Brief Title
Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients
Official Title
Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients
Brief Summary
Title: Comparison of different prophylaxis regimens for hemophilia A pediatric patients Principal Investigator: Assistant Professor Darintr Sosothikul, MD Research Question: Does different factor VIII dosage effect outcome of hemophilia treatment in term of breakthrough bleeding, number of hospital stay and day-off from school? Type Research: Clinical research Study design: Single center clinical trials Concise methodology: 1. Study Population: Children with hemophilia A who received treatment at KCMH from May 2015 to March 2016 will be enrolled in this study. The consent will be obtained before the study. 2. Observation and measurement: 1. History, interesting clinical data and laboratory data will be recorded in Clinical record Form (CRF) 2. Measurement: i. Complete blood count (CBC), Factor VIII level, Factor VIII inhibitor level ii. Number of breakthrough bleedings, number of hospital stay and day-off from school iii. Joint score from Hemophilia Joint Health Score 2.1 iv. EQ-5D-5L quality of life assessment score 3. Data analysis: The p-value of less than 0.05 will be considered statistically significant. Mann-Whitney test will be used to test correlation of these variables (CBC, Factor VIII level, Factor VIII inhibitor level, Number of breakthrough bleedings, number of hospital stay, day-off from school, Hemophilia Joint Health Score 2.1, EQ-5D-5L quality of life assessment score) Sample size: 16 patients Potential impacts: The outcomes of different factor VIII concentrate dose between 15-20 U/kg/dose 2 times/week and 35-40 U/kg/dose 1 time/week will be revealed. These outcomes include number of breakthrough bleeding, number of hospital stay, day-off from school, joint health and quality of life. The result of this study will guide further study on optimal dose and duration of factor VIII treatment of hemophilia A patients in the future.
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
Annualized bleeding rate per year
Secondary Outcome
Hemophilia joint health score
Condition
Hemophilia A
Intervention
FVIII
Study Arms / Comparison Groups
Arm 1
Description: FVIII concentration at 35-40 U/kg/dose 1 time/week for 5 months
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
Drug
Estimated Enrollment
16
Start Date
May 2015
Completion Date
March 2016
Primary Completion Date
March 2016
Eligibility Criteria
Inclusion Criteria: - Hemophilia patients with moderate (factor level 1-3%) or severe (factor level <1%) severity Exclusion Criteria: - Platelet count less than 100,000 /mm3 or other bleeding tendency - Hemophilia patients who have FVIII inhibitor >0.6 BU (modified Nijmogen method) - Hemophilia patients who have no bleeding symptoms
Gender
All
Ages
6 Months - 20 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Contacts
Darintr Sosothikul, MD, ,
Location Countries
Thailand
Location Countries
Thailand
Administrative Informations
NCT ID
NCT02727647
Organization ID
3125056
Responsible Party
Principal Investigator
Study Sponsor
Chulalongkorn University
Study Sponsor
Darintr Sosothikul, MD, Principal Investigator, Chulalongkorn University
Verification Date
October 2015