Investigation on a Dynamic Cohort of Italian Patients With Active ITP

NCT ID: NCT03465020

Last Updated: 2022-07-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

861 participants

Study Classification

OBSERVATIONAL

Study Start Date

2018-10-15

Study Completion Date

2023-07-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Idiopathic Thrombocytopenic Purpura

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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ITP patients

On active treatment

Observation

Intervention Type OTHER

Safety and efficacy outcomes of patients on different treatments

Interventions

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Observation

Safety and efficacy outcomes of patients on different treatments

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Patients never treated before for whom an ITP treatment is initiated for the first time by the recruiting center
* Patients already treated for whom a new line of ITP treatment is initiated by the recruiting center (the patients could have been treated elsewhere before)
* Patients who, under any ITP treatment at the recruiting center.

Exclusion Criteria

-Undefined
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Fondazione Progetto Ematologia

OTHER

Sponsor Role collaborator

Gruppo Italiano Malattie EMatologiche dell'Adulto

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Francesco Rodeghiero

Role: STUDY_CHAIR

Department of Hematology, S. Bortolo Hospital of Vicenza

Marco Ruggeri

Role: STUDY_DIRECTOR

Department of Hematology, S. Bortolo Hospital, Vicenza

Locations

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Sc Ematologia E Centro Trapianti Midollo Osseo

Parma, SC, Italy

Site Status RECRUITING

Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica

Ancona, , Italy

Site Status RECRUITING

Aou Consorziale Policlinico - Bari - Uo Ematologia Con Trapianto

Bari, , Italy

Site Status RECRUITING

Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia

Bologna, , Italy

Site Status RECRUITING

Ao Brotzu, Presidio Ospedaliero A. Businco - Cagliari - Sc Ematologia E Ctmo

Cagliari, , Italy

Site Status RECRUITING

Ctc U.O Di Ematologia Con Trapianto Di Midollo Osseo - Catania

Capranica, , Italy

Site Status RECRUITING

Arnas Garibaldi, Po Garibaldi-Nesima - Catania - Uoc Ematologia

Catania, , Italy

Site Status RECRUITING

Aou Careggi - Firenze - Sod Ematologia

Florence, , Italy

Site Status RECRUITING

Asst Grande Ospedale Metropolitano Niguarda - Milano - Sc Ematologia

Milan, , Italy

Site Status RECRUITING

Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora

Milan, , Italy

Site Status RECRUITING

Asst Di Monza, Ospedale S. Eugenio - Uo Ematologia E Cta

Monza, , Italy

Site Status RECRUITING

Ao Di Rilievo Nazionale Antonio Cardarelli - Napoli - Uoc Ematologia Con Trapianto Di Midollo

Napoli, , Italy

Site Status RECRUITING

Aou Federico Ii - Napoli - Uoc Ematologia

Napoli, , Italy

Site Status RECRUITING

Aou Maggiore Della Carita' Di Novara - Scdu Ematologia

Novara, , Italy

Site Status RECRUITING

Aou Policlinico P. Giaccone - Palermo - Uo Ematologia

Palermo, , Italy

Site Status RECRUITING

Ospedale S.M. della Misericordia - STROKE UNIT

Perugia, , Italy

Site Status RECRUITING

Asl Di Piacenza, Ospedale "Guglielmo Da Saliceto" - Ematologia E Centro Trapianti

Piacenza, , Italy

Site Status RECRUITING

Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia

Reggio Emilia, , Italy

Site Status RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli Irccs - Roma - Area Ematologica

Roma, , Italy

Site Status RECRUITING

Ente Ecclesiastico Casa Sollievo Della Sofferenza - San Giovanni Rotondo - Ematologia

San Giovanni Rotondo, , Italy

Site Status RECRUITING

Asl Taranto, Ospedale Ss. Annunziata - Uoc Ematologia

Taranto, , Italy

Site Status RECRUITING

Aou Città Della Salute E Della Scienza, Ospedale S. Giovanni Battista Molinette - Torino - Sc Ematologia - Università Degli Studi Di Torino

Torino, , Italy

Site Status RECRUITING

Ente Ecclesiastico Cardinale G. Panico - Tricase - Uo Ematologia

Tricase, , Italy

Site Status RECRUITING

Azienda Sanitaria Universitaria Integrata Di Trieste - Sc Ematologia

Trieste, , Italy

Site Status RECRUITING

Asui Di Udine - Presidio Ospedaliero "Santa Maria Della Misericordia" - Clinica Ematologica

Udine, , Italy

Site Status RECRUITING

Aulss 8 Berica - Ospedale Di Vicenza - Uoc Ematologia

Vicenza, , Italy

Site Status RECRUITING

Countries

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Italy

Central Contacts

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Paola Fazi

Role: CONTACT

+39 06.70390521

Enrico Crea

Role: CONTACT

+39 0670390514

Facility Contacts

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Crugnola

Role: primary

Antonella Poloni

Role: primary

Alessandra Ricco

Role: primary

Nicola Vianelli

Role: primary

Maria Pina Simula

Role: primary

Francesco Di Raimondo

Role: primary

Ugo Consoli

Role: primary

Valentina Carrai

Role: primary

Silvia Cantoni

Role: primary

Wilma Barcellini

Role: primary

Monica Carpenedo

Role: primary

Felicetto T Ferrara

Role: primary

Fabrizio U Pane

Role: primary

Gianluca Gaidano

Role: primary

Sergio Siragusa

Role: primary

Gresele

Role: primary

Costanza Bosi

Role: primary

Elena Rivolti

Role: primary

Elena Rossi

Role: primary

Potito Rosario Scanzulli

Role: primary

Patrizio Mazza

Role: primary

Alessandra Borchiellini

Role: primary

Vincenzo Pavone

Role: primary

Francesco S Zaja

Role: primary

Davide Lazzarotto

Role: primary

Marco Ruggeri

Role: primary

Other Identifiers

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ITP0918

Identifier Type: -

Identifier Source: org_study_id

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