Clinical trial NCT01817868: Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients
Official title: Evaluation of Efficacy, Safety and Costs of Recombinant FVIII Products Applied to Severe Hemophilia A Patients: Observational Data Collection Study Evaluating On-demand Treatment and Secondary Prophylaxis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia
Keywords: Hemophilia
Turkey
Observational
Adult
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2013-03-26
Enrollment: 73
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult haemophilia patients already on treatment with commercially available recombinant FVIII products.

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.


Eligibility criteria: 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

Primary outcomes:
measure: Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group
timeFrame: After 4 years
measure: Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group
timeFrame: After 4 years
Secondary outcomes:
measure: Number of overall bleeding episodes
timeFrame: After 4 years
measure: Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score)
timeFrame: After 4 years
measure: Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score)
timeFrame: After 4 years
measure: Cost-effectiveness (cost of additional joint bleed)
timeFrame: After 4 years
measure: Cost-utility
timeFrame: After 4 years
measure: Comparison of patient compliance between prophylaxis and on-demand therapy groups
timeFrame: After 4 years
measure: Number of spontaneous bleeds
timeFrame: After 4 years
measure: Quality of life as measured with the SF-36
timeFrame: Baseline and after 4 years
measure: Quality of life as measured with Hemo-QoL
timeFrame: Baseline and after 4 years
Other outcomes:
Locations:
city: Multiple Locations
country: Turkey
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References: