Monitoring the success of novel hemophilia therapies
| 1. Hemostasis restoration |
| a. Bleeding (patient-reported bleeding, objective documentation with US, MRI) |
| b. Factor level: one-stage and chromogenic assays |
| c. Global assay of hemostasis: thrombin generation, thromboelastography, microfluidic assay |
| 2. Joint damage |
| a. Sensitive physical exam, such as the HJHS |
| b. Structural changes on ultrasound or MRI |
| c. Functional impact on gait via gait lab assessment |
| d. Bone health: bone density scanning, bone biomarkers |
| 3. Patient-reported outcomes |
| a. Quality of life |
| i. Generic: SF36, PROMIS measures, Peds QL |
| ii. Hemophilia specific: PROBE61 and Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) |
| b. Pain |
| i. McGill Pain Scale |
| ii. Brief Pain Inventory |
| iii. PROMIS |
| iv. PROBE |
| v. CHO-KLAT |
| 1. Hemostasis restoration |
| a. Bleeding (patient-reported bleeding, objective documentation with US, MRI) |
| b. Factor level: one-stage and chromogenic assays |
| c. Global assay of hemostasis: thrombin generation, thromboelastography, microfluidic assay |
| 2. Joint damage |
| a. Sensitive physical exam, such as the HJHS |
| b. Structural changes on ultrasound or MRI |
| c. Functional impact on gait via gait lab assessment |
| d. Bone health: bone density scanning, bone biomarkers |
| 3. Patient-reported outcomes |
| a. Quality of life |
| i. Generic: SF36, PROMIS measures, Peds QL |
| ii. Hemophilia specific: PROBE61 and Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) |
| b. Pain |
| i. McGill Pain Scale |
| ii. Brief Pain Inventory |
| iii. PROMIS |
| iv. PROBE |
| v. CHO-KLAT |
US, ultrasound.