Clinical trial NCT02319070: 2-cohort Study of Adult Patients With Severe Hemophilia A in Greece
Official title: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Keywords: Cost
Clinical Outcomes
Quality of Life
On Demand Treatment
Secondary Prophylaxis
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2014-12-18
Enrollment: 72
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: This will be a single center prospective two-cohort observational study conducted in a hospital setting.Patients will be enrolled from the biggest specialty management center in Greece

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Annual direct medical cost of specialized hemophilia centre for patients with the on-demand strategy
timeFrame: At 18 months
measure: Annual direct medical cost of specialized hemophilia centre for patients with secondary prophylaxis
timeFrame: At 18 months
Secondary outcomes:
measure: Mean cost per joint bleed episodes for overall patients
timeFrame: At 18 months
measure: Mean cost per joint bleed episodes by cohort for patients
timeFrame: At 18 months
measure: Number of joint bleed episodes for overall patients
timeFrame: At 18 months
measure: Number of joint bleed episodes by cohort for patients.
timeFrame: At 18 months
measure: Severity of joint bleed episodes for overall patients.
timeFrame: At 18 months
measure: Severity of joint bleed episodes by cohort for patients.
timeFrame: At 18 months
measure: Mean EQ-VAS score as reported from patients at the time of a major bleed.
timeFrame: At 18 months
measure: Mean EQ-5D score as reported from patients at the time of a major bleed.
timeFrame: At 18 months
measure: Mean change from baseline in Quality of Life scores derived from the Haem-A-Qol and EQ-5D questionnaires
timeFrame: Baseline and at 18 months
Other outcomes:
Locations:
city: Multiple Locations
country: Greece
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References: