STUDY OF MICROVASCULAR DAMAGE IN PATIENTS WITH THROMBOTIC THROMBOCYTOPENIC PURPURA
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 79
- 试验地点
- 1
研究概览
简要总结
The goal of this interventional, non-pharmacological clinical study is to assess microcirculatory dysfunction in patients with thrombotic thrombocytopenic purpura (TTP), both during the acute phase and in remission, compared with the general population.
Microcirculatory damage will be defined as the presence of at least one abnormal instrumental finding among brain magnetic resonance imaging (MRI), optical coherence tomography angiography (OCT-A), and nailfold videocapillaroscopy.
This is a monocentric, national study including two patient cohorts:
Cohort 1: patients enrolled during the acute phase Cohort 2: patients enrolled during remission Participants will be followed for three years with a total of five study visits.
Researchers will not test any drug but will perform detailed clinical, laboratory, and instrumental assessments to evaluate microvascular involvement over time.
Participants will:
- Undergo clinical evaluation, including internal medicine and neurological assessments
- Provide blood and urine samples for laboratory analyses
- Undergo instrumental examinations such as brain MRI, OCT-A, and nailfold videocapillaroscopy
- Receive psychological/neuropsychological evaluations
- Attend follow-up visits over a 3-year period (at baseline, 3 months [cohort 1 only], 12, 24, and 36 months)
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •aged ≥18 years
- •diagnosis of TTP (either congenital or acquired immune-mediated TTP)
- •referred at our hospital (Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, S.C. Medicina - Emostasi e Trombosi)
- •providing a signed informed consent
排除标准
- •Patients who do not possess the above inclusion criteria
- •Patients who have known contraindications to the instrumental exam included in the study.
研究组 & 干预措施
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: Factor VIII (FVIII) (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: Von Willebrand factor (VWF) antigen (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: VWF:RCo (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: VWF-propeptide (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: VWF multimers (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: ICAM (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: sP-selectin (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: sCD40L (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: F1+2 (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: VEGF-A (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: VCAM (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: ICAM (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: IL-1β (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: PTX3 (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: BlyS (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: Neutrophil elastase and myeloperoxidase (NETs) markers (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: soluble C5b-9 (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: C5a (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: NFL (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: GFAP (Diagnostic Test)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: Whole exome sequencing - WES (Genetic)
Acute TTP
Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
干预措施: Proteomic Analysis (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: Factor VIII (FVIII) (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: Von Willebrand factor (VWF) antigen (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: VWF:RCo (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: VWF-propeptide (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: VWF multimers (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: sP-selectin (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: sCD40L (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: F1+2 (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: VEGF-A (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: VCAM (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: IL-1β (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: PTX3 (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: Neutrophil elastase and myeloperoxidase (NETs) markers (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: soluble C5b-9 (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: C5a (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: NFL (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: GFAP (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: evaluation of thrombus formation with Total Thrombus formation Analysis System (T-TAS) (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: Microfluidic Flow Chamber (Diagnostic Test)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: Whole exome sequencing - WES (Genetic)
Remission TTP
Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode
干预措施: Proteomic Analysis (Diagnostic Test)
研究者
Flora Peyvandi
Director of S.C. Medicina Emostasi e Trombosi
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico