A Prospective, International, Longitudinal, Observational Disease Registry of Patient-reported Outcomes (PROs) and the Association With Hemophilia A and Its Treatment in Patients With Moderate to Severe Hemophilia A
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Bayer
- 入组人数
- 272
- 主要终点
- Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
研究概览
简要总结
The purpose of the study is to improve the understanding of key patient reported outcomes such as quality of life as well as clinical outcomes in hemophilia A, in a global real world setting.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Age 16 or over.
- •Have documentation of physician-confirmed diagnosis of moderate or severe Hemophilia A (severity defined as moderate = FVIII activity 1% to 5% and severe = FVIII activity ≤1%).
- •Signed written informed consent provided by the patient or the patient's parents for patients under the age of 18 (dependent of local regulations).
- •Signed written assent is also required for patients under the age 18 years (dependent on local regulations).
- •Plan to receive at least half of their Hemophilia care at the registry site.
- •Willing and able to enter data as per the data collection schedule.
- •Currently receiving prophylactic or on demand treatment (including within last 6months for on demand).
- •Expected life expectancy of at least 2 years.
排除标准
- •Patients with Hemophilia B
- •Patients with von Willebrand disease (vWD)
- •Patients with other rare bleeding disorders
- •Unable to comply with the study protocol
研究组 & 干预措施
Patients with moderate to severe Hemophilia A / Cohort 1
Patients aged over 16 years, with documented physician-confirmed diagnosis of moderate or severe Hemophilia A (severity defined as moderate = FVIII activity 1% to 5% and severe = FVIII activity ≤1%)
干预措施: Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222) (Drug)
结局指标
主要结局
Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
时间窗: Baseline and at 60 months
Patient Reported Outcome: Change of Quality of Life as measured by EuroQol-5 Dimension questionnaire 5 Level (EQ5D), Short Form Health Survey (SF-12), Hemophilia Quality of Life Measure for Adults (HAEMO-QoL-A)
时间窗: Baseline and at 60 months
Patient Reported Outcome: Change of treatment patterns as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
时间窗: Baseline and at 60 months
Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
时间窗: Baseline and at 60 months
Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
时间窗: Baseline and at 60 months
Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (Short Form) (BPI-SF)
时间窗: Baseline and at 12 months
Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)
时间窗: Baseline and at 60 months
Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
时间窗: Baseline and at 60 months
Patient Reported outcome: Change of well-being as measured by Psychological General Well Being Schedule (PGWBI) as well as Smoking and drinking behavior and recreational drug use
时间窗: Baseline and at 60 months
次要结局
- Clinical Outcome: Change of Hemophilia A status(Baseline and every 12 months up to 60 months)
- Clinical Outcome: Change of Joint Status(Baseline and every 12 months up to 60 months)
- Clinical Outcome: Baseline disease characteristics(Baseline)
- Clinical Outcome: Change of treatment patterns including Hemophilia treatments via patient chart(Baseline and every 12 months up to 60 months)