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

Overview

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.

Study Type

• Study Type: Interventional
• Study Design
  • Allocation: Non-Randomized
  • Intervention Model: Single Group Assignment
  • Primary Purpose: Treatment
  • Masking: None (Open Label)
• Study Primary Completion Date: March 2016

Interventions

• Drug: FVIII
  • FVIII concentration 35-40 U/kg/dose 1 time/week for 5 months
• Drug: FVIII
  • FVIII concentration 15-20 U/kg/dose 1 time/week for 5 months

Arms, Groups and Cohorts

• Experimental: Arm 1
  • FVIII concentration at 35-40 U/kg/dose 1 time/week for 5 months
• Experimental: Arm 2
  • FVIII concentration at 15-20 U/kg/dose 2 time/week for 5 months

Clinical Trial Outcome Measures

Primary Measures

• Annualized bleeding rate per year
  • Time Frame: 10 months

Secondary Measures

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

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 6 Months

Maximum Age: 20 Years

Are Healthy Volunteers Accepted: Accepts Healthy Volunteers

Investigator Details

• Lead Sponsor
  • Chulalongkorn University
• Provider of Information About this Clinical Study
  • Principal Investigator: Darintr Sosothikul, Associate professor – Chulalongkorn University
• Overall Official(s)
  • Darintr Sosothikul, MD, Principal Investigator, Chulalongkorn University