Clinical and Health-related Outcome of rFVIIIFc Prophylaxis

Study Overview
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Brief Summary
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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.

Official Title
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Clinical and Health-related Outcome of rFVIIIFc Prophylaxis in Patients With Haemophilia A

Conditions
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Hemophilia A

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients suffering from moderate to severe haemophilia A Age ≥ 18-years Treatment with FVIII prophylaxis Submitted written informed consent Exclusion Criteria: 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)

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients with Haemophilia A<br>Patients suffering from moderate to severe haemophilia A Age ≥ 18-years Treatment with FVIII prophylaxis Submitted written informed consent | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| total annual bleeding rate | number of total bleedings | one year | 
| orthopaedic joint status (Haemophilia health joint score) | 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). | one year | 
| pressure pain thresholds | physiological Parameter in Newton | one year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| spontaneous joint ABR | number of total joint bleedings | one year | 
| subjective quality of life (SF-36) | 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 | two years | 
| subjective physical performance (HEP-Test-Q) | 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 | two years | 
| self-perceived functional abilities (Haemophilia Activities List) | 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) | two years |