Clinical Spectrum and Management of Von Willebrand Disease Among Children in Assiut Governorate (VWD-ASSIUT)

February 7, 2026 updated by: Ali Maher Ebied Maher, Assiut University

Clinical Spectrum of Von Willebrand Disease Among Children: Frequency, Management, and Outcomes in Assiut Governorate

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

Von Willebrand disease (VWD) is the most common inherited bleeding disorder worldwide and represents a significant cause of mucocutaneous bleeding in children. It results from quantitative or qualitative defects of von Willebrand factor (VWF), a glycoprotein that plays a key role in platelet adhesion and stabilization of factor VIII. VWD is classified into three main types according to International Society on Thrombosis and Hemostasis (ISTH) criteria: type 1 (partial quantitative deficiency), type 2 (qualitative defects with several subtypes), and type 3 (severe quantitative deficiency).

The clinical presentation of VWD in children is highly variable and may include epistaxis, easy bruising, gingival bleeding, prolonged bleeding after minor trauma or surgery, and menorrhagia in adolescent females. The severity and frequency of bleeding episodes are influenced by disease subtype, VWF levels, age, and associated conditions. In pediatric populations, diagnosis may be delayed or missed due to mild symptoms, age-related physiological variations in VWF levels, and limited awareness.

This study is designed as a descriptive retrospective-prospective observational study conducted at Assiut University Children's Hospital. The target population includes children aged 0-18 years with suspected or confirmed von Willebrand disease who are residents of Assiut Governorate or receive care at the study center. Patients with other inherited or acquired bleeding disorders will be excluded.

During the retrospective phase, medical records of previously diagnosed VWD patients will be reviewed to collect demographic data, family history, clinical presentation, laboratory findings, disease subtype, treatment received, and documented outcomes. In the prospective phase, children presenting with bleeding symptoms suggestive of VWD will undergo standardized clinical assessment, including a structured bleeding questionnaire and physical examination, followed by laboratory evaluation.

Laboratory investigations will include complete blood count, coagulation profile, VWF antigen level, VWF activity, and factor VIII activity, with additional specialized testing when available. Disease classification will be performed according to ISTH guidelines.

Management data will be collected to describe current treatment practices, including on-demand therapy during bleeding episodes and prophylactic therapy for patients with recurrent or severe bleeding. Treatment response, frequency of bleeding episodes, need for hospital visits, and occurrence of adverse events will be monitored during follow-up.

The primary outcomes of the study include the frequency of von Willebrand disease among the investigated pediatric population, distribution of disease subtypes, and characterization of clinical bleeding patterns. Secondary outcomes include assessment of treatment modalities, recurrence of bleeding episodes, and short-term clinical outcomes.

By providing comprehensive data on the clinical spectrum and management of VWD among children in Assiut Governorate, this study aims to enhance early diagnosis, guide evidence-based management strategies, and improve overall patient care in this setting.

Study Type

Observational

Enrollment (Estimated)

25

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Egypt
      • Asyut, Egypt
        • Assiut University
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

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

Description

Inclusion Criteria:

  1. Age 0-18 years.
  2. Residents of Assiut Governorate or receiving care at Assiut University Children's Hospital.
  3. Suspected or confirmed von Willebrand disease (VWD) based on clinical bleeding symptoms or referral for evaluation.
  4. Patients diagnosed with VWD using standard laboratory tests, including:
  5. VWF antigen (VWF:Ag).
  6. VWF ristocetin cofactor activity (VWF:RCo).
  7. Factor VIII activity.

Exclusion Criteria:

  1. Other inherited bleeding disorders, such as:
  2. Hemophilia A or B.
  3. Rare coagulation factor deficiencies (e.g., factors I, V, VII, X, XI deficiency).
  4. Platelet function disorders.

  5. 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).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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
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.
Drug: 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.
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
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.
Drug: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frequency of Von Willebrand Disease Among Investigated Children
Time Frame: at enrollment
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
Time Frame: 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.
Within 2 weeks of enrollment
Clinical Bleeding Patterns in Children With Von Willebrand Disease
Time Frame: at enrollment.
Assessment of bleeding manifestations, including epistaxis, bruising, mucosal bleeding, postsurgical bleeding, and menorrhagia, using standardized clinical evaluation and bleeding assessment tools.
at enrollment.

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Use of Von Willebrand Factor-Containing Concentrates
Time Frame: From enrollment up to 6 months of follow-up
Frequency of administration of VWF-containing concentrates during bleeding episodes or as prophylaxis.
From enrollment up to 6 months of follow-up
Healthcare utilization
Time Frame: Up to 6 months following enrollment
Number of emergency department visits or hospital admissions related to bleeding episodes during the follow-up period.
Up to 6 months following enrollment
Adverse Events Related to Treatment
Time Frame: From enrollment up to 6 months of follow-up
Occurrence of treatment-related adverse events, including allergic reactions or other reported complications.
From enrollment up to 6 months of follow-up

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assiut University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

March 1, 2026

Primary Completion (Estimated)

September 1, 2026

Study Completion (Estimated)

December 1, 2026

Study Registration Dates

First Submitted

January 29, 2026

First Submitted That Met QC Criteria

February 7, 2026

First Posted (Actual)

February 13, 2026

Study Record Updates

Last Update Posted (Actual)

February 13, 2026

Last Update Submitted That Met QC Criteria

February 7, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AUN-PED-VWD-2026-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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