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

Completed

• Conditions: Hemophilia
• Interventions: Other: Recombinant Factor VIII (Kogenate, BAY14-2222)

• Registration Number: NCT01817868
• Lead Sponsor: Bayer

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-01-04
• Study First Submitted: 2013-03-12
• Study First Submitted QC: 2013-03-21
• Study First Posted: 2013-03-26
• Primary Completion: 2018-07-16
• Study Completion: 2019-05-23
• Status Verified: 2020-05
• Last Update Submitted: 2020-05-22
• Last Update Posted: 2020-05-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 73

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

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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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Group 1	Recombinant Factor VIII (Kogenate, BAY14-2222)	-

Primary Outcome Measures

Name	Time	Method
Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group	After 4 years
Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group	After 4 years

Secondary Outcome Measures

Name	Time	Method
Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score)	After 4 years
Number of overall bleeding episodes	After 4 years
Cost-utility	After 4 years	The costs of care of subjects with haemophilia will be evaluated adopting the perspective of the payer, i.e. the Social Security Institution \[SSI\].
Quality of life as measured with the SF-36	Baseline and after 4 years
Cost-effectiveness (cost of additional joint bleed)	After 4 years
Number of spontaneous bleeds	After 4 years
Quality of life as measured with Hemo-QoL	Baseline and after 4 years
Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score)	After 4 years
Comparison of patient compliance between prophylaxis and on-demand therapy groups	After 4 years