Development Of Spanish-Portuguese Thrombotic Thrombocytopenic Purpura Registry (REPTT): A Study Proposal Of The Spanish Society Of Hematology And Hemotherapy (SEHH) Whit The Portuguese Society Of Hematology (SPH)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 300
- 试验地点
- 7
- 主要终点
- Morbidity of TTP in Spain/Portugal
研究概览
简要总结
REPTT is an observational, prospective, multi-country, multicentre and non-interventional registry in which at least 300 patients with Thrombotic thrombocytopenic purpura (TTP) in Spain and Portugal will be evaluated.
The study will be carried out in the context of the usual clinical practice conditions, not imposing restrictions on the participating physician or influencing their normal clinical practice.
详细描述
TTP is a rare life-threatening haematological disease characterised by thrombotic microangiopathy (TMA) with an average annual prevalence of approximately 10 cases/million people worldwide and an annual incidence between 1.5 and 6.0 cases per million according to different studies conducted in France, the United States and in the United Kingdom. In Spain the incidence is 2,67 cases / million population per year.
Acute TTP episodes cause sequelae like vascular disease or kidney damage along with other symptoms more subtle like small neurocognitive deficits and myocardial infarction.
Thus, prompt resolution of acute episodes along with a better understanding of the cardiac abnormalities may allow to prevent further complications, to develop targeted rehabilitation techniques for TTP patients and to improve their quality of life.
This project will collect a big database capable of providing better answers to questions related with treatment efficacy, associated morbidity and mortality, and the possible neurocognitive and cardiac sequelae derived from relapses and acute episodes. Additionally, this project will be linked to obtaining biological samples for a serum and DNA library from patients with TTP.
Patients will be recruited by medical researchers specialized in haematology or by other investigators specialized in thrombotic microangiopathies disease management. This recruitment will be performed in a competitive manner. The collection period will be at least 3 years with the possibility of extending it.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 99 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with diagnosis of TTP according to International Consensus criteria from centres in Spain and Portugal.
- •Patients that voluntarily sign the informed consent. For subjects unable to provide informed consent, a fully recognized medical authority may be used according to local laws.
- •Patients between 0 to 99 years old at the time of diagnosis.
- •Note: Decision was taken to treat the patient with an specific treatment prior and independently of patient inclusion in this non interventional study.
排除标准
- •Inability to comply with study procedures and follow-up exams.
- •Patients with any type of alteration that compromises their ability to grant written informed consent.
- •Patients that do not consent to participate in the study and do not sign informed consent.
- •Patients that do not meet the criteria previously mentioned for TTP.
结局指标
主要结局
Morbidity of TTP in Spain/Portugal
时间窗: Throughout the study period, calculated for the 3 years of expected duration.
Percentage of patients diagnosed with TTP and enrolled in the registry among spanish / portuguese population per year
Incidence of TTP in Spain/Portugal
时间窗: Throughout the study period, calculated for the 3 years of expected duration.
Number of patients diagnosed with TTP and enrolled in the registry per year
Mortality of TTP in Spain/Portugal
时间窗: Throughout the study period, calculated for the 3 years of expected duration.
Percentage of patients diagnosed with TTP and enrolled in the registry per year who died due to the disease
次要结局
- TTP Clinical remission rate(Throughout the study period, approximately 3 years per patient)
- TTP Exacerbation rate(Throughout the study period, approximately 3 years per patient)
- Frequency of serious adverse events (SAEs)(Throughout the study period, approximately 3 years per patient)
- Rate of complications associated with plasma exchange treatment(Throughout the study period, approximately 3 years per patient)
- Overall survival (OS)(Throughout the study period, approximately 3 years per patient)
- Clinical response to treatment rate(Throughout the study period, approximately 3 years per patient)
- Refractory TTP rate(Throughout the study period, approximately 3 years per patient)
- Duration of response (DoR)(Throughout the study period, approximately 3 years per patient)
- TTP Relapse rate(Throughout the study period, approximately 3 years per patient)
- Relapse-free survival (RFS)(Throughout the study period, approximately 3 years per patient)
- Frequency of complications associated with plasma exchange treatment(Throughout the study period, approximately 3 years per patient)
- Time-to-response (TTR)(Throughout the study period, approximately 3 years per patient)