NCT04106323

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

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 100 (estimated)
• Sponsor: Safaa AA Khaled · Academic / Other
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

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.

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.

Conditions

• Hemophilia
• Von Willebrand Diseases
• Christmas Disease

Timeline

• Start date: 2020-05-10
• Primary completion: 2020-11-10
• Completion: 2021-08-20
• First posted: 2019-09-27
• Last updated: 2020-03-12