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.
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.
Study Type
OBSERVATIONAL
Enrollment
132
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, MI, Italy
RECRUITINGAge at onset
Age at the first acute iTTP episode in years
Time frame: 3 years
Sex
Time frame: 3 years
Birth Country/Region
Time frame: 3 years
Race
Time frame: 3 years
Blood group
ABO/Rh blood group
Time frame: 3 years
BMI
Body mass index in kg/m\^2
Time frame: 3 years
Proportion of patients with comorbidities, including: autoimmune diseases, cancer, HIV infection, hypertension, type 2 diabetes, hypercholesterolemia, cardiovascular disease, chronic renal failure, liver disease, depression.
Proportion of iTTP patients with comorbidities
Time frame: 3 years
Proportion of acute iTTP episodes preceded by potential triggering factors including: infections, pregnancy, surgery, psychological trauma, vaccination, drugs
Proportion of potential triggering conditions/events/drugs occured/taken in the 3 months prior the acute iTTP episode
Time frame: 3 years
Incidence, type and severity of clinical manifestations, including: bleeding, cardiovascular, neurological, renal and systemic signs and symptoms
Incidence, type and severity of clinical manifestations at presentation of the acute iTTP episode
Time frame: 3 years
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Platelet count lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
Platelet count at presentation of the acute iTTP episode, expressed in number x 10\^9/L
Time frame: 3 years
Hemoglobin lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
Hemoglobin level at presentation of the acute iTTP episode, expressed in g/dL
Time frame: 3 years
Lactate dehydrogenase (LDH) lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
LDH level at presentation of the acute iTTP episode, expressed in IU/L
Time frame: 3 years
Creatinine lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
Creatinine level at presentation of the acute iTTP episode, expressed in mg/dL
Time frame: 3 years
Cardiac troponin
Cardiac troponin level at presentation of the acute iTTP episode, expressed in ng/L
Time frame: 3 years
ADAMTS13 activity
Level of functional ADAMTS13 activity expressed in IU/dL or %
Time frame: 6 years
Anti-ADAMTS13 antibodies
Concentration or presence/absence of anti-ADAMTS13 antibodies
Time frame: 6 years
Number of daily therapeutic plasma exchange procedures
Number of daily therapeutic plasma exchange procedures to achieve clinical response of the acute iTTP episode
Time frame: 3 years
Proportion of acute iTTP patients treated with rituximab
Time frame: 6 years
Proportion of acute iTTP patients treated with immunosuppressors other than steroids and rituximab
Time frame: 6 years
Proportion of iTTP patients treated with caplacizumab
Time frame: 3 years
Incidence, type and severity of TTP-related drugs adverse events
Incidence, type and severity of TTP-related drugs adverse events recorded during the acute iTTP episode and disease remission of iTTP patients
Time frame: 6 years
Proportion of iTTP patients achieving clinical remission
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 frame: 6 years
Proportion of iTTP patients refractory to acute iTTP treatment
Proportion of iTTP patients refractory to acute iTTP treatment. Refractoriness defined as persistent thrombocytopenia and a persistently raised LDH level despite treatment.
Time frame: 6 years
Proportion of iTTP patients experiencing complications during hospitalization, including: bleeding, thrombosis, neurological, renal, cardiac complications
Proportion of patients who experience complications during the hospitalization for acute iTTP
Time frame: 6 years
Proportion of iTTP patients experiencing clinical exacerbation
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.
Time frame: 6 years
Proportion of iTTP patients achieving ADAMTS13 remission
Proportion of iTTP patients achieving ADAMTS13 remission defined as ADAMTS13 activity ≥ 20% to \< LLN (partial) or ADAMTS13 activity ≥ LLN (complete).
Time frame: 6 years
Time to clinical response
Time frame: 6 years
Time to clinical remission
Time frame: 6 years
Time to ADAMTS13 remission
Time frame: 6 years
Proportion of iTTP patients with a clinical relapse
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.
Time frame: 6 years
Proportion of iTTP patients with an ADAMTS13 relapse
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 frame: 6 years
Time to clinical relapse
Time frame: 6 years
Time to ADAMTS13 relapse
Time frame: 6 years
Incidence, type and severity of pregnancy complications in iTTP pregnant women
Time frame: 6 years
iTTP incidence in Italy
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).
Time frame: 3 years