2-cohort Study of Adult Patients With Severe Hemophilia A in Greece

Completed

• Conditions: Hemophilia A
• Interventions: Other: Survey

• Registration Number: NCT02319070
• Lead Sponsor: Bayer

Brief Summary

This study is a prospective, single center, observational, 2-cohort study of adult patients with severe Hemophilia A. There is no randomization procedure and all patients will be treated as per usual clinical practice. Patients will be followed up for 18 months after enrolment.

Detailed Description

The primary therapy for hemophilia is coagulation factor replacement, given either episodically on demand (OD) for the treatment of acute bleeds or prophylactically to prevent them.

The last years there has been an interest to study the comparative effectiveness of OD vs. SP (Secondary Prophylaxis) treatment strategies so as to create an evidence based platform to guide both clinical decision making and quantify the economic aspects of each treatment alternative. In Greece relevant information is lacking, thus it is imperative to study how patients on different treatment strategies are managed in a real-life setting in Greece, including the main factors associated with increased health care utilization, clinical success and patient well being.

Study Timeline

• Study First Submitted: 2014-12-12
• Study First Submitted QC: 2014-12-17
• Study First Posted: 2014-12-18
• Study Start: 2015-01-13
• Primary Completion: 2017-05-28
• Study Completion: 2018-02-28
• Status Verified: 2018-06
• Last Update Submitted: 2018-06-19
• Last Update Posted: 2018-06-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 72

Inclusion Criteria

• Patients diagnosed with severe Hemophilia A.
• Age ≥18 years old
• Patients able to read and write
• Patients receiving Factor VIII substitute therapy, either on-demand or as secondary prophylaxis.
• Patients under the same therapeutic strategy (On Demand or Secondary Prophylaxis) for at least 6 months prior to enrolment.

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Exclusion Criteria

• Patients that have developed inhibitors against factor VIII.
• Patients participating in an investigational program with interventions outside of routine clinical practice.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Cohort 1	Survey	Adult patients with severe Hemophilia A.(25 patients on Secondary Prophylaxis treatment)
Cohort 2	Survey	Adult patients with severe Hemophilia A.(50 patients on On Demand treatment)

Primary Outcome Measures

Name	Time	Method
Annual direct medical cost of specialized hemophilia centre for patients with secondary prophylaxis	At 18 months
Annual direct medical cost of specialized hemophilia centre for patients with the on-demand strategy	At 18 months

Secondary Outcome Measures

Name	Time	Method
Mean cost per joint bleed episodes for overall patients	At 18 months
Mean cost per joint bleed episodes by cohort for patients	At 18 months
Number of joint bleed episodes for overall patients	At 18 months
Severity of joint bleed episodes by cohort for patients.	At 18 months
Mean change from baseline in Quality of Life scores derived from the Haem-A-Qol and EQ-5D questionnaires	Baseline and at 18 months	Haem-A-Qol Specific questionnaire for hemophilia and EQ-5D The EuroQol standardized instrument of health outcome
Number of joint bleed episodes by cohort for patients.	At 18 months
Mean EQ-VAS score as reported from patients at the time of a major bleed.	At 18 months	EQ-VAS score is Standardized questionnaire for hemophilia
Severity of joint bleed episodes for overall patients.	At 18 months
Mean EQ-5D score as reported from patients at the time of a major bleed.	At 18 months	EQ-5D The EuroQol standardized instrument of health outcome