Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients
Status:
Completed
Trial end date:
2016-03-01
Target enrollment:
Participant gender:
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.