TTP - Thrombotic Thrombocytopenic Purpura
Conditions
Brief summary
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)
Interventions
DIAGNOSTIC_TESTFactor VIII (FVIII)
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTVon Willebrand factor (VWF) antigen
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTVWF:RCo
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTVWF-propeptide
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTVWF multimers
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTsP-selectin
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTsCD40L
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTF1+2
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTVEGF-A
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTVCAM
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTICAM
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTIL-1β
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTIL-6
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTPTX3
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTBlyS
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTNeutrophil elastase and myeloperoxidase (NETs) markers
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTsoluble C5b-9
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTC5a
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTNFL
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTGFAP
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTevaluation of thrombus formation with Total Thrombus formation Analysis System (T-TAS)
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTMicrofluidic Flow Chamber
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
GENETICWhole exome sequencing - WES
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
DIAGNOSTIC_TESTProteomic Analysis
The study is defined as interventional because it includes assessments that are not part of routine clinical practice
Sponsors
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Istituto Auxologico Italiano
Centro Cardiologico Monzino
Politecnico di Milano
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
DIAGNOSTIC
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* Patients who do not possess the above inclusion criteria * Patients who have known contraindications to the instrumental exam included in the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Prevalence | From enrollment to the last visit (after 36 months from Time 0) | Prevalence of microcirculation damage in iTTP patients compared to general population |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cerebral microcirculation lesions | From enrollment to the last visit (after 36 months from Time 0) | Evaluate cerebral microcirculation lesions (via brain MRI) in both acute and remission phases of TTP to monitor organ damage progression over time |
| Retinal microcirculation changes | From enrollment to the last visit (after 36 months from Time 0) | Assess retinal microcirculation changes (using OCT and OCT-A) during acute and remission phases of TTP to track organ damage evolution |
| Peripheral microcirculation | From enrollment to the last visit (after 36 months from Time 0) | Analyze peripheral microcirculation (with nailfold videocapillaroscopy) in acute and remission phases to evaluate progression of organ involvement in TTP |
| Association between cerebral MRI lesions and cognitive or psychological symptoms | From enrollment to the last visit (36 months from Time 0) | Explore the relationship between cerebral MRI lesions and cognitive or psychological symptoms during TTP remission, assessing symptom progression |
| Modification of preclinical biomarkers | From enrollment to the last visit (36 months from Time 0) | Explore the modification of preclinical biomarkers over time at different stage of the disease |
| Associations between preclinical biomarkers and microcirculatory damage | From enrollment to the last visit (36 months from Time 0) | Identify associations between preclinical biomarkers and microcirculatory damage in both acute and remission phases of TTP to predict risk and progression |
Countries
Italy
Outcome results
None listed