Hemophilia A
Conditions
Keywords
Moderate hemophilia A, Severe hemophilia A, Intermediate dose prophylaxis, Low dose prophylaxis
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.
Interventions
DRUGFVIII
FVIII concentration 35-40 U/kg/dose 1 time/week for 5 months
Sponsors
Chulalongkorn University
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Months to 20 Years
Healthy volunteers
Yes
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annualized bleeding rate per year | 10 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Hemophilia joint health score | 10 months | — |
| Amount of FVIII use | 10 months | — |
| Quality of life score | 10 months | EQ-5D-5L (Thailand version) |
| Number of hospital stays | 10 months | — |
| Number of school days loss | 10 months | — |
Countries
Thailand
Outcome results
None listed