Clinical and Health-related Outcome of rFVIIIFc Prophylaxis in Patients With Haemophilia A
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 48
- 试验地点
- 2
- 主要终点
- total annual bleeding rate
研究概览
简要总结
Current standard therapy for patients with haemophilia (PwH) in the prevention of bleeding episodes is a prophylactic intravenous treatment with recombinant coagulation factor (F) VIII (Haemophilia A) or rather FIX (Haemophilia B) two to three times weekly. With the development of recombinant factor VIII Fc fusion protein (rFVIIIFc) the conventional routine prophylaxis regime is complemented by an extended half-life (EHL) factor replacement prophylaxis with the potential of improved bleed prevention and reduced injection frequency at similar factor consumption. Aim of this longitudinal multicentre study is to evaluate the influence of an EHL factor replacement regime with rFVIIIFc on haemophilic specific parameters (annual bleeding rate, bleeding localisation), joint status, pain, functional parameters, treatment adherence and health-related quality of life in PwH A.
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 suffering from moderate to severe haemophilia A
- •Age ≥ 18-years
- •Treatment with FVIII prophylaxis
- •Submitted written informed consent
排除标准
- •Patients suffering from other bleeding diseases
- •Patients with inhibitors
- •Patients without written informed consent
- •Age < 18-year-old
- •Any surgeries up to 6 months before the examination date
- •Suffering from different rheumatologic diseases like M. Bechterew, Psoriasis or other local or generalized joint infections (Borreliosis, septic arthritis)
结局指标
主要结局
total annual bleeding rate
时间窗: one year
number of total bleedings
orthopaedic joint status (Haemophilia health joint score)
时间窗: one year
The clinical joint status will be examined in all patients with haemophilia by the World Federation Joint Examination Score and Haemophilia Joint Health Score. Higher score points imply an increased deficit in the functional and structural joint status as a sign of more pronounced haemophilic arthropathy, with a maximum possible value of 124 (no deficits = 0).
pressure pain thresholds
时间窗: one year
physiological Parameter in Newton
次要结局
- spontaneous joint ABR(one year)
- subjective quality of life (SF-36)(two years)
- subjective physical performance (HEP-Test-Q)(two years)
- self-perceived functional abilities (Haemophilia Activities List)(two years)
研究者
Prof. Dr. Dr. Thomas Hilberg
Head of Department
University of Wuppertal