Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients

Bayer

Status: Completed

Conditions: Hemophilia

Treatments: Other: Recombinant Factor VIII (Kogenate, BAY14-2222)

Study type: Observational

Funder types: Industry

Identifiers

NCT01817868

KG1210TR (Other Identifier)

16368

Details and patient eligibility

About

The project is an observational, multi-central, prospective, non-interventional and open-label data collection study on secondary prophylaxis with recombinant FVIII products in adolescents and adults with severe hemophilia A (FVIII < 1%).

It will be a controlled observation of patients on secondary prophylaxis versus on-demand treatment regimen. Patients will be enrolled preferably on a 1:1 basis with regards to prophylaxis and on-demand treatment.

The patient enrollment period will be 2 years with a follow-up (observation period) of 2 years for each patient. Based on the primary effectiveness parameters (joint bleeds and overall bleeds per year) an observation period of 2 years is considered sufficient although it has to be admitted that it is rather short to assess the progression of orthopedic status. Previously treated prophylaxis patients with at least 50 exposure days and patients with continuing prophylaxis treatment will be included.

Enrollment: 73 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age ≥ 18 years
• Severe hemophilia A (FVIII<1%) diagnosis
• Prior treatment or ongoing treatment with on-demand or prophylaxis regimens according to Turkish guidelines and requirements
• Previously treated patients with at least >50 exposure days
• Written informed consent signed by patient/legal representative

Exclusion criteria

• Currently on immune tolerance treatment
• Platelet count < 75,000/mm3
• Participation in another study
• Existence of inhibitors in the past and in the last currently available blood sample prior to study start (Bethesda titer < 0.6 BU/ml)
• Existence of inhibitor history in family members who also are diagnosed with hemophilia A
• Having been on primary prophylaxis as defined in the introduction
• Participation in another study

Trial design

73 participants in 1 patient group

Group 1

Treatment:

Other: Recombinant Factor VIII (Kogenate, BAY14-2222)