Low VW Activity in Adolescent HMB
- Conditions
- Von Willebrand Factor Deficiency
- Interventions
- Other: Genetic AnalysisOther: Medical Record Data AbstractionOther: Pictorial Blood Assessment Chart (PBAC) scoreOther: Complete Bleeding Symptom ISTH Bleeding Assessment Tool
- Registration Number
- NCT02933411
- Lead Sponsor
- Baylor College of Medicine
- Brief Summary
This is a research study for patients diagnosed with heavy menstrual bleeding (HMB) and low Von Willebrand Factor (VWF). Menstruation, also known as a period, is the regular discharge of blood and tissues from the uterus. HMB is having a heavier amount of discharge during menstrual period. Low Von Willebrand Factor means that the participant has lower level of a blood protein that is important for clotting of blood and so, the participant is at a higher risk for bleeding.
The purpose of this project is to study the genetic differences of adolescent females with HMB and low VWF activity and compare the genetic differences with their bleeding manifestations, response to medications and outcome.
- Detailed Description
One hundred and twenty subjects will be enrolled. Adolescent females with heavy menstrual bleeding (HMB) and low Von Willebrand Factor (VWF) will be recruited.
Data collection will occur from participant's medical records in regards to their low VWF activity and HMB medical history.
Participants will be asked to complete symptom questionnaires in regards to their HMB.
A blood sample will be collected to analyze how many participants have the disease causing sequence variation in the VWF gene and other genes affecting bleeding, clotting and blood vessel biology and correlated with their bleeding history.
The blood sample will be deidentified and stored indefinitely for future research.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- Female
- Target Recruitment
- 120
- Post-menarchal females less than 21 years of age
- HMB defined as PBAC score greater than 100
- VWF:Activity more than or equal to 30 and less than or equal to 50 IU/dL x 2
- VWF: Activity /VWF:Ag ratio greater than or equal to 0.6
- Normal VW multimers, if performed
- Post menarchal females age greater than or equal to 21 years
- VWF: Activity less than 30 or greater than 50 IU/dL consistently, type 2 or type 3 VWD
- Presence of other bleeding disorders (thrombocytopenia, platelet function defect, coagulation factor deficiency, fibrinogen defect or deficiency)
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Group A Complete Bleeding Symptom ISTH Bleeding Assessment Tool Adolescent women with heavy menstrual bleeding and low von willebrand factor activity. Group A Pictorial Blood Assessment Chart (PBAC) score Adolescent women with heavy menstrual bleeding and low von willebrand factor activity. Group A Medical Record Data Abstraction Adolescent women with heavy menstrual bleeding and low von willebrand factor activity. Group A Genetic Analysis Adolescent women with heavy menstrual bleeding and low von willebrand factor activity.
- Primary Outcome Measures
Name Time Method Number of adolescents with Low VWF and HMB with genetic variations in VWF gene and other genes affecting bleeding, clotting and blood vessel biology 3 years The genetic variations of adolescent females with heavy menstrual bleeding and low von Willebrand factor activity in VWF gene and other genes affecting bleeding, clotting and blood vessel biology
- Secondary Outcome Measures
Name Time Method Number of adolescents with Low VWF and HMB with genetic variations and bleeding phenotype (including PBAC score and ISTH-BAT score, response to DDAVP challenge, HMB therapy) 3 years The correlation of subjects with and without genetic variations with bleeding phenotype (including PBAC score, ISTH BAT score, response to DDAVP challenge and HMB therapy)
Trial Locations
- Locations (12)
Children's Hospital of Atlanta
🇺🇸Atlanta, Georgia, United States
Joseph M Sanzari Children's Hospital
🇺🇸Hackensack, New Jersey, United States
Michigan State University
🇺🇸East Lansing, Michigan, United States
University of Texas Southwestern Medical Center- Children's Medical Center
🇺🇸Dallas, Texas, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Mary M. Gooley Hemophilia Center
🇺🇸Rochester, New York, United States
Hemophilia Center of Western New York
🇺🇸Buffalo, New York, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
University of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States