Evaluation of the Use of Thrombopoietin Receptor Agonists in Adults With Primary ITP in Europe

NCT06408324 | Completed

• 1 other identifier: VERTEX 3.0
• Study Type: observational
• Target: 275
• Locations: 6 countries
• Sites: 17

Brief Summary

The goal of this multicenter, observational, retrospective is to evaluate the standards of use of thrombopoietin receptor agonists (TPO-RA) in adult patients with primary immune thrombocytopenia (ITP), with a particular interest on phase of ITP (defined according to time when diagnosis of ITP is established in the medical records: newly diagnosed, 0-3 months; persistent, 3-12 months, and chronic, \> 12 months), tolerability, safety, posology and remissions, outside controlled clinical trials. Secondary Objectives: To analyze the patient characteristics that could affect the choice of thrombopoietin receptor agonist for the treatment of ITP, considering specific clinical aspects (such as previous thromboembolic disease, bleeding, platelet count, surgical procedures, etc.). To evaluate the degree of adherence to international guidelines related to the use of thrombopoietin receptor agonists in regular clinical practice. Data will be collected through a retrospective chart review of patients with ITP who started TPO-RA treatment between January 2014 and December 2018.

Conditions

Primary Immune Thrombocytopenia (ITP)

Keywords

Immune Thrombocytopenia; ITP; Thrombopoietin receptor agonists; TPO-RA; Eltrombopag; Romiplostim

Outcome Measures

Primary Outcomes (1)

• Evaluation of the standards of use of TPO-RA in regular clinical practice in Europe

  To evaluate the standards of use of TPO-RA in regular clinical practice in Europe, with reference to phase of ITP (defined according to time when diagnosis of ITP is established in the medical records: newly diagnosed; persistent and chronic), tolerability, safety, posology and remissions, outside controlled clinical trials. The primary end-point is the proportion of patients achieving a complete response (platelet count of ≥100 × 109 /L) or a response (platelet count of ≥30 × 109 /L) after TPO-RA initiation and upon switching. Responses are recorded for any TPO-RA treatment initiated between 1 January 2014 and 31 December 2018 up to the discontinuation of the last line of treatment with TPO-RA.

  From the first TPO-RA treatment initiated between 1 January 2014 and 31 December 2018 up to the discontinuation of the last line of treatment with TPO-RA. The maximum follow-up period could be of 10 years.

Secondary Outcomes (1)

• To analyze the patient characteristics that could affect the choice of TPO-RA for the treatment of ITP and to evaluate the degree of adherence to international guidelines

  From the first TPO-RA treatment initiated between 1 January 2014 and 31 December 2018 up to the discontinuation of the last line of treatment with TPO-RA. The maximum follow-up period could be of 10 years.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Approximately 350 patients will be included in the study. Each site will collect data of ITP patients who meet the screening criteria and agree to participate in the study, considering that it must include patients treated with romiplostim and eltrombopag ideally with the largest follow-up, meaning those who started agonist therapy on the date closest to January 2014. For the selection of patients, the investigators will detect among the patients at their center (who generally will have outpatient follow-up in consultations), those who meet the inclusion criteria defined below and who do not present any exclusion criteria.

You may qualify if:

• Patients over age 18 when TPO-RA was started
• Diagnosis of primary immune thrombocytopenia
• Patients who started treatment for ITP with thrombopoietin receptor agonists (romiplostim/eltrombopag) between January 2014 and December 2018.
• The investigators will review the medical records of patients to include those with longer follow-up in the study, meaning those who started with the agonist on the date closest to January 2014. Patients will be included in chronological order based on date of TPO-RA initiation, and signed informed consent.
• \- Signed Informed consent\*
• \* An exception will be the case of deceased patients, in which the investigator will sign and date the informed consent document, and will specify the absence of the patient's signature due to death. However, in the event that the codes of the Member State that regulate the processing of personal data of deceased persons do not enable to obtain this information, the specific regulations of each State will be followed.

You may not qualify if:

• Patients who had participated in studies with investigational drugs when TPO-RA was started
• Patients with a diagnosis of secondary immune thrombocytopenia when TPO-RA was started

Sponsors & Collaborators

• European Research Consortium on ITP: lead
• Fondazione Progetto Ematologia: collaborator

Study Sites (17)

Hôpital Universitaire Henri Mondor

Créteil, France

Location

CHU Dijon - Hôpital François Mitterrand Médecine 1 - Etudes Cliniques

Dijon, France

Location

CHU Toulouse - Hôtel-Dieu Saint-Jacques

Toulouse, France

Location

Careggi Hospital

Florence, Italy

Location

Fondazione Policlinico A. Gemelli IRCCS

Roma, Italy

Location

San Bortolo Hospital, Vicenza

Vicenza, Italy

Location

Bergen Hospital

Bergen, Norway

Location

Oslo Hospital

Oslo, Norway

Location

Østfold Hospital

Oslo, Norway

Location

Hospital del Mar, Barcelona

Barcelona, Spain

Location

Hospital General Universitario de Burgos

Burgos, Spain

Location

Hospital Morales Meseguer

Murcia, Spain

Location

Basel university hospital

Basel, Switzerland

Location

Bern university hospital

Bern, Switzerland

Location

Lausanne University hospital

Lausanne, Switzerland

Location

Imperial College NHS Trust

London, United Kingdom

Location

University Hospitals Plymouth NHS Trust

Plymouth, United Kingdom

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Maria L. Lozano, M.D.

  Hematology and Medical Oncology Dept. Hospital General Universitario José María Morales Meseguer

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2023
• First Posted: May 10, 2024
• Study Start: November 24, 2022
• Primary Completion: April 5, 2024
• Study Completion: April 5, 2024
• Last Updated: May 16, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

ES (3); GB (2); IT (3); CH (3); FR (3); NO (3)