Italian iTTP Registry

Condition(s) studied

Study 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.

Eligibility

Primary outcome measure(s)

• Age at onset — 3 years
  Age at the first acute iTTP episode in years
• Sex — 3 years
• Birth Country/Region — 3 years
• Race — 3 years
• Blood group — 3 years
  ABO/Rh blood group
• BMI — 3 years
  Body mass index in kg/m\^2
• 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
• 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
• 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
• 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
• 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
• 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
• Cardiac troponin — 3 years
  Cardiac troponin level at presentation of the acute iTTP episode, expressed in ng/L
• ADAMTS13 activity — 6 years
  Level of functional ADAMTS13 activity expressed in IU/dL or %
• Anti-ADAMTS13 antibodies — 6 years
  Concentration or presence/absence of anti-ADAMTS13 antibodies
• 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 acute iTTP patients treated with rituximab — 6 years
• Proportion of acute iTTP patients treated with immunosuppressors other than steroids and rituximab — 6 years
• Proportion of iTTP patients treated with caplacizumab — 3 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 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.
• 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 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).
• Time to clinical response — 6 years
• Time to clinical remission — 6 years
• Time to ADAMTS13 remission — 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 clinical relapse — 6 years
• Time to ADAMTS13 relapse — 6 years
• Incidence, type and severity of pregnancy complications in iTTP pregnant women — 6 years

Trial sites (1)

Other trials for the same condition