Combination Therapy of Low Doses of rFVIIa and FEIBA for Severe Hemophilia A Patients With an Inhibitor to Factor VIII
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 5
- 主要终点
- Hemostasis achieved post therapy
研究概览
简要总结
Patients with severe hemophilia and inhibitors can be treated effectively by Activated Prothrombin Complex Concentrates (APCC, eg. FEIBA) or High dose recombinant factor VIIa (rFVIIa). Rarely, such patients develop refractoriness to these products for whom therapy with sequential FEIBA and rFVIIa has been recently suggested.
The impetus for the present report was a hemophilia A patient with high titer inhibitor (1300BU) who had life threatening hematuria that was resistant to repeated doses of 400µg/kg rFVIIa up to a cumulative dose of 1200 µg/kg given over 6-9 hours.
Thrombin generation (TG) tested in vitro was consistent with resistance to high concentrations of rFVIIa but yielded good response to combinations of low doses of rFVIIa+FEIBA. In a desperate attempt to control the bleeding, concomitant therapy of 25 U/kg FEIBA and 40µg/kg rFVIIa was infused and resulted in arrest of bleeding within minutes. Over a span of about one year the patient has been successfully treated by this combination for more than 200 bleeding episodes in muscles and joints.
详细描述
Inhibitor patients with HR inhibitors were eligible for study enrollment. After consent blood was drawn and ex- vivo spiked with rFVIIa/FEIBA and combinations, assayed by thrombin generation tests.
The combination yielding sufficient hemostasis was depicted for patients' therapy of future bleeding episodes.
Following actual therapy hemostasis and safety parameters were monitored.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Hemophilia patients with inhibitors
- •Patients signing informed consent
排除标准
- •Patients under 16 or above 65
- •Patients with allergic reaction or adverse events in previous use the concentrates
- •Patients with high risk of thrombosis
研究组 & 干预措施
feiba-VIIa, hemophilia A-inhibitor therapy
COMBINED PATIENT- TAILORED THERAPY WITH CONCOMITANT ADMINISTRATION OF BOTH DRUGS , FOLLOWING EX VIVO THROMBIN GENERATION PREDICTING ASSAYS
干预措施: rFVIIa-FEIBA therapy for hemophilia A inhibitors (Drug)
feiba-VIIa, hemophilia A-inhibitor therapy
COMBINED PATIENT- TAILORED THERAPY WITH CONCOMITANT ADMINISTRATION OF BOTH DRUGS , FOLLOWING EX VIVO THROMBIN GENERATION PREDICTING ASSAYS
干预措施: FEIBA- Activated Prothrombin Complexes (Drug)
结局指标
主要结局
Hemostasis achieved post therapy
时间窗: 6-24 hours
Following acute bleeding therapy hemostasis was defined as good, partial or non-satisfactory
Safety
时间窗: 0-24 HOURS
Following therapy presence of any adverse events, especially thromboembolic complications was assessed
次要结局
- Time to Hemostasis(0-24 HOURS)
研究者
Prof. Uriel Martinowitz
Prof. Uri Martinowitz
Sheba Medical Center