Brief Title
Outcomes of Prophylaxis and On-demand Treatment for Severe Hemophiliacs and Collection of Baseline Data in Hemophiliacs in Taiwan
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.
Study Type
Observational [Patient Registry]
Primary Outcome
annual bleeding rate
Condition
Hemophilia
Intervention
collection of baseline data
Study Arms / Comparison Groups
Prophylaxis
Description: 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
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Other
Estimated Enrollment
350
Start Date
February 2015
Completion Date
December 2018
Primary Completion Date
December 2018
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)
Gender
Male
Ages
2 Years - 80 Years
Accepts Healthy Volunteers
No
Contacts
Ming-Ching Shen, BS, ,
Administrative Informations
NCT ID
NCT02908243
Organization ID
141115
Responsible Party
Sponsor
Study Sponsor
Taiwan Society of Thrombosis and Hemostasis
Collaborators
Changhua Christian Hospital
Study Sponsor
Ming-Ching Shen, BS, Study Chair, Changhua Christian Hospital, Changhua, Taiwan
Verification Date
September 2016