A Chart Review Study of Patients With Haemophilia A With Inhibitors Treated With rFVIIIFc (Elocta®) for Immune Tolerance Induction
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 44
- 试验地点
- 3
- 主要终点
- Outcome of ITI with rFVIIIFc: Recovery level
研究概览
简要总结
A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.
详细描述
A multicenter, international, non- interventional, retrospective and prospective medical chart review study. Data will be collected from medical records for patients diagnosed with haemophilia A who have been, or who are currently, treated with rFVIIIFc for ITI . The study will be descriptive in nature and report on baseline characteristics, treatment and outcomes for patients who have been, or who are currently, treated with rFVIIIFc for ITI.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed with haemophilia A who have been, or who are currently, treated with rFVIIIFc for ITI.
- •Signed and dated informed consent provided by the patient, or the patient's legally acceptable representative for patients under the legal age, before any study-related data collection are undertaken. Assent should be obtained from paediatric patients according to local regulations.
排除标准
- •Current participation in any investigational medicinal product trial.
结局指标
主要结局
Outcome of ITI with rFVIIIFc: Recovery level
时间窗: From 2018 to 2022
FVIII recovery level (%)
ITI with rFVIIIFc: Duration
时间窗: From 2018 to 2022
Number of treatment months
Outcome of ITI with rFVIIIFc: Overall outcome
时间窗: From 2018 to 2022
The Investigator will assess overall outcome as: success, partial success, failure, early withdrawal or other.
Outcome of ITI with rFVIIIFc: Time to normal recovery
时间窗: From 2018 to 2022
Treatment time to reach normal recovery levels (≥66% of the expected value)
ITI with rFVIIIFc: Main dose
时间窗: From 2018 to 2022
Main dose will be assessed on the prescribed dose (IU/kg)
Outcome of ITI with rFVIIIFc: Time to undetectable inhibitor titer
时间窗: From 2018 to 2022
Treatment time to reach undetectable inhibitor levels (\<0.6 BU/ml)
Long-term outcome after ITI with rFVIIIFc: Treatment regimen
时间窗: From 2018 to 2022
Treatment regimen will be described as: ITI, prophylaxis or on-demand; and also by product used.
Long-term outcome after ITI with rFVIIIFc: Occurrence of relapse
时间窗: From 2018 to 2022
Occurrence of relapse (Yes/No) will be assessed by the investigator.
ITI with rFVIIIFc: Main injection frequency
时间窗: From 2018 to 2022
Main injection frequency will be assessed on the prescribed frequency
Long-term outcome after ITI with rFVIIIFc: Time to relapse
时间窗: From 2018 to 2022
Time to occurrence of relapse (see outcome #13)
ITI with rFVIIIFc: Concomitant by-passing agents
时间窗: From 2018 to 2022
Product name and main dose will be used to describe any concomitant use of by-passing agents.
Outcome of ITI with rFVIIIFc: Half-life
时间窗: From 2018 to 2022
FVIII half-life (hours)
Outcome of ITI with rFVIIIFc: Time to success
时间窗: From 2018 to 2022
Treatment time to reach success (see outcome #5)
Outcome of ITI with rFVIIIFc: Inhibitor titer levels
时间窗: From 2018 to 2022
BU/ml
Outcome of ITI with rFVIIIFc: Bleeds
时间窗: From 2018 to 2022
Number of bleeds per month during ITI-treatment
Long-term outcome after ITI with rFVIIIFc: Bleeds
时间窗: From 2018 to 2022
Number of bleeds per month.
次要结局
未报告次要终点