Prophylaxis Versus On-demand Therapy Through Economic Report

Completed

• Conditions: Hemophilia A
• Interventions: Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

• Registration Number: NCT01159587
• Lead Sponsor: Bayer

Brief Summary

The project is a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII \< 1%).

The observational period will cover at least 5 years per patient.

The long-term secondary prophylaxis group will be compared versus on-demand treatment group by the assessment of orthopedic status progression and pharmacoeconomics evaluation.

Detailed Description

Not available

Study Timeline

• Study Start: 2004-07
• Study First Submitted: 2010-07-08
• Study First Submitted QC: 2010-07-08
• Study First Posted: 2010-07-09
• Primary Completion: 2010-12
• Study Completion: 2010-12
• Status Verified: 2011-04
• Last Update Submitted: 2011-04-12
• Last Update Posted: 2011-04-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 58

Inclusion Criteria

• age ≥ 12 years and ≤ 55 years
• severe haemophilia A (FVIII < 1%)
• absence of inhibitors (Bethesda titre < 0.6 BU/ml)
• Previous Treated Patients (prior exposure days > 200)
• Kogenate Bayer administered 20-30 IU/kg -3 times a week, for the prophylaxis group
• ≥ 6 joint bleeds requiring treatment with FVIII concentrates in the previous 6 months before enrollment, for on-demand group
• written informed consent

Read More

Exclusion Criteria

• concomitant severe and chronic diseases or congenital skeletal malformation
• unreliability of patient or likelihood of follow-up failure
• presence of inhibitors or history of inhibitors (in the previous 2 years)
• currently on immune tolerance treatment
• hepatic cirrhosis or liver disease in rapid progression
• AIDS
• platelet count < 75,000/mm3
• presence of conditions that influence negatively patient´s compliance
• participation in another study

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Group 1	Recombinant Factor VIII (Kogenate FS, BAY14-2222)	-
Group 2	Recombinant Factor VIII (Kogenate FS, BAY14-2222)	-

Primary Outcome Measures

Name	Time	Method
Evaluate the efficacy, safety and pharmacoeconomic of long-term secondary prophylaxis with Kogenate Bayer in comparison to on-demand treatment with Kogenate Bayer in terms of joint bleeding episodes per year	Every 6 months

Secondary Outcome Measures

Name	Time	Method
Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes	Every 6 months
Muscolo skeletal evaluation by Orthopedic Joint Score	Every 12 months
Radiological Evaluation by Pettersson Score	Baseline and after 3-5 years
Health related quality of life	Every 12 months
cost-effectiveness and utility, patient compliance, adverse events	Every 6 months