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.
Age 0-18 years.
Residents of Assiut Governorate or receiving care at Assiut University Children's Hospital.
Suspected or confirmed von Willebrand disease (VWD) based on clinical bleeding symptoms or referral for evaluation.
VWF antigen (VWF:Ag).
Hemophilia A or B.
Rare coagulation factor deficiencies (e.g., factors I, V, VII, X, XI deficiency).
Platelet function disorders.
Liver disease.
This study includes pediatric patients aged 0 to 18 years with suspected or confirmed von Willebrand disease (VWD) who are residents of Assiut Governorate or are receiving medical care at Assiut University Children's Hospital. The study population represents children evaluated for bleeding symptoms or referred for assessment of possible inherited bleeding disorders within a tertiary pediatric healthcare setting. Eligible participants are identified based on clinical presentation suggestive of VWD, such as recurrent epistaxis, easy bruising, mucocutaneous bleeding, prolonged bleeding following trauma or surgical procedures, and heavy menstrual bleeding in adolescent females. Both newly evaluated patients and previously diagnosed cases with accessible medical records are included to allow comprehensive assessment of disease frequency, clinical spectrum, and management outcomes. All participants undergo standardized clinical evaluation, including detailed medical history, family history
Cohort
Other
Tranexamic Acid
DrugTranexamic 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
DrugPlasma-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.
Frequency of Von Willebrand Disease Among Investigated Children
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
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
Assessment of bleeding manifestations, including epistaxis, bruising, mucosal bleeding, postsurgical bleeding, and menorrhagia, using standardized clinical evaluation and bleeding assessment tools.
Use of Von Willebrand Factor-Containing Concentrates
Frequency of administration of VWF-containing concentrates during bleeding episodes or as prophylaxis.
Healthcare utilization
Number of emergency department visits or hospital admissions related to bleeding episodes during the follow-up period.
Adverse Events Related to Treatment
Occurrence of treatment-related adverse events, including allergic reactions or other reported complications.
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