Clinical trial NCT01159587: Prophylaxis Versus On-demand Therapy Through Economic Report
Official title: Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A. POTTER
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2010-07-09
Enrollment: 58
Sex: ALL
Minimum age (years): 12
Maximum age (years): 55
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Haemophilics

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.


Eligibility criteria: 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

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

Primary outcomes:
measure: 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
timeFrame: Every 6 months
Secondary outcomes:
measure: Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes
timeFrame: Every 6 months
measure: Muscolo skeletal evaluation by Orthopedic Joint Score
timeFrame: Every 12 months
measure: Radiological Evaluation by Pettersson Score
timeFrame: Baseline and after 3-5 years
measure: Health related quality of life
timeFrame: Every 12 months
measure: cost-effectiveness and utility, patient compliance, adverse events
timeFrame: Every 6 months
Other outcomes:
Locations:
city: Many Locations
country: Italy
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References:
citation: Tagliaferri A, Feola G, Molinari AC, Santoro C, Rivolta GF, Cultrera DB, Gagliano F, Zanon E, Mancuso ME, Valdre L, Mameli L, Amoresano S, Mathew P, Coppola A; POTTER Study Group. Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study. Thromb Haemost. 2015 Jul;114(1):35-45. doi: 10.1160/TH14-05-0407. Epub 2015 Apr 9.