Clinical trial NCT02727647: Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients
Official title: Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hemophilia A
Keywords: Moderate hemophilia A
Severe hemophilia A
Intermediate dose prophylaxis
Low dose prophylaxis
Lead sponsor: Chulalongkorn University
Sponsor class: OTHER
First posted: 2016-04-04
Enrollment: 16
Sex: ALL
Minimum age (years): 6
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Annualized bleeding rate per year
timeFrame: 10 months
Secondary outcomes:
measure: Hemophilia joint health score
timeFrame: 10 months
measure: Amount of FVIII use
timeFrame: 10 months
measure: Quality of life score
timeFrame: 10 months
measure: Number of hospital stays
timeFrame: 10 months
measure: Number of school days loss
timeFrame: 10 months
Other outcomes:
Locations:
city: Bangkok
country: Thailand
facility: Division of Hemato/oncology, Department of Pediatrics, Faculty of medicine,Chulalongkorn U
state: Pathumwan
Study officials:
affiliation: Chulalongkorn University
name: Darintr Sosothikul, MD
role: PRINCIPAL_INVESTIGATOR
References: