Hemophilia Inhibitor Research
| 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 |