Clinical trial NCT02581969: Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A."
Official title: Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A." POTTER-10
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Keywords: Hemophilia A, Congenital
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2015-10-21
Enrollment: 43
Sex: MALE
Minimum age (years): 17
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: A total of approximately 50 patients with a diagnosis of severe haemophilia A (FVIII \< 1%) , who are already on Kogenate Bayer treatment ongoing for long-term secondary prophylaxis or on-demand therapy and have properly completed the follow-up in the original Potter study are expected to be enrolled.

Brief summary: Following the performance of the POTTER observational study, whose primary objective was to collect data on the benefits of secondary prophylaxis versus on demand treatment in terms of prevention of bleeding episodes, the present study aims to extend the observation time up to 10 years, of the same population involved in the previous study.

The present study has been designed to allow a focus on long term disease-related damage at joint level in subjects with severe haemophilia A, as well as on the pharmacoeconomics impact of the two different treatment regimens.


Eligibility criteria: Inclusion Criteria:

* Completion of follow-up in the original Potter study without having being excluded from the Intention To Treat efficacy analysis of the primary endpoint;
* Written informed consent specifically issued for the 5-year extension.

Exclusion Criteria:

* Switching of treatment to a Factor VIII concentrate different from Kogenate Bayer/Helixate NexGen after the end of the previous follow-up period.

Primary outcomes:
measure: Number of joint bleeding episodes per year
timeFrame: 5 years period
Secondary outcomes:
measure: Total number of bleeding episodes per year
timeFrame: 5 years period
measure: Total amount of Recombinant Factor VIII consumption
timeFrame: 5 years period
measure: Changes of prophylaxis dose
timeFrame: 5 years period
measure: Changes of frequency of infusions
timeFrame: 5 years period
measure: Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score)
timeFrame: 5 years period
measure: Proportions of patients who will change the therapeutic regimen during the study
timeFrame: 5 years period
measure: Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score)
timeFrame: 5 years period
measure: Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol)
timeFrame: 5 years period
measure: Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36)
timeFrame: 5 years period
measure: Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D)
timeFrame: 5 years period
measure: Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related
timeFrame: 5 years period
measure: Number of patients with poor compliance
timeFrame: 5 years period
measure: Number of patients with adverse events
timeFrame: 5 years period
Other outcomes:
Locations:
city: Multiple Locations
country: Italy
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References: