Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs) |
|
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Primary |
Percentage of Participants With SAEs and AESIs |
|
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Primary |
Number of SAEs and AESIs |
|
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
Number of Bleeding Episodes |
The total, spontaneous, traumatic, and joint bleeds will be analyzed. |
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
Annualized Bleeding Rate (ABR) |
The total, spontaneous, traumatic, and joint bleeds will be analyzed. ABR is calculated as the total bleeding episodes divided by the total time at risk. |
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
Number of Participants With Zero Bleeds |
|
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
Correlation Analysis of ABR as a Function of Mean Factor IX (FIX) Activity |
Correlation between ABR and mean FIX activity will be explored using a statistical modeling approach (like, generalized estimating equations [GEE] model for ABR with mean FIX activity as the independent variable). |
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
Number of Participants With FIX Activity |
FIX activity will be assessed using the one-stage activated partial thromboplastin time assay and a chromogenic assay. |
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
Number of Participants Using FIX Replacement Therapy |
|
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
Number of Participants Remaining Free of Previous Continuous Routine FIX Prophylaxis |
|
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
Number of Participants With New Target Joints |
Target joint is defined as 3 or more spontaneous bleeding episodes into a single joint within a consecutive 6-month period. |
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
Percentage of Resolution of Target Joints |
Target joint is defined as 3 or more spontaneous bleeding episodes into a single joint within a consecutive 6-month period. |
From Year 5 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Utility Value |
The EQ-5D-5L questionnaire consists of the EQ-5D-5L descriptive system and the EQ-5D visual analogue scale (VAS). The EQ-5D-5L descriptive system of health-related QoL consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single overall health state utility value. This utility value will be derived using the EQ-5D-5L Crosswalk Index Value Calculator v2.xls [Hernández Alava et al, 2023], using the United Kingdom value set. |
From Year 6 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
EQ-5D-5L VAS Overall Score |
The EQ-5D-5L questionnaire consists of the EQ-5D-5L descriptive system and the EQ-5D VAS. The EQ-5D VAS, which measures overall health status on a vertical visual analogue scale, ranges from 0 to 100. A higher score indicates better QoL. |
From Year 6 after administration of CSL222 in parent study up to Year 15 |
|
| Secondary |
Hem-A-QoL Overall Score |
The Hem-A-QoL (Hemophilia Quality of Life Questionnaire for Adults) consists of 46 items comprising 10 domains: physical health, feelings, treatment, work and school, dealing with hemophilia, family planning, future, partnerships and sexuality, sports and leisure, and view of yourself for which responses will be recorded from 5 options (never, seldom, sometimes, often, and always; for some items, there is also a "not applicable" option). The total score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflecting a better QoL. |
From Year 6 after administration of CSL222 in parent study up to Year 15 |
|
