End points for cardiovascular complications associated with SCD
| Condition . | End point . | Test . | Outcome/outcome measure . | Type of end point . | Ages . |
|---|---|---|---|---|---|
| PH and risk of death | Right heart catheter defined PH | RAP, CI, mPAP, and PVR | Percent change in RAP, CI, mPAP, TPG, or PVR | Direct: RAP, mPAP, CI; surrogate: PVR | Adults |
| PH and risk of death | TRV | Doppler echocardiography | TRV ≥3.0 m/s or TRV ≥2.5 m/s and NT-pro-BNP level >160 pg/mL | Surrogate | Adults |
| PH and risk of death | Composite model | Doppler echocardiography, NT-pro-BNP, and 6-min walk test | TRV >2.5 m/s, NT-pro-BNP level ≥160 pg/mL, and 6-min walk test <330 m | Surrogate | Adults |
| Diastolic dysfunction | E/e′ and E/A; myocardial fibrosis | Echocardiogram; cardiac MRI | Percent improvement toward normal | Future | All age groups |
| Functional exercise capacity | Walk distance | 6-min walk test | Change in distance walked (30-40 m) | Direct | Adults |
| ACS | Incidence using an accepted definition; progression using an accepted definition | Clinically defined | Percentage of patients who develop ACS; percent of patients that clinically worsen; respiratory support, O2 requirement, transfusion therapy | Direct | All age groups |
| Asthma | Development of ACS, pain event, or asthma exacerbation | Clinically defined | Time to event in incidence of ACS, pain crisis, or use of rescue inhaler (number of events/days using rescue inhaler) low FEV1 at baseline | Direct | All age groups |
| Indirect | |||||
| Thromboembolism | Pulmonary embolism, VTE | Clinically defined thrombus | Percentage of patients with either pulmonary or VTE | Direct | All age groups |
| Condition . | End point . | Test . | Outcome/outcome measure . | Type of end point . | Ages . |
|---|---|---|---|---|---|
| PH and risk of death | Right heart catheter defined PH | RAP, CI, mPAP, and PVR | Percent change in RAP, CI, mPAP, TPG, or PVR | Direct: RAP, mPAP, CI; surrogate: PVR | Adults |
| PH and risk of death | TRV | Doppler echocardiography | TRV ≥3.0 m/s or TRV ≥2.5 m/s and NT-pro-BNP level >160 pg/mL | Surrogate | Adults |
| PH and risk of death | Composite model | Doppler echocardiography, NT-pro-BNP, and 6-min walk test | TRV >2.5 m/s, NT-pro-BNP level ≥160 pg/mL, and 6-min walk test <330 m | Surrogate | Adults |
| Diastolic dysfunction | E/e′ and E/A; myocardial fibrosis | Echocardiogram; cardiac MRI | Percent improvement toward normal | Future | All age groups |
| Functional exercise capacity | Walk distance | 6-min walk test | Change in distance walked (30-40 m) | Direct | Adults |
| ACS | Incidence using an accepted definition; progression using an accepted definition | Clinically defined | Percentage of patients who develop ACS; percent of patients that clinically worsen; respiratory support, O2 requirement, transfusion therapy | Direct | All age groups |
| Asthma | Development of ACS, pain event, or asthma exacerbation | Clinically defined | Time to event in incidence of ACS, pain crisis, or use of rescue inhaler (number of events/days using rescue inhaler) low FEV1 at baseline | Direct | All age groups |
| Indirect | |||||
| Thromboembolism | Pulmonary embolism, VTE | Clinically defined thrombus | Percentage of patients with either pulmonary or VTE | Direct | All age groups |
FEV1, forced expiratory volume in 1 second; MRI, magnetic resonance imaging.