Patient Functioning and Well-being, Economic, and Clinical Impact of Hemophilia A and Its Treatment

Completed

• Conditions: Hemophilia A
• Interventions: Drug: Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222)

• Registration Number: NCT02396862
• Lead Sponsor: Bayer

Brief Summary

The purpose of the study is to improve the understanding of key patient reported outcomes such as quality of life as well as clinical outcomes in hemophilia A, in a global real world setting.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2015-03-19
• Study First Submitted QC: 2015-03-19
• Study First Posted: 2015-03-24
• Study Start: 2015-12-09
• Primary Completion: 2018-01-17
• Study Completion: 2018-03-26
• Status Verified: 2019-03
• Last Update Submitted: 2019-03-26
• Last Update Posted: 2019-03-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 272

Inclusion Criteria

• Age 16 or over.
• Have documentation of physician-confirmed diagnosis of moderate or severe Hemophilia A (severity defined as moderate = FVIII activity 1% to 5% and severe = FVIII activity ≤1%).
• Signed written informed consent provided by the patient or the patient's parents for patients under the age of 18 (dependent of local regulations).
• Signed written assent is also required for patients under the age 18 years (dependent on local regulations).
• Plan to receive at least half of their Hemophilia care at the registry site.
• Willing and able to enter data as per the data collection schedule.
• Currently receiving prophylactic or on demand treatment (including within last 6months for on demand).
• Expected life expectancy of at least 2 years.

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Exclusion Criteria

• Patients with Hemophilia B
• Patients with von Willebrand disease (vWD)
• Patients with other rare bleeding disorders
• Unable to comply with the study protocol

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients with moderate to severe Hemophilia A / Cohort 1	Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222)	Patients aged over 16 years, with documented physician-confirmed diagnosis of moderate or severe Hemophilia A (severity defined as moderate = FVIII activity 1% to 5% and severe = FVIII activity ≤1%)

Primary Outcome Measures

Name	Time	Method
Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).	Baseline and at 60 months
Patient Reported Outcome: Change of Quality of Life as measured by EuroQol-5 Dimension questionnaire 5 Level (EQ5D), Short Form Health Survey (SF-12), Hemophilia Quality of Life Measure for Adults (HAEMO-QoL-A)	Baseline and at 60 months
Patient Reported Outcome: Change of treatment patterns as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)	Baseline and at 60 months
Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)	Baseline and at 60 months
Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)	Baseline and at 60 months
Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (Short Form) (BPI-SF)	Baseline and at 12 months
Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)	Baseline and at 60 months
Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective	Baseline and at 60 months
Patient Reported outcome: Change of well-being as measured by Psychological General Well Being Schedule (PGWBI) as well as Smoking and drinking behavior and recreational drug use	Baseline and at 60 months

Secondary Outcome Measures

Name	Time	Method
Clinical Outcome: Change of Hemophilia A status	Baseline and every 12 months up to 60 months	Hemophilia A status as defined by: - Last measured FVIII level - History of intracranial hemorrhage and age of occurrence - Number of Bleeding episodes/events in the last year - Location of bleed - Confirmed microbleeds on past imaging - PK assessment (if available): Name of FVIII product for most recent PK evaluation - Dose of Factor VIII product administered for most recent PK evaluation - Measured Factor VIII level - Factor VIII half-life and time point of measurement -- Central Venous Access Device (CVAD) or AV (arterial-venous) fistulae
Clinical Outcome: Change of Joint Status	Baseline and every 12 months up to 60 months	Joint Status as defined by: - Hemophilia Joint Health Score/ Pettersson Additive Scale - Range of motion - Imaging (where available) - Target joints and past surgical procedures
Clinical Outcome: Baseline disease characteristics	Baseline	Baseline disease characteristics and disease course as defined by: - Date of diagnosis of Hemophilia A - Age at first treatment with factor replacement therapy (child/adult) - Type of factor VIII gene mutation (if available) - Factor VIII level at initial diagnosis (if available) - History of inhibitor and assay used for detection - Date of determination of peak level of inhibitor
Clinical Outcome: Change of treatment patterns including Hemophilia treatments via patient chart	Baseline and every 12 months up to 60 months	Treatment patterns as measured by: - Treatment choice and dose (Factor VIII use and type) - Treatment Regimen - Immune tolerance therapy - Products for inhibitors - Blood bank products - Non plasma and topical products