Priority end points and measures for pain (non-PRO) in SCD
| Tier . | Prioritization . | Domains . | End point/outcome . | Direct/indirect . | Pros . | Cons . |
|---|---|---|---|---|---|---|
| I | End points currently being used and recommended by the panel | Measure of pain control/function | Heath care utilization: ED/hospitalization | Indirect | Experience with its use in SCD and other pain conditions | Can be affected by many other factors1 |
| Length of stay | ||||||
| Analgesic use (opioid) | ||||||
| Missing work/school | ||||||
| Activity/function | ||||||
| II | End points with experience in SCD in pipeline and available for use | Measures of pain endophenotype | QST | Indirect Not an assessment of pain | Experience with its use in SCD and other pain conditions | Cost Expertise Evoked stimuli (QST) Operator dependence (QST) |
| Functional neuroimaging | High potential for being secondary or correlative end point | Possible impact of SCD biology (imaging) | ||||
| Measures indicating mechanisms: circulating biomarkers/rheology | Variable based on underlying mechanism (eg, selectins) and proposed interventions | Indirect Select based on the mechanism/target | Useful for targeted therapies | Need validation with clinical improvement | ||
| III | Promising end points in animal model/need further exploration | Measures indicating mechanism/function/degree of pain | Grip force | Indirect | Can be translated to humans for further investigation | Needs further validation with clinical pain |
| Facial expression analysis | Direct | |||||
| Skin biopsy | Indirect/pathological changes | |||||
| Circulating biomarkers | Indirect/mechanism based |
| Tier . | Prioritization . | Domains . | End point/outcome . | Direct/indirect . | Pros . | Cons . |
|---|---|---|---|---|---|---|
| I | End points currently being used and recommended by the panel | Measure of pain control/function | Heath care utilization: ED/hospitalization | Indirect | Experience with its use in SCD and other pain conditions | Can be affected by many other factors1 |
| Length of stay | ||||||
| Analgesic use (opioid) | ||||||
| Missing work/school | ||||||
| Activity/function | ||||||
| II | End points with experience in SCD in pipeline and available for use | Measures of pain endophenotype | QST | Indirect Not an assessment of pain | Experience with its use in SCD and other pain conditions | Cost Expertise Evoked stimuli (QST) Operator dependence (QST) |
| Functional neuroimaging | High potential for being secondary or correlative end point | Possible impact of SCD biology (imaging) | ||||
| Measures indicating mechanisms: circulating biomarkers/rheology | Variable based on underlying mechanism (eg, selectins) and proposed interventions | Indirect Select based on the mechanism/target | Useful for targeted therapies | Need validation with clinical improvement | ||
| III | Promising end points in animal model/need further exploration | Measures indicating mechanism/function/degree of pain | Grip force | Indirect | Can be translated to humans for further investigation | Needs further validation with clinical pain |
| Facial expression analysis | Direct | |||||
| Skin biopsy | Indirect/pathological changes | |||||
| Circulating biomarkers | Indirect/mechanism based |
QST, quantitative sensory testing.