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

Study Overview
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Brief Summary
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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
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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.

Official Title
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Prospective, Observational, 2-cohort Study of Adult Patients With Severe Hemophilia A in Greece. Cost,Clinical Outcomes and Quality of Life Comparison Between on Demand and Secondary Prophylaxis Treatment Strategies

Conditions
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Hemophilia A

Intervention / Treatment
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* Other: Survey
* Other: Survey


Participation Criteria
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Eligibility Criteria
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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. Exclusion Criteria: Patients that have developed inhibitors against factor VIII. Patients participating in an investigational program with interventions outside of routine clinical practice.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Cohort 1<br>Adult patients with severe Hemophilia A.(25 patients on Secondary Prophylaxis treatment) | Other: Survey<br>* Recombinant antihemophilic factor VIII (FVIII) on Secondary Prophylaxis treatment scheduled according to daily routine<br>|
| Cohort 2<br>Adult patients with severe Hemophilia A.(50 patients on On Demand treatment) | Other: Survey<br>* Recombinant antihemophilic factor VIII (FVIII) On Demand treatment scheduled according to daily routine<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annual direct medical cost of specialized hemophilia centre for patients with the on-demand strategy |  | At 18 months | 
| Annual direct medical cost of specialized hemophilia centre for patients with secondary prophylaxis |  | At 18 months | 

Secondary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cost, Clinical Outcomes, Quality of Life, On Demand Treatment, Secondary Prophylaxis