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