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Hemophilia Inhibitor Scope

Element Considered Final Measures in Toolkit
Inhibitors Factor IX Activity in Plasma
Factor VIII Activity in Plasma
Quantitative Measure of Factor IX Inhibitor Activity
Quantitative Measure of Factor VIII Inhibitor Activity
Quantitative Measure of Factor VIII Inhibitor Activity in the Presence of Emicizumab
Bleeding Counts of Acute Bleeds in Hemophilia
Outcome measures to evaluate effectiveness of therapy Response to Factor IX Infusion - Individual Pharmacokinetic Study
Response to Factor IX Infusion - Population-based Pharmacokinetic Study
Response to Factor VIII Infusion - Individual Pharmacokinetic Study
Response to Factor VIII Infusion - Population-based Pharmacokinetic Study
Response to Factor VIII Infusion in the Presence of Emicizumab - Individual Pharmacokinetic Study
Response to Factor VIII Infusion in the Presence of Emicizumab - Population-based Pharmacokinetic Study
Patient reported outcomes School and Work Attendance
Disease-specific Health-related Quality of Life in Hemophilia
Physical activity and function Hemophilia-specific Functional Activity
Joint health Score
Adherence to treatment Patient Recall of Clotting Factor Administration
Utilization of health care