Clinical and Health-related Outcome of rFVIIIFc Prophylaxis
- Conditions
- Hemophilia A
- Registration Number
- NCT04583930
- Lead Sponsor
- Prof. Dr. Dr. Thomas Hilberg
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- Male
- Target Recruitment
- 48
- Patients suffering from moderate to severe haemophilia A
- Age ≥ 18-years
- Treatment with FVIII prophylaxis
- Submitted written informed consent
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)
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method total annual bleeding rate one year number of total bleedings
orthopaedic joint status (Haemophilia health joint score) 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 one year physiological Parameter in Newton
- Secondary Outcome Measures
Name Time Method spontaneous joint ABR one year number of total joint bleedings
subjective quality of life (SF-36) two years The standarized questionnaire SF-36 includes a physical and psychological score. The higher the score, the higher the physical or psychological quality of life. A higher score implies a better physical and mental quality of life
subjective physical performance (HEP-Test-Q) two years The final version of the standarized questionnaire consists of 25 items pertaining to the domains 'mobility', 'strength \& coordination', 'endurance' and 'body perception'. The response options were a five-point Likert scale (ranging from 1 = never to 5 = always). Some of the items had to be re-coded; subscales and the total score were transformed to a scale of 0-100 with high scores indicating better physical performance
self-perceived functional abilities (Haemophilia Activities List) two years The Haemophilia Activities List measures the impact of hemophilia on self-perceived functional abilities in adults. It contains 42 multiple choice questions in seven domains: Lying/sitting/kneeling/standing (8 items), Functions of the legs (9 items), Functions of the arms (4 items), Use of transportation (3 items), Self-care (5 items), Household tasks (6 items), Leisure activities and sports (7 items)
Trial Locations
- Locations (2)
Department of Sports Medicine
🇩🇪Wuppertal, Northwest, Germany
Department of Sports Medicine, University of Wuppertal
🇩🇪Wuppertal, North Rhine-Westphalia, Germany