| 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 |
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