Clinical Spectrum and Management of Von Willebrand Disease Among Children in Assiut Governorate

Condition(s) studied

Investigational drug(s) / intervention(s)

Tranexamic Acid: Tranexamic acid is used as an antifibrinolytic agent for the management of mucocutaneous bleeding episodes in children with von Willebrand disease, according to standard clinical practice.

Von Willebrand Factor-Containing Concentrates: Plasma-derived von Willebrand factor/factor VIII concentrates are administered either on-demand during bleeding episodes or as regular prophylactic therapy in patients with recurrent or severe bleeding, based on clinical need.

Study summary

Von Willebrand disease (VWD) is the most common inherited bleeding disorder in children. It occurs due to a deficiency or dysfunction of von Willebrand factor, a protein that plays an essential role in blood clotting. Children with VWD may experience frequent nosebleeds, easy bruising, prolonged bleeding after injuries or surgeries, and, in adolescent girls, heavy menstrual bleeding. The severity of symptoms varies widely depending on the type of the disease and the level of the clotting factor.

Despite its clinical importance, data about the frequency, clinical presentation, and treatment outcomes of von Willebrand disease among children in Upper Egypt are limited. Early recognition and appropriate management are crucial to prevent complications, reduce hospital visits, and improve quality of life.

This observational study aims to assess the frequency of von Willebrand disease among children attending Assiut University Children's Hospital, describe the different disease subtypes, and evaluate the clinical bleeding patterns and management strategies used in routine practice. The study will include children aged 0-18 years with suspected or confirmed VWD.

Information will be collected from medical records and clinical evaluations, including bleeding symptoms, laboratory test results, disease classification, and treatment approaches. The results of this study are expected to improve understanding of von Willebrand disease in children in this region and support better diagnostic and therapeutic planning for affected patients.

Eligibility

Primary outcome measure(s)

• Frequency of Von Willebrand Disease Among Investigated Children — at enrollment
  The proportion of children diagnosed with von Willebrand disease among those evaluated for suspected bleeding disorders at Assiut University Children's Hospital.
• Distribution of Von Willebrand Disease Subtypes — Within 2 weeks of enrollment
  Classification and relative frequency of von Willebrand disease types (Type 1, Type 2, and Type 3) according to ISTH diagnostic criteria based on laboratory findings.
• Clinical Bleeding Patterns in Children With Von Willebrand Disease — at enrollment.
  Assessment of bleeding manifestations, including epistaxis, bruising, mucosal bleeding, postsurgical bleeding, and menorrhagia, using standardized clinical evaluation and bleeding assessment tools.

Trial sites (1)

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