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

Phase 4

Completed

• Conditions: Hemophilia A
• Interventions: Drug: FVIII

• Registration Number: NCT02727647
• Lead Sponsor: Chulalongkorn University

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2015-05
• Status Verified: 2015-10
• Primary Completion: 2016-03
• Study Completion: 2016-03
• Study First Submitted: 2016-03-30
• Study First Submitted QC: 2016-04-01
• Last Update Submitted: 2016-04-01
• Study First Posted: 2016-04-04
• Last Update Posted: 2016-04-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 16

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Arm 1	FVIII	FVIII concentration at 35-40 U/kg/dose 1 time/week for 5 months
Arm 2	FVIII	FVIII concentration at 15-20 U/kg/dose 2 time/week for 5 months

Primary Outcome Measures

Name	Time	Method
Annualized bleeding rate per year	10 months

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Division of Hemato/oncology, Department of Pediatrics, Faculty of medicine,Chulalongkorn U; 🇹🇭 Bangkok, Pathumwan, Thailand