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

NCT02908243 | Unknown DMC

• 1 other identifier: 141115
• Study Type: observational
• Target: 350
• Locations: 0 countries
• Sites: N/A

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

Keywords

Hemophilia; Primary prophylaxis; Pettersson Score; Joint function study; Comorbidity

Outcome Measures

Primary Outcomes (1)

• annual bleeding rate

  spontaneous and traumatic joint and other site bleedings

  through study completion, an average of 1 year

Study Arms (3)

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

Interventions

collection of baseline data OTHER

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

Collection of baseline data in all hemophilia A and B patients

Eligibility Criteria

• Age: 2 Years - 80 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

200 Severe hemophilia A patients

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Taiwan Society of Thrombosis and Hemostasis: lead
• Changhua Christian Hospital: collaborator
• Taipei Medical University Hospital: collaborator
• Taipei Veterans General Hospital, Taiwan: collaborator
• Taipei Medical University Shuang Ho Hospital: collaborator
• Taichung Veterans General Hospital: collaborator
• China Medical University Hospital: collaborator
• Kaohsiung Medical University Chung-Ho Memorial Hospital: collaborator

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Ming-Ching Shen, BS

  Changhua Christian Hospital, Changhua, Taiwan

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Target Duration: 3 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 8, 2016
• First Posted: September 20, 2016
• Study Start: February 1, 2015
• Primary Completion: December 1, 2018
• Study Completion: December 1, 2018
• Last Updated: September 20, 2016

Record last verified: 2016-09

Data Sharing

• IPD Sharing: Will share