Prospective, Observational, 2-cohort Study of Adult Patients With Severe Hemophilia A in Greece. Cost,Clinical Outcomes and Quality of Life Comparison Between on Demand and Secondary Prophylaxis Treatment Strategies
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Bayer
- 入组人数
- 72
- 主要终点
- Annual direct medical cost of specialized hemophilia centre for patients with secondary prophylaxis
研究概览
简要总结
This study is a prospective, single center, observational, 2-cohort study of adult patients with severe Hemophilia A. There is no randomization procedure and all patients will be treated as per usual clinical practice. Patients will be followed up for 18 months after enrolment.
详细描述
The primary therapy for hemophilia is coagulation factor replacement, given either episodically on demand (OD) for the treatment of acute bleeds or prophylactically to prevent them.
The last years there has been an interest to study the comparative effectiveness of OD vs. SP (Secondary Prophylaxis) treatment strategies so as to create an evidence based platform to guide both clinical decision making and quantify the economic aspects of each treatment alternative. In Greece relevant information is lacking, thus it is imperative to study how patients on different treatment strategies are managed in a real-life setting in Greece, including the main factors associated with increased health care utilization, clinical success and patient well being.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed with severe Hemophilia A.
- •Age ≥18 years old
- •Patients able to read and write
- •Patients receiving Factor VIII substitute therapy, either on-demand or as secondary prophylaxis.
- •Patients under the same therapeutic strategy (On Demand or Secondary Prophylaxis) for at least 6 months prior to enrolment.
排除标准
- •Patients that have developed inhibitors against factor VIII.
- •Patients participating in an investigational program with interventions outside of routine clinical practice.
结局指标
主要结局
Annual direct medical cost of specialized hemophilia centre for patients with secondary prophylaxis
时间窗: At 18 months
Annual direct medical cost of specialized hemophilia centre for patients with the on-demand strategy
时间窗: At 18 months
次要结局
- Mean cost per joint bleed episodes for overall patients(At 18 months)
- Mean cost per joint bleed episodes by cohort for patients(At 18 months)
- Number of joint bleed episodes for overall patients(At 18 months)
- Severity of joint bleed episodes by cohort for patients.(At 18 months)
- Mean change from baseline in Quality of Life scores derived from the Haem-A-Qol and EQ-5D questionnaires(Baseline and at 18 months)
- Mean EQ-5D score as reported from patients at the time of a major bleed.(At 18 months)
- Severity of joint bleed episodes for overall patients.(At 18 months)
- Number of joint bleed episodes by cohort for patients.(At 18 months)
- Mean EQ-VAS score as reported from patients at the time of a major bleed.(At 18 months)