Table 1 Summary of the outcomes of importance and metrics to consider
Outcomes of importance | Relevant metrics to consider |
|---|---|
Reduced burden of living with haemophilia | |
• Life • Family • Education/school • Employment • Activities | • Family life, marital status, children • Educational attendance, attainment • Employment duration, underemployment, attendance • Impact on daily living, activities of daily living, mobility impairment, assistance required • Current health status (HRQoL) |
Reduced complications associated with haemophilia and treatment | |
• Joint disease • Pain/depression/anxiety • HIV/HCV • Obesity • Other comorbidities | • Joint status • Pain (chronic, acute, interference with activity, timing, medication) • Depression • Resource utilization • Mortality, longevity |