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

Hemophilia Clinical Trial

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT02908243
• Other study ID #: 141115
• Status: Enrolling by invitation
• Phase: N/A
• First received: September 8, 2016
• Last updated: September 16, 2016
• Start date: February 2015
• Est. completion date: December 2018

Study information

• Verified date: September 2016
• Source: Taiwan Society of Thrombosis and Hemostasis
• Contact: n/a
• Is FDA regulated: No
• Health authority: Taiwan: Institutional Review Board
• Study type: Observational [Patient Registry]

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 350
• Est. completion date: December 2018
• Est. primary completion date: December 2018
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 2 Years to 80 Years

Eligibility

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)

Study Design

Observational Model: Cohort

Related Conditions & MeSH terms

• Hemophilia
• Hemophilia A

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (8)

Lead Sponsor	Collaborator
Taiwan Society of Thrombosis and Hemostasis	Changhua Christian Hospital, China Medical University Hospital, Kaohsiung Medical University Chung-Ho Memorial Hospital, Taichung Veterans General Hospital, Taipei Medical University Hospital, Taipei Medical University Shuang Ho Hospital, Taipei Veterans General Hospital, Taiwan

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	annual bleeding rate	spontaneous and traumatic joint and other site bleedings	through study completion, an average of 1 year	No