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

NCT07410130 | Not Yet Recruiting

• 1 other identifier: AUN-PED-VWD-2026-01
• Study Type: observational
• Target: 25
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Von Willebrand Disease (VWD); Inherited Bleeding Disorders in Children; Pediatric Hemostatic Disorders

Keywords

Von Willebrand Disease; VWD; Pediatric bleeding disorders; Inherited coagulation disorders; Von willebrand factor; Mucocutaneous bleeding; Epistaxis in children; Menorrhagia; Bleeding assessment tool; VWD antigen; VWD activity; Hemostasis; Pediatric hematology; Factor VIII; Observational study

Outcome Measures

Primary Outcomes (3)

• 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.

  at enrollment

• 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.

  Within 2 weeks of enrollment

• 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.

  at enrollment.

Other Outcomes (3)

• Use of Von Willebrand Factor-Containing Concentrates

  From enrollment up to 6 months of follow-up

• Healthcare utilization

  Up to 6 months following enrollment

• Adverse Events Related to Treatment

  From enrollment up to 6 months of follow-up

Study Arms (2)

On-Demand Therapy Group

This cohort includes children with confirmed von Willebrand disease who receive treatment only during active bleeding episodes or prior to invasive procedures. Management is based on clinical indication and routine care practices, without scheduled prophylactic therapy. Bleeding frequency, treatment response, and short-term outcomes are documented during follow-up.

Drug: Tranexamic Acid; Drug: Von Willebrand Factor-Containing Concentrates

Prophylaxis Therapy Group

This cohort includes children with von Willebrand disease who experience recurrent, severe, or clinically significant bleeding and therefore receive regular prophylactic treatment with von Willebrand factor-containing concentrates. Patients are followed prospectively to assess bleeding frequency, treatment effectiveness, and clinical outcomes under scheduled preventive therapy.

Drug: Tranexamic Acid; Drug: Von Willebrand Factor-Containing Concentrates

Interventions

Tranexamic Acid DRUG

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.

On-Demand Therapy Group; Prophylaxis Therapy Group

Von Willebrand Factor-Containing Concentrates DRUG

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.

On-Demand Therapy Group; Prophylaxis Therapy Group

Eligibility Criteria

• Age: 0 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.
• Patients diagnosed with VWD using standard laboratory tests, including:
• VWF antigen (VWF:Ag).
• VWF ristocetin cofactor activity (VWF:RCo).
• Factor VIII activity.

You may not qualify if:

• Other inherited bleeding disorders, such as:
• Hemophilia A or B.
• Rare coagulation factor deficiencies (e.g., factors I, V, VII, X, XI deficiency).
• Platelet function disorders.
• Acquired bleeding disorders, including:
• Liver disease.
• Renal insufficiency.
• Vitamin K deficiency.
• Disseminated intravascular coagulation (DIC).
• Use of medications that may interfere with coagulation testing (e.g., anticoagulants, antiplatelet drugs).
• Incomplete clinical or laboratory data (for retrospective cases).
• Refusal of consent for participation (for prospective cases).

Sponsors & Collaborators

• Assiut University: lead

Study Sites (1)

Assiut university

Asyut, Egypt

Location

Related Publications (8)

• Ahmed EH, et al. Screening of Von Willebrand Disease Among Children with Severe or Recurrent Epistaxis. Egyptian Journal, 2025.

  BACKGROUND

• Brauchli YB, Wais T, Gratwohl A, Heim D, Schipf A, Diebold J, Krahenbuhl S. Fatal myocardial infarction during nilotinib treatment in a 60-year-old male patient. Acta Oncol. 2010 May;49(4):523-5. doi: 10.3109/02841861003691952. No abstract available.

  PMID: 20307244 BACKGROUND

• Jaffe JJ, Chrin LR. Thymidylate synthetase activity in normal and brugia pahangi-infected aedes aegypti. Biochem Pharmacol. 1979 Jun 15;28(12):1831-5. doi: 10.1016/0006-2952(79)90633-6. No abstract available.

  PMID: 454454 BACKGROUND

• Sharma R, Haberichter SL. New advances in the diagnosis of von Willebrand disease. Hematology Am Soc Hematol Educ Program. 2019 Dec 6;2019(1):596-600. doi: 10.1182/hematology.2019000064.

  PMID: 31808831 BACKGROUND

• Shui M, D'Angelo L, Croteau SE. Low von Willebrand factor in pediatric patients: Retrospective analysis of 293 cases informs diagnostic and therapeutic decision making. Pediatr Blood Cancer. 2020 Sep;67(9):e28497. doi: 10.1002/pbc.28497. Epub 2020 Jun 23.

  PMID: 32573918 BACKGROUND

• Abe K, Dupervil B, O'Brien SH, Oakley M, Kulkarni R, Gill JC, Byams V, Soucie MJ; US Hemophilia Treatment Center Network. Higher rates of bleeding and use of treatment products among young boys compared to girls with von Willebrand disease. Am J Hematol. 2020 Jan;95(1):10-17. doi: 10.1002/ajh.25656. Epub 2019 Oct 29.

  PMID: 31612544 BACKGROUND

• Sanders YV, Fijnvandraat K, Boender J, Mauser-Bunschoten EP, van der Bom JG, de Meris J, Smiers FJ, Granzen B, Brons P, Tamminga RY, Cnossen MH, Leebeek FW; WiN Study Group. Bleeding spectrum in children with moderate or severe von Willebrand disease: Relevance of pediatric-specific bleeding. Am J Hematol. 2015 Dec;90(12):1142-8. doi: 10.1002/ajh.24195. Epub 2015 Nov 17.

  PMID: 26375306 BACKGROUND

• Halimeh S, Krumpel A, Rott H, Bogdanova N, Budde U, Manner D, Faeser B, Mesters R, Nowak-Gottl U. Long-term secondary prophylaxis in children, adolescents and young adults with von Willebrand disease. Results of a cohort study. Thromb Haemost. 2011 Apr;105(4):597-604. doi: 10.1160/TH10-09-0616. Epub 2011 Feb 8.

  PMID: 21301780 BACKGROUND

Related Links

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MeSH Terms

Conditions

von Willebrand Diseases; Blood Coagulation Disorders, Inherited; Menorrhagia; Hemophilia A

Interventions

Tranexamic Acid

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Uterine Hemorrhage; Uterine Diseases; Genital Diseases, Female; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Genital Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Menstruation Disturbances

Intervention Hierarchy (Ancestors)

Cyclohexanecarboxylic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals

Central Study Contacts

Ali Maher Maher

CONTACT

01141914839; Ali.15235776@med.aun.edu.eg

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: resident at pediatric department

Study Record Dates

• First Submitted: January 29, 2026
• First Posted: February 13, 2026
• Study Start: March 1, 2026
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: February 13, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

EG (1)