Italian iTTP Registry
- Conditions
- TTP - Thrombotic Thrombocytopenic Purpura
- Registration Number
- NCT06376786
- Lead Sponsor
- Fondazione Luigi Villa
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 132
- 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
- Patients who have not signed the informed consent for the participation to the study
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method BMI 3 years Body mass index in kg/m\^2
Race 3 years Proportion of acute iTTP patients treated with immunosuppressors other than steroids and rituximab 6 years Time to ADAMTS13 remission 6 years Blood group 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. 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 3 years Proportion of potential triggering conditions/events/drugs occured/taken in the 3 months prior the acute iTTP episode
Sex 3 years Birth Country/Region 3 years Age at onset 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 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 3 years Hemoglobin level at presentation of the acute iTTP episode, expressed in g/dL
Cardiac troponin 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 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 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 3 years Proportion of iTTP patients experiencing complications during hospitalization, including: bleeding, thrombosis, neurological, renal, cardiac complications 6 years Proportion of patients who experience complications during the hospitalization for acute iTTP
Proportion of iTTP patients experiencing clinical exacerbation 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 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 3 years LDH level at presentation of the acute iTTP episode, expressed in IU/L
ADAMTS13 activity 6 years Level of functional ADAMTS13 activity expressed in IU/dL or %
Creatinine lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin 3 years Creatinine level at presentation of the acute iTTP episode, expressed in mg/dL
Anti-ADAMTS13 antibodies 6 years Concentration or presence/absence of anti-ADAMTS13 antibodies
Proportion of acute iTTP patients treated with rituximab 6 years Incidence, type and severity of TTP-related drugs adverse events 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 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 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 6 years Incidence, type and severity of pregnancy complications in iTTP pregnant women 6 years Time to clinical response 6 years Proportion of iTTP patients with a clinical relapse 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 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 6 years Time to clinical remission 6 years
- Secondary Outcome Measures
Name Time Method iTTP incidence in Italy 3 years The number of all TTP events (first events and relapses) and first TTP events will be divided by the number of people at risk multiplied by the observation time to estimate the incidence rate of iTTP events and iTTP incident cases, respectively (in persons-years).
Trial Locations
- Locations (1)
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
🇮🇹Milan, MI, Italy