End points for renal complications associated with SCD
| Condition . | End point . | Test . | Outcome/outcome measure . | Type of end point . | Ages . |
|---|---|---|---|---|---|
| CKD, albuminuria | Albuminuria or proteinuria | First morning void or 2 consecutive untimed albumin/creatinine ratio or protein/creatine ratio | Percent decline in albuminuria | Direct: severe albuminuria; surrogate: moderate albuminuria | All age groups |
| CKD, GFR | Renal replacement therapy | Initiation of renal replacement therapy (may differ based on clinical practice) | Percentage of patients who require initiation of renal replacement therapy | Direct | All age groups |
| CKD, GFR | GFR | Measured GFR and estimated GFR | Percentage of patients who progress in stage of CKD or percentage of decline in GFR | Surrogate | Adults |
| Hypertension | Cardiovascular end point | Clinically defined | Number of patients who develop stroke, myocardial infarction, death | Direct | All age groups |
| Hypertension | Systolic or diastolic blood pressure; pediatric studies should consider use of ambulatory blood pressure monitoring | Blood pressure measurement | Change in systolic or diastolic blood pressure (5 mm Hg) | Future | All age groups |
| Hyposthenuria | Urine concentration availability | Water deprivation test; not >10 h in children; meaningful tests are those concentrating urine >500 mOsm/kg H2O | Percentage of patients with preservation of urine concentration ability | Surrogate | Infants or toddlers |
| Kidney injury | Acute kidney injury defined by serum creatinine or urine output; biomarker of kidney injury | Serum creatinine or urine output; novel biomarkers of kidney injury (eg, NGAL, KIM-1) | Percentage of patients who develop acute kidney injury; reduction in urine biomarkers (25% change) | Future | All age groups |
| Condition . | End point . | Test . | Outcome/outcome measure . | Type of end point . | Ages . |
|---|---|---|---|---|---|
| CKD, albuminuria | Albuminuria or proteinuria | First morning void or 2 consecutive untimed albumin/creatinine ratio or protein/creatine ratio | Percent decline in albuminuria | Direct: severe albuminuria; surrogate: moderate albuminuria | All age groups |
| CKD, GFR | Renal replacement therapy | Initiation of renal replacement therapy (may differ based on clinical practice) | Percentage of patients who require initiation of renal replacement therapy | Direct | All age groups |
| CKD, GFR | GFR | Measured GFR and estimated GFR | Percentage of patients who progress in stage of CKD or percentage of decline in GFR | Surrogate | Adults |
| Hypertension | Cardiovascular end point | Clinically defined | Number of patients who develop stroke, myocardial infarction, death | Direct | All age groups |
| Hypertension | Systolic or diastolic blood pressure; pediatric studies should consider use of ambulatory blood pressure monitoring | Blood pressure measurement | Change in systolic or diastolic blood pressure (5 mm Hg) | Future | All age groups |
| Hyposthenuria | Urine concentration availability | Water deprivation test; not >10 h in children; meaningful tests are those concentrating urine >500 mOsm/kg H2O | Percentage of patients with preservation of urine concentration ability | Surrogate | Infants or toddlers |
| Kidney injury | Acute kidney injury defined by serum creatinine or urine output; biomarker of kidney injury | Serum creatinine or urine output; novel biomarkers of kidney injury (eg, NGAL, KIM-1) | Percentage of patients who develop acute kidney injury; reduction in urine biomarkers (25% change) | Future | All age groups |
KIM-1, kidney injury molecule 1; NGAL, neutrophil gelatinase-associated lipoprotein.