error including mix, measure error, and selection bias Have a biologically plausible dose–response relationship For the risk-outcome pairs, significant associations were required after considering the source of potential bias |
Probable | Evidence from at least two independent cohort studies or at least five case-control studies No apparent unexplained heterogeneity between the studies This research is of high quality and thus is safe to conclude from that the relation is not caused by random or systematic error including mix, measure error and, selection bias Have evidence of a biological plausibility |
Suggestive | Have evidence from at least two independent cohort studies or at least five case-control studies Although there may be some unexplained heterogeneity, the direction of the effects is generally consistent Have evidence of a biological plausibility |
Inconclusive | Allow any risk exposure with sufficient data to consider but insufficient evidence to exist for more definitive grading |
Surrogate Outcome Indicators | Level |
---|---|
Proteinuria [36,37,38,39,40,41,42,43,44,45,46,47] | Convincing |
Blood Pressure [41,48,49,50] | Convincing |
Anemia [51] | Inconclusive |
Hypoproteinemia [43,49,51] | Inconclusive |
Dyslipidemia [43,52] | Inconclusive |
Hyperphosphatemia [53] | Inconclusive |
Hypocalcemia [54] | Inconclusive |
Hyperuricemia [55] | Inconclusive |
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Liang, Z.; He, G.; Tao, L.; Zhong, X.; Lin, T.; Jiang, X.; Ding, J. Outcome Measures of Clinical Trials in Pediatric Chronic Kidney Disease. Future 2024, 2, 56-66. https://doi.org/10.3390/future2020005
Liang Z, He G, Tao L, Zhong X, Lin T, Jiang X, Ding J. Outcome Measures of Clinical Trials in Pediatric Chronic Kidney Disease. Future. 2024; 2(2):56-66. https://doi.org/10.3390/future2020005
Chicago/Turabian StyleLiang, Ziyun, Guohua He, Liyuan Tao, Xuhui Zhong, Tianxin Lin, Xiaoyun Jiang, and Jie Ding. 2024. "Outcome Measures of Clinical Trials in Pediatric Chronic Kidney Disease" Future 2, no. 2: 56-66. https://doi.org/10.3390/future2020005