Clinical Trials Logo

Clinical Trial Summary

The objective of the study is to produce a dynamic picture of the adult ITP natural history and management in Italy by collecting standardized information retrospective and prospective data of patients in active management through a nation-wide registry.


Clinical Trial Description

Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder affecting both children and adults, characterized by a platelet count below 100 x 109/L in the absence of any underlying disease explaining the thrombocytopenia. Clinical manifestations may be absent or include cutaneous mucosal or organ bleeding, resulting in an overall mortality rate of less than 1-2%. Previously, 'acute ITP' was used to describe a self-limited form of the disease and 'chronic ITP' to describe the disease if it lasted for more than 6 months. In 2009, a new terminology for ITP was agreed upon based on the duration of the disease. The new terms for ITP are: 'newly diagnosed ITP (from diagnosis to 3 months), 'persistent ITP' (3-12 months) and 'chronic ITP' (lasting for more than 12 months) [2]. Chronic primary ITP in adults is slightly more common in women of childbearing age but the sex incidence is similar in patients over 60. The incidence of ITP in adults is around 4 per 100.000 people per year, with a UK prevalence of up to 24 per 100.000 people [4-6]. No more than 2 per 100.000 will require ITP treatment. Bleeding is highly variable and there is great heterogeneity. Bleeding is most commonly mucocutaneous, involving the skin and subcutaneous tissue (petechiae, ecchymoses, subcutaneous hematomas), the external mucosae (epistaxis, gum bleeding, blood blisters in the mouth), muscles (muscle hematomata) or the various organs and internal mucosae (hematuria, gastrointestinal bleeding, menorrhagia, intracerebral bleeding). Major bleeding is not common if the platelet count is above 30x109/L. Intracranial hemorrhage is rare and is most often seen in older patients who have additional comorbidities and in patients who fail to respond to therapy. The yearly risk of fatal hemorrhage is around 1.6-3.9% in unresponsive patients. This risk varies with age, from 0.4% per year in patients below 40 years old to 13% per year in those over 60. The natural history of ITP with currently available treatments is not well known. Available data are mostly based on the era when rituximab and thrombopoietin-receptor agonists (TPO-RA) were not in use. There is a lack reliable data on how recent treatments have modified the course of the disease. Bleeding frequency and severity, complications like infections, thrombosis, ITP-related symptoms like fatigue, etc. are also largely unknown as are women's related issues. Similarly, the cost of treatment for a patient with ITP is not available. Safety and efficacy of specific recent treatments have been measured mostly in industry-sponsored studies. Furthermore, most studies did not conform to the International Working Group (IWG) on ITP recommendations concerning terminology, definitions, outcome criteria and assessment of bleeding. To the investigator's knowledge, an ITP Registry is ongoing in UK and in Australia and registries are being planned in other European countries with a prospective of creating an international network. The only established international registry, the ITP PARC study, is operative since 2004 but covers different aspects of the disease. The few Italian registries are of regional dimension and are not aligned to collect standardized information in a uniform way. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03465020
Study type Observational [Patient Registry]
Source Gruppo Italiano Malattie EMatologiche dell'Adulto
Contact Paola Fazi
Phone +39 06.70390521
Email p.fazi@gimema.it
Status Recruiting
Phase
Start date October 15, 2018
Completion date July 2023

See also
  Status Clinical Trial Phase
Completed NCT05220878 - Comparative Study of Clinical Efficacy and Safety of GNR-069 and Nplate in Patients With ITP Phase 3
Completed NCT00451594 - High Dose Dexamethasone Vs. Conventional Dose Prednisolone in Adult ITP Phase 3
Withdrawn NCT01276561 - Single Incision Versus Standard Laparoscopic Splenectomy N/A
Completed NCT01713855 - Vaccine Response After Rituximab for Chronic, Severe Idiopathic Thrombocytopenic Purpura N/A
Completed NCT00603642 - P3 Study to Evaluate Efficacy and Safety of AMG 531 in Thrombocytopenic Japanese Subjects With Immune (Idiopathic) Thrombocytopenic Purpura Phase 3
Completed NCT01143038 - Interventional Study in Adults With Immune Thrombocytopenia Purpura (ITP) Receiving Romiplostim Phase 2
Not yet recruiting NCT04128358 - Triple Therapy in Patients With Idiopathic Thrombocytopenic Purpura : What is Behind? N/A
Completed NCT00128882 - Treatment of Idiopathic Thrombocytopenic Purpura (ITP) With Subcutaneously Administered Anti-D Phase 2
Completed NCT01525836 - rhTPO Combining Rituximab Versus Low-dose Rituximab in Management of ITP Phase 3
Completed NCT00888901 - Platelet Function in Idiopathic Thrombocytopenic Purpura (ITP) Patients With Eltrombopag Phase 4
Completed NCT00828750 - Clinical Evaluation of Eltrombopag in Chronic Idiopathic Thrombocytopenic Purpura (ITP) Phase 3
Completed NCT00475423 - A Study of MabThera (Rituximab) in Patients With Idiopathic Thrombocytopenic Purpura. Phase 2
Completed NCT00625443 - Phase 2, Parallel Group, Rollover Study of AKR-501 in Patients With ChronicITP Who Completed 28 Days of Study Treatment in Protocol 501-CL-003 Phase 2
Completed NCT00454857 - Retrospective & Prospective Observational Study of Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) N/A
Completed NCT00102323 - AMG 531 Treatment of Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Refractory to Splenectomy Phase 3
Completed NCT05492409 - Study of the Safety and Immunogenicity of Long-term GNR-069 Therapy in ITP Patients Phase 3
Terminated NCT01433978 - A Phase 3, Multicenter, Randomized, Double-blind,Active-controlled, Parallel-group Trial With an Open-labelExtension Phase to Evaluate the Efficacy and Safety of OralE5501 Versus Eltrombopag, in Adults With Chronic ImmuneThrombocytopenia (Idiopathic Thrombocytopenic Purpura) Phase 3
Withdrawn NCT01443351 - Long-term Safety Study of Treatment With the Thrombopoietin Agonists Eltrombopag and Romiplostim in Patients With Immune Thrombocytopenia (ITP)
Completed NCT01520909 - Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body. Phase 3
Not yet recruiting NCT06287567 - Lusutrombopag in the Treatment of Immune Thrombocytopenia (ITP) Phase 4