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."

N/ACompletedNCT02581969

Bayer43 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Hemophilia A

Interventions

Recombinant Factor VIII (Kogenate FS, BAY14-2222)DRUG

Administered 20-30 IU/kg 3 times a week

Eligibility

Sex: MALEMin age: 17 YearsMax age: 60 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Number of joint bleeding episodes per year

Time frame: 5 years period

Secondary Outcomes

Total number of bleeding episodes per year

Time frame: 5 years period

Total amount of Recombinant Factor VIII consumption

Time frame: 5 years period

Changes of prophylaxis dose

Time frame: 5 years period

Changes of frequency of infusions

Time frame: 5 years period

Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score)

Time frame: 5 years period

Proportions of patients who will change the therapeutic regimen during the study

Time frame: 5 years period

Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score)

Time frame: 5 years period

Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol)

Time frame: 5 years period

Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36)

Time frame: 5 years period

Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D)

Time frame: 5 years period

Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related

Time frame: 5 years period

Number of patients with poor compliance

Time frame: 5 years period

Number of patients with adverse events

Time frame: 5 years period

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."

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes