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

N/ACompletedNCT02319070

Bayer72 enrolled

Overview

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.

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 Type

OBSERVATIONAL

Enrollment

Conditions

Hemophilia A

Interventions

SurveyOTHER

Recombinant antihemophilic factor VIII (FVIII) on Secondary Prophylaxis treatment scheduled according to daily routine

SurveyOTHER

Recombinant antihemophilic factor VIII (FVIII) On Demand treatment scheduled according to daily routine

Eligibility

Sex: MALEMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

Unnamed facility

Multiple Locations, Greece

Outcomes

Primary Outcomes

Annual direct medical cost of specialized hemophilia centre for patients with the on-demand strategy

Time frame: At 18 months

Annual direct medical cost of specialized hemophilia centre for patients with secondary prophylaxis

Time frame: At 18 months

Secondary Outcomes

Mean cost per joint bleed episodes for overall patients

Time frame: At 18 months

Mean cost per joint bleed episodes by cohort for patients

Time frame: At 18 months

Number of joint bleed episodes for overall patients

Time frame: At 18 months

Number of joint bleed episodes by cohort for patients.

Time frame: At 18 months

Severity of joint bleed episodes for overall patients.

Time frame: At 18 months

Severity of joint bleed episodes by cohort for patients.

Time frame: 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

Time frame: 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

Time frame: At 18 months

Data from ClinicalTrials.gov

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