120
Participants
Start Date
January 5, 2017
Primary Completion Date
June 30, 2020
Study Completion Date
November 30, 2025
Genetic Analysis
Blood sample will be drawn to assess the number of participants enrolled that have sequence variation in the von willebrand factor gene and other genes affecting bleeding, clotting and blood vessel biology.
Medical Record Data Abstraction
"The subject's response to intranasal or intravenous desmopressin (DDAVP) challenge performed as part of standard of care will be recorded. The desmopressin is a drug that increases the clotting factor in blood to prevent bleeding.~Medical and family history including and not limited to age, diagnoses, race/ethnicity, lab values, HMB and low VWF activity diagnoses, and treatment history and outcome will also be recorded."
Pictorial Blood Assessment Chart (PBAC) score
PBAC is a pictorial tool to assess menstrual blood loss. Study team will complete the PBAC assessment with each study participant evaluating and measure response to different treatments for menstrual blood loss in the clinic setting between those with VWD versus other bleeding disorders.
Complete Bleeding Symptom ISTH Bleeding Assessment Tool
Study participants will complete the ISTH BAT assessment. The ISTH BAT is a questionnaire to aid in the standardized evaluation of the presence and severity of bleeding symptoms.
Hemophilia Center of Western New York, Buffalo
Mary M. Gooley Hemophilia Center, Rochester
University of Pittsburgh, Pittsburgh
Children's Hospital of Atlanta, Atlanta
Vanderbilt University Medical Center, Nashville
Nationwide Children's Hospital, Columbus
Cincinnati Children's Hospital Medical Center, Cincinnati
Michigan State University, East Lansing
Children's Mercy Hospital, Kansas City
University of Texas Southwestern Medical Center- Children's Medical Center, Dallas
Texas Children's Hospital, Houston
Joseph M Sanzari Children's Hospital, Hackensack
Collaborators (1)
Shire
INDUSTRY
Baylor College of Medicine
OTHER