Outcomes of Prophylaxis and On-demand Treatment for Severe Hemophiliacs and Collection of Baseline Data in Hemophiliacs in Taiwan

NCT ID: NCT02908243

Last Updated: 2016-09-20

Basic Information

• Recruitment Status: UNKNOWN
• Total Enrollment: 350 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2015-02-28
• Study Completion Date: 2018-12-31

Brief Summary

Primary prophylaxis in severe hemophiliacs is defined that prophylaxis therapy starts before 2 years of age and prior to any clinically evident joint bleeding or after first joint bleeding and prior to the onset of joint damage irrespective of age, joints can be kept normal or very mildly damaged till teenage or adulthood. Primary prophylaxis has been proved to be more beneficial and cause less damage to joint than "on-demand" therapy. Primary prophylaxis is also known to be able to decrease the occurrence of factor VIII inhibitor and is the most advanced and useful, cost-effective therapy for hemophilia care. However, it requires 2 to 3 injections of factor VIII or IX of 20-50 IU/Kg doses every week, it costs a lot of expenses. In the year 2013, we tried hard to discuss with Bureau of National Health Insurance (BNHI) and have meeting a couple of time, eventually a guideline of an intermediate-dose prophylaxis for severe hemophilia was established and a consensus was reached that this prophylactic treatment will be cost effective without increased burden of total budget. This guideline was finally approved by BNHI and will be implemented from July 1st, 2014. In oder to evaluate the efficacy of prophylaxis treatment, patients will be arranged to come back to each hemophilia center at least once a year to have investigation of doses and annual consumption of clotting factors, frequencies and causes of bleedings, especially joint bleedings, joint outcome by studies of hemophilia joint health score (HJHS), hemophilia actives list and health-related quality of life. These results will be collected and compared between intermediate-dose prophylaxis group of patients and on demand treatment group of patients.

The life span of hemophiliacs has been improved remarkably in recent years due to sufficient and adequate treatments, especially prophylaxis treatment, therefore comorbidities in the hemophilic population, e.g. hypertension, diabetes, hyperlipidemia and cancer, etc, have been found with prevalences close to those in non-hemophilic population. It is worth that the prevalence of these comorbidity will also be investigated. In addition, basic data of the patients including age, sex, severity, the development of inhibitor and viral infection etc will also be collected for analysis.

Conditions

Hemophilia

Study Design

• Observational Model Type: COHORT

Study Groups

Prophylaxis

Severe hemophilia A patients who receive factor VIII with dose of 15-25 IU/Kg, 2-3 times/week Severe hemophilia B patients who receive factor IX with dose of 30-50 IU/Kg, 1-2 times/week

No interventions assigned to this group

On-demand

Severe hemophilia A patients who receive factor VIII injection when bleeding occurs with the dose according to the criteria of World Federation of Hemophilia (WFH); Severe hemophilia B patients who receive factor IX injection when bleeding occurs with the dose according to the criteria of World Federation of Hemophilia (WFH)

No interventions assigned to this group

Collection of baseline data in all hemophilia A and B patients

All severe, moderate and mild types of hemophilia A and B patients

collection of baseline data

Intervention Type: OTHER

collection of baseline data including ages, types, severity, inhibitors and comorbidities

Interventions

collection of baseline data

collection of baseline data including ages, types, severity, inhibitors and comorbidities

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• all severe hemophilia A and B patients more than 2 years of age and without inhibitor

Exclusion Criteria

• female hemophilia
• hemophiliac patients with inhibitor > = 0.6 Bethesda unit (BU)

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Changhua Christian Hospital

OTHER

Sponsor Role: collaborator

Taipei Medical University Hospital

OTHER

Sponsor Role: collaborator

Taipei Veterans General Hospital, Taiwan

OTHER_GOV

Sponsor Role: collaborator

Taipei Medical University Shuang Ho Hospital

OTHER

Sponsor Role: collaborator

Taichung Veterans General Hospital

OTHER

Sponsor Role: collaborator

China Medical University Hospital

OTHER

Sponsor Role: collaborator

Kaohsiung Medical University Chung-Ho Memorial Hospital

OTHER

Sponsor Role: collaborator

Taiwan Society of Thrombosis and Hemostasis

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ming-Ching Shen, BS

Role: STUDY_CHAIR

Changhua Christian Hospital, Changhua, Taiwan

Other Identifiers

141115

Identifier Type: -

Identifier Source: org_study_id