Hemophilia A
Conditions
Keywords
Hemophilia A, Congenital
Brief summary
Following the performance of the POTTER observational study, whose primary objective was to collect data on the benefits of secondary prophylaxis versus on demand treatment in terms of prevention of bleeding episodes, the present study aims to extend the observation time up to 10 years, of the same population involved in the previous study. The present study has been designed to allow a focus on long term disease-related damage at joint level in subjects with severe haemophilia A, as well as on the pharmacoeconomics impact of the two different treatment regimens.
Interventions
Administered 20-30 IU/kg 3 times a week
Sponsors
Bayer
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
17 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
* Completion of follow-up in the original Potter study without having being excluded from the Intention To Treat efficacy analysis of the primary endpoint; * Written informed consent specifically issued for the 5-year extension.
Exclusion criteria
* Switching of treatment to a Factor VIII concentrate different from Kogenate Bayer/Helixate NexGen after the end of the previous follow-up period.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of joint bleeding episodes per year | 5 years period |
Secondary
| Measure | Time frame |
|---|---|
| Total amount of Recombinant Factor VIII consumption | 5 years period |
| Changes of prophylaxis dose | 5 years period |
| Changes of frequency of infusions | 5 years period |
| Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score) | 5 years period |
| Proportions of patients who will change the therapeutic regimen during the study | 5 years period |
| Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score) | 5 years period |
| Total number of bleeding episodes per year | 5 years period |
| Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36) | 5 years period |
| Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D) | 5 years period |
| Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related | 5 years period |
| Number of patients with poor compliance | 5 years period |
| Number of patients with adverse events | 5 years period |
| Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol) | 5 years period |
Countries
Italy
Outcome results
None listed