Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients

Study Overview
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Brief Summary
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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: 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. Observation and measurement: History, interesting clinical data and laboratory data will be recorded in Clinical record Form (CRF) 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 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.

Official Title
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Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients

Conditions
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Hemophilia A

Intervention / Treatment
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* Drug: FVIII
* Drug: FVIII


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arm 1<br>FVIII concentration at 35-40 U/kg/dose 1 time/week for 5 months | Drug: FVIII<br>* FVIII concentration 35-40 U/kg/dose 1 time/week for 5 months<br>* Other names: Hemofil-M;|
| Experimental: Arm 2<br>FVIII concentration at 15-20 U/kg/dose 2 time/week for 5 months | Drug: FVIII<br>* FVIII concentration 15-20 U/kg/dose 1 time/week for 5 months<br>* Other names: Hemofil-M;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annualized bleeding rate per year |  | 10 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hemophilia joint health score |  | 10 months | 
| Amount of FVIII use |  | 10 months | 
| Quality of life score | EQ-5D-5L (Thailand version) | 10 months | 
| Number of hospital stays |  | 10 months | 
| Number of school days loss |  | 10 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Moderate hemophilia A, Severe hemophilia A, Intermediate dose prophylaxis, Low dose prophylaxis