A Study of Factor Inhibitors in Adult Patients With Hemophilia and Von Willebrand's Disease in Upper Egypt

March 10, 2020 updated by: Safaa AA Khaled

Hemophilia A and B are bleeding disorders caused by deficiency of factor VIII and IX, respectively. The deficiency of one of these coagulation factors is due to a mutation on the X chromosome. Accordingly replacement of the deficient factor is currently the main treatment for these disorders. The most disappointing complication of replacement therapy in hemophilia is the development of inhibitors. Unlike haemophilia , inhibitor development in patients with V Willebrand's Disease (VWD) is a rare complication of treatment. Studies on inhibitors whether on hemophilia or VWD are limited in our region.

This study aims to

  1. To estimate the frequency of factor inhibitors in hemophilia and VWD patients in our region.
  2. To investigate modifiable risk factors associated with development of inhibitors in both diseases.
  3. To correlate the level of inhibitor with the clinical presentation of the patients.
  4. To assess influence of factor inhibitors on quality of life in patients who developed factor inhibitors in both diseases.

Study Overview

Detailed Description

Individuals with hemophilia are deficient in one of the clotting factor proteins that are vital in the formation of a clot. Classic hemophilia or hemophilia A is a deficiency of factor VIII, while Christmas Disease or Hemophilia B is a deficiency of factor IX. The prevalence of hemophilia A or B varies in different countries and geographic regions.

Patients with either type of hemophilia are at risk for prolonged bleeding, replacement of the deficient protein is the main therapy . The most serious complication of replacement therapy in hemophilia is the development of inhibitors.An inhibitor is a polyclonal high-affinity immunoglobulin G (IgG) that is directed against the clotting factorI protein. These antibodies can be either inhibitory or non inhibitory.

Inhibitors neutralize the administered clotting factor so that bleeding does not stop. Inhibitors are the most significant risk factor for morbidity and mortality associated with hemophilia, and patients with inhibitors present complex patient management challenges.

Few studies investigated development of factor inhibitor in Egyptian patients, however most of them concentrated on pediatric patients, also data regarding factor inhibitors in Upper Egypt was limited.

VonWillebrand's disease is a bleeding disorder caused by deficiency of VWF. The treatment of VWD is somewhat similar to that of patients with hemophilia which consists of infusions to replace the missing factors as on demand regimen using plasma derived (PD) products which contains both FVIII and VWF. Furthermore, many of the patients are currently on some form of prophylaxis to eliminate or decrease the frequency of bleeding episodes.

Nearly, 7.5 % of VWD patients develop inhibitors to VWF becoming non- responsive to replacement therapy, and prone to develop severe anaphylactic and life threatening reactions when exposed to any product that contains VWF.

Unlike hemophilia clinical presentation of VWD patients who developed inhibitors is not serious.

Again data on factor inhibitors in VWD is deficient in many countries worldwide particularly Egypt . Investigators assumed that this is the first study that well assess factor inhibitors in VWD in Upper Egypt.

Study Type

Observational

Enrollment (Anticipated)

100

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

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

18 years to 55 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with hemophilia A, B, and Von willebrand's disease who will be admitted at the clinical hematology unit ( Department of Internal Medicine), Assiut University Hospital over one year will be included in the study.

Description

Inclusion Criteria:

  • Patients diagnosed congenital hemophilia A, Christmas disease, and VWD

Exclusion Criteria

  • Patients diagnosed with acquired hemophilia
  • Patients below 18 years
  • Patients with other bleeding tendencies

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of patients with inhibitors
Time Frame: 4-days
Frequency of inhibitors among patients with hemophilia A and B, and VWD
4-days
Number of patients on demand replacement therapy
Time Frame: 7-days
Identification of the relationship between on demand replacement therapy and development of factor inhibitors in the study patients
7-days
Number of participants with low or high responding inhibitors
Time Frame: 7-days
This would be assessed by of the relationship between inhibitor level and severity of clinical presentation of the patient
7-days

Collaborators and Investigators

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

Sponsor

Investigators

  • Study Director: Howaida A. Nafady, Prof., Assiut University Hospial

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

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 (Anticipated)

May 10, 2020

Primary Completion (Anticipated)

November 10, 2020

Study Completion (Anticipated)

August 20, 2021

Study Registration Dates

First Submitted

September 19, 2019

First Submitted That Met QC Criteria

September 25, 2019

First Posted (Actual)

September 27, 2019

Study Record Updates

Last Update Posted (Actual)

March 12, 2020

Last Update Submitted That Met QC Criteria

March 10, 2020

Last Verified

October 1, 2019

More Information

Terms related to this study

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.

Clinical Trials on Hemophilia

3
Subscribe