A Study of Factor Inhibitors in Adult Patients With Hemophilia and Von Willebrand's Disease in Upper Egypt
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 100
- 主要终点
- Number of participants with low or high responding inhibitors
研究概览
简要总结
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
- To estimate the frequency of factor inhibitors in hemophilia and VWD patients in our region.
- To investigate modifiable risk factors associated with development of inhibitors in both diseases.
- To correlate the level of inhibitor with the clinical presentation of the patients.
- To assess influence of factor inhibitors on quality of life in patients who developed factor inhibitors in both diseases.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- 未提供
结局指标
主要结局
Number of participants with low or high responding inhibitors
时间窗: 7-days
This would be assessed by of the relationship between inhibitor level and severity of clinical presentation of the patient
Number of patients with inhibitors
时间窗: 4-days
Frequency of inhibitors among patients with hemophilia A and B, and VWD
Number of patients on demand replacement therapy
时间窗: 7-days
Identification of the relationship between on demand replacement therapy and development of factor inhibitors in the study patients
次要结局
未报告次要终点
研究者
Safaa AA Khaled
Clinical Professor
Assiut University