Real-World Effectiveness of PEGylated, Recombinant Antihemophilic Factor (Adynovate) Prophylaxis in Patients With Hemophilia A in Canada: A Retrospective, Intra-patient Comparison With a Before-After Design
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 153
- 试验地点
- 1
- 主要终点
- Percentage of Time Spent With Factor Levels Above 0.01 IU/ml
研究概览
简要总结
The main aims of the study are to assess the safety profile of Adynovate as well as how well people respond to the preventive treatment with Adynovate.
This study is about reviewing and collecting data of the participants before and after the switch to Adynovate that are already available. No new information will be collected during this study. The total time for data collection in the study will be approximately 72 months (36 months before and 36 months after switching to Adynovate). Participants will not receive Adynovate as part of this study.
As participants are not treated in this study, they do not need to visit their doctor in addition to their normal visits.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with severe hemophilia A.
- •All age groups (less than [<] 12 and greater than [>] 12 years of age).
- •Participant with greater than equal to (>=) 150 documented exposure days (EDs).
- •Treated with Adynovate:
- •Having a PK analysis done/data available for post-hoc modeling
- •Having recorded clinical outcomes analysis: for > 6 months
- •Treated with a SHL/EHL-FVIII product for at least six months before switching to Adynovate
- •To qualify for the secondary objective participants will need to have a WAPPS study performed on Adynovate and on the SHL/EHL-FVIII they were treated before switching.
排除标准
- •Any participant who meets any of the following criteria will not qualify for entry into the study:
- •Participants with only on-demand Factor VIII (FVIII) use.
- •Current presence of FVIII inhibitory antibodies. (Participants with a history of inhibitors, if any, will be considered for a sensitivity analysis).
- •Diagnosis of other inherited or acquired hemostatic defect other than hemophilia A.
结局指标
主要结局
Percentage of Time Spent With Factor Levels Above 0.01 IU/ml
时间窗: approximately 72 months
Percentage of time spent with factor levels above 0.01 (International units per milliliter \[IU/ml\], is calculated as: time spent with factor levels above 0.01 IU/ml/ total time in the study. The calculation will be performed by simulating, based on the infusions recorded in the treatment diaries and the individual PK profiles, all the times intervals between each infusion and the time at which the concentration of 0.01 IU/mL is reached, and the time between reaching the 0.01 IU/mL and the subsequent infusion.
Number of Participants With Adverse Events (AEs)
时间窗: approximately 72 months
An AEs is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. Number of participants with AEs related to inhibitor development, thrombosis, death, infection, cancer and other will be reported.
次要结局
- Terminal Half-life of Adynovate(approximately 72 months)
- Annualized Total Factor Consumption for Bleeds(approximately 72 months)
- Theoretical Factor Consumption(approximately 72 months)
- Hemophilia Joint Health Score (HJHS)- Total Score(approximately 72 months)
- Maximum Observed Drug Concentration (Cmax) of Adynovate(approximately 72 months)
- Annualized Bleeding Rate (ABR)(approximately 72 months)
- Annualized Spontaneous Bleeding Rate (AsBR),(approximately 72 months)
- Health-Related Quality of Life (HRQoL) Assessed by Patient-reported Outcome Burdens and Experiences (PROBE) Questionnaire(approximately 72 months)
- Percentage of Time Spent With Factor Levels Above 0.03 IU/ml(approximately 72 months)
- Annualized Total Factor Consumption(approximately 72 months)
- Estimated Factor Consumption(approximately 72 months)
- Percentage of Time Spent With Factor Levels Above 0.05 IU/ml(approximately 72 months)
- Clearance (Cl) of Adynovate(approximately 72 months)
- Area Under the Curve (AUC) of Adynovate(approximately 72 months)
- Annualized Joint Bleeding Rate (AjBR)(approximately 72 months)
- HRQoL Assessed by EuroQoL Group 5-Dimension 5-Level Self-Report (EQ-5D-5L)(approximately 72 months)