D66
Conditions
Interventions
Group 1: Für die Dokumentation (Einschlusskriterien) kommen Probanden in Frage, die die Kriterien einer mittelschweren oder schweren Hämophilie A (Ausgangswert FVIII <5%) erfüllen, beide Geschlechter,
Sponsors
Takeda Pharma Vertrieb GmbH & Co. KG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subject has moderate or severe hemophilia A (baseline factor VIII < 5%) and is being treated with ADVATE or ADYNOVI on any treatment regimen recommended by his/her treating physician. Subject, parent(s) or legally authorized representative has provided written informed consent. For ADYNOVI: Subject aged = 12 years
Exclusion criteria
Exclusion criteria: Subject has known hypersensitivity to the active substance or to any of the excipients. Subject has a known allergic reaction to mouse or hamster proteins.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of haemophilia-affected joint-arthropathy by imaging techniques (e.g. MRI, X-ray, ultrasound) and by assessment of the treating physician using only the pain, bleeding, and physical exam parameter of the Gilbert Scale. | — |
Secondary
| Measure | Time frame |
|---|---|
| Incidence of joint replacement therapies; incidence of target joint operations; Incidence of pseudo-tumor development; Pain associated with bleeding event according to VAS-Score; Quality of life using the Haemo-QoL, SF-10TM, SF-12v2TM and Haem-A-QoL questionnaires; Annualized bleed rate; Hemostatic effectiveness rating of bleeding episodes treated with ADVATE or ADYNOVI; Number of units required for bleed resolution; Number of infusions required for bleed resolution; Number of days off from school or work due to hemophilia A bleeding episodes; Incidence of inhibitors in PTPs; Incidence of inhibitors in PUPs; Incidence of inhibitors after switching to ADYNOVI; Incidence of treatment-related serious AEs; Incidence of treatment-related non-serious AEs; Compliance with the dosing prescribed and its relationship with effectiveness in prophylaxis regimen; Modalities of switching from a standard FVIII product to ADYNOVI (prophylaxis regimen) - Difference in number of weekly prophylactic infusions between previous regimen and ADYNOVI - Difference in number of weekly doses between previous regimen and ADYNOVI; Status of joint health using the Hemophilia Joint Health Score (HJHS) in patients on ADYNOVI | — |
Countries
Germany
Contacts
Public ContactStephan Regensburger
Takeda Pharma Vertrieb GmbH & Co. KG
Outcome results
None listed