Italian iTTP Registry (a Prospective Observational Study)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 132
- 试验地点
- 1
- 主要终点
- BMI
研究概览
简要总结
ItaliTTP is an observational, prospective, single-arm, national, multicenter, non-pharmacological cohort study aimed at better defining and understanding the natural history, disease severity, and clinical outcomes of patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP) in Italy.
A minimum of 132 consecutive patients with acute iTTP (first event or relapse) will be enrolled for 3 years, with the possibility of extension, with a follow-up period of 3 years.
详细描述
Acquired immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening thrombotic microangiopathy characterized by episodes of thrombocytopenia, microangiopathic hemolytic anemia, and extensive microvascular thrombosis leading to multiorgan involvement. Despite advances in understanding iTTP etiology and management in the acute phase, significant gaps in knowledge about its progression, particularly during clinical remission and concerning long-term complications, persist.
ItaliTTP, a national, multicenter, observational, prospective, non-pharmacological cohort study, aims to elucidate the natural history, severity, and outcomes of iTTP in Italy. The study will enroll hospitalized iTTP patients (experiencing either initial or recurrent episodes) and follow them in outpatient settings across participating Italian centers. The study plans to include at least 132 patients of any gender, aged 12 to 99, over a three-year period, with an option for extension, and a three-year follow-up. During hospitalization and subsequent outpatient visits, participants will undergo routine clinical assessments and laboratory tests. In addition to these data, peripheral blood samples will be collected for ADAMTS13 analysis and potential future research.
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
- 12 Years 至 99 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with an acute iTTP episode (first event or relapse), defined by thrombocytopenia and microangiopathic hemolytic anemia, in the absence of alternative causes, and the presence of severe deficiency of ADAMTS13 activity (< 10 IU/dL or <10% of normal value) and anti-ADAMTS13 autoantibodies
- •Both male and female patients, aged 12 years or older
- •Patients who have signed the informed consent for the participation to the study
排除标准
- •Patients who have not signed the informed consent for the participation to the study
结局指标
主要结局
BMI
时间窗: 3 years
Body mass index in kg/m\^2
Race
时间窗: 3 years
Proportion of acute iTTP patients treated with immunosuppressors other than steroids and rituximab
时间窗: 6 years
Time to ADAMTS13 remission
时间窗: 6 years
Blood group
时间窗: 3 years
ABO/Rh blood group
Proportion of patients with comorbidities, including: autoimmune diseases, cancer, HIV infection, hypertension, type 2 diabetes, hypercholesterolemia, cardiovascular disease, chronic renal failure, liver disease, depression.
时间窗: 3 years
Proportion of iTTP patients with comorbidities
Proportion of acute iTTP episodes preceded by potential triggering factors including: infections, pregnancy, surgery, psychological trauma, vaccination, drugs
时间窗: 3 years
Proportion of potential triggering conditions/events/drugs occured/taken in the 3 months prior the acute iTTP episode
Sex
时间窗: 3 years
Birth Country/Region
时间窗: 3 years
Age at onset
时间窗: 3 years
Age at the first acute iTTP episode in years
Incidence, type and severity of clinical manifestations, including: bleeding, cardiovascular, neurological, renal and systemic signs and symptoms
时间窗: 3 years
Incidence, type and severity of clinical manifestations at presentation of the acute iTTP episode
Hemoglobin lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
时间窗: 3 years
Hemoglobin level at presentation of the acute iTTP episode, expressed in g/dL
Cardiac troponin
时间窗: 3 years
Cardiac troponin level at presentation of the acute iTTP episode, expressed in ng/L
Platelet count lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
时间窗: 3 years
Platelet count at presentation of the acute iTTP episode, expressed in number x 10\^9/L
Number of daily therapeutic plasma exchange procedures
时间窗: 3 years
Number of daily therapeutic plasma exchange procedures to achieve clinical response of the acute iTTP episode
Proportion of iTTP patients treated with caplacizumab
时间窗: 3 years
Proportion of iTTP patients experiencing complications during hospitalization, including: bleeding, thrombosis, neurological, renal, cardiac complications
时间窗: 6 years
Proportion of patients who experience complications during the hospitalization for acute iTTP
Proportion of iTTP patients experiencing clinical exacerbation
时间窗: 6 years
Proportion of iTTP patients experiencing clinical exacerbation defined as sustained platelet count ≥ 150 × 109/L (or above the local lower limit of normal \[LLN\]) and LDH \< 1.5 times hte upper limit of normal (ULN) and no clinical evidence of new or progressive ischemic organ injury.
Proportion of iTTP patients achieving ADAMTS13 remission
时间窗: 6 years
Proportion of iTTP patients achieving ADAMTS13 remission defined as ADAMTS13 activity ≥ 20% to \< LLN (partial) or ADAMTS13 activity ≥ LLN (complete).
Lactate dehydrogenase (LDH) lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
时间窗: 3 years
LDH level at presentation of the acute iTTP episode, expressed in IU/L
ADAMTS13 activity
时间窗: 6 years
Level of functional ADAMTS13 activity expressed in IU/dL or %
Creatinine lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
时间窗: 3 years
Creatinine level at presentation of the acute iTTP episode, expressed in mg/dL
Anti-ADAMTS13 antibodies
时间窗: 6 years
Concentration or presence/absence of anti-ADAMTS13 antibodies
Proportion of acute iTTP patients treated with rituximab
时间窗: 6 years
Incidence, type and severity of TTP-related drugs adverse events
时间窗: 6 years
Incidence, type and severity of TTP-related drugs adverse events recorded during the acute iTTP episode and disease remission of iTTP patients
Proportion of iTTP patients refractory to acute iTTP treatment
时间窗: 6 years
Proportion of iTTP patients refractory to acute iTTP treatment. Refractoriness defined as persistent thrombocytopenia and a persistently raised LDH level despite treatment.
Proportion of iTTP patients achieving clinical remission
时间窗: 6 years
Proportion of iTTP patients achieving clinical remission defined as sustained clinical response with either no therapeutic plasma exchange (TPE) and no anti-von Willebrand factor (VWF) therapy for ≥ 30 days or with attainment of ADAMTS13 remission, whichever occurs first.
Time to clinical relapse
时间窗: 6 years
Incidence, type and severity of pregnancy complications in iTTP pregnant women
时间窗: 6 years
Time to clinical response
时间窗: 6 years
Proportion of iTTP patients with a clinical relapse
时间窗: 6 years
Proportion of iTTP patients with a clinical relapse defined as a platelet count decrease to \< 150 × 109/L (with other causes of thrombocytopenia ruled out), with or without clinical evidence of new ischemic organ injury, after a clinical remission.
Proportion of iTTP patients with an ADAMTS13 relapse
时间窗: 6 years
Proportion of iTTP patients with an ADAMTS13 relapse defined as a decrease of ADAMTS13 activity to \< 20% after a partial or complete ADAMTS13 remission.
Time to ADAMTS13 relapse
时间窗: 6 years
Time to clinical remission
时间窗: 6 years
次要结局
- iTTP incidence in Italy(3 years)