D66
Conditions
Interventions
Group 1: Each patient will be followed up for about 3 years. Visits at the sites will follow routine practice and are anticipated to take place in semi-annual intervals.
Sponsors
CSL Behring GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • The treatment decision was made by the attending physician in accordance with standard medical practice and independently of this study • Written and personally dated consent of the patient or for minors of the legal representative after a detailed explanation of the observational study by the attending physician. The consent must correspond to the presumed will of the minor, if one can be determined. The underage patient should also be given the opportunity to consent in writing, provided that his / her age allows to do so according to the physician • diagnosed with Hemophilia A • treatment with AFSTYLA® according to the medical practice and the latest product information for the treatment and prevention of bleeding events
Exclusion criteria
Exclusion criteria: • currently participating in a clinical trial • known hypersensitivity to the active substance or other ingredients • known allergic reaction to hamster proteins • presence of inhibitors to FVIII or to AFSTYLA® (from = 0,6 BE /ml) and/or immune tolerance treatment at the time of recruitment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - annualized bleeding rate (ABR) for all bleeds, - annualized spontaneous bleeding rate (AsBR), - hemostatic effectiveness of AFSTYLA® for the treatment of bleeding episodes (number of injections and dose required) - investigator’s rating of hemostatic effectiveness of AFSTYLA® for prophylaxis, prevention of bleeding episodes in times with risk and on-demand treatment by a 4-point-verbal-rating-scale | — |
Secondary
| Measure | Time frame |
|---|---|
| - patients’ consumption of AFSTYLA® (overall consumption and consumption for prophylactic treatment), - characteristics of bleeds (e.g. severity and location), - number of patients receiving prophylaxis, incl. dosing of AFSTYLA® (in IU/kg bodyweight) and infusion frequency during prophylactic treatment, - safety endpoints: -- number of patients developing inhibitors to FVIII or to AFSTYLA® -- number, type and severity of adverse events - assessment of physical activity level of the patients by a verbal 5-point VRS - quality of life assessed by the Haem-A-QoL for adults and the Haemo-QoL for children and adolescents - joint status assessed by the Hemophilia Joint Health Score (HJHS 2.1-score) or WFH (World Federation of Hemophilia)-Score (if recorded) - recording of other medications (e.g Hemlibra® as basic therapy and AFSTYLA® as on-demand medication) Haem-A-QoL / Haemo-QoL will be completed once within 6 months of the start of observation and once more approximately 12 months after initial filling. All other parameters are documented if they are collected within the medical routine. | — |
Countries
Austria, Germany
Contacts
Public ContactThomas Linhoff
CSL Behring GmbH
Outcome results
None listed