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

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 Overview

• Status: Unknown
• Conditions: Hemophilia
• Intervention / Treatment: Other: collection of baseline data

• Study Type: Observational
• Enrollment (Anticipated): 350

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 80 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Probability Sample

Study Population

200 Severe hemophilia A patients

Description

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 Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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
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)
Collection of baseline data in all hemophilia A and B patients; All severe, moderate and mild types of hemophilia A and B patients	Other: collection of baseline data; collection of baseline data including ages, types, severity, inhibitors and comorbidities

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
annual bleeding rate	spontaneous and traumatic joint and other site bleedings	through study completion, an average of 1 year

Collaborators and Investigators

• Sponsor: Taiwan Society of Thrombosis and Hemostasis
• Collaborators: China Medical University Hospital; Kaohsiung Medical University Chung-Ho Memorial Hospital; Taipei Veterans General Hospital, Taiwan; Taipei Medical University Hospital; Taipei Medical University Shuang Ho Hospital; Changhua Christian Hospital; Taichung Veterans General Hospital
• Investigators: Study Chair: Ming-Ching Shen, BS, Changhua Christian Hospital, Changhua, Taiwan

Study record dates

Study Major Dates

• Study Start: February 1, 2015
• Primary Completion (Anticipated): December 1, 2018
• Study Completion (Anticipated): December 1, 2018

Study Registration Dates

• First Submitted: September 8, 2016
• First Submitted That Met QC Criteria: September 16, 2016
• First Posted (Estimate): September 20, 2016

Study Record Updates

• Last Update Posted (Estimate): September 20, 2016
• Last Update Submitted That Met QC Criteria: September 16, 2016
• Last Verified: September 1, 2016

More Information

Terms related to this study

• Keywords: Comorbidity; Hemophilia; Primary prophylaxis; Pettersson Score; Joint function study
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: 141115

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Plan to share whole data