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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-05-31

Study Completion Date

2016-03-31

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:

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.

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Detailed Description

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Conditions

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Hemophilia A

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Arm 1

FVIII concentration at 35-40 U/kg/dose 1 time/week for 5 months

Group Type EXPERIMENTAL

FVIII

Intervention Type DRUG

FVIII concentration 35-40 U/kg/dose 1 time/week for 5 months

Arm 2

FVIII concentration at 15-20 U/kg/dose 2 time/week for 5 months

Group Type EXPERIMENTAL

FVIII

Intervention Type DRUG

FVIII concentration 15-20 U/kg/dose 1 time/week for 5 months

Interventions

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FVIII

FVIII concentration 35-40 U/kg/dose 1 time/week for 5 months

Intervention Type DRUG

FVIII

FVIII concentration 15-20 U/kg/dose 1 time/week for 5 months

Intervention Type DRUG

Other Intervention Names

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Hemofil-M Hemofil-M

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

Minimum Eligible Age

6 Months

Maximum Eligible Age

20 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes