Response Rate and Duration After Discontinuation Thrombopoietin Receptor Agonists Primary Immune Thrombocytopenia

Response Rate and Response Duration After Discontinuation of Treatment With Thrombopoietin Receptor Agonists in Patients Affected by Primary Immune Thrombocytopenia (pITP): Retrospective Study

The primary objective of the study is the evaluation of sustained response rate after discontinuation of treatment with TPO-RAs, Eltrombopag and Romiplostim, in persistent or chronic pITP patients who failed one or more therapy lines, splenectomy included.

Study Overview

• Status: Completed
• Conditions: Immune Thrombocytopenia
• Intervention / Treatment: Drug: Eltrombopag; Drug: Romiplostin

Detailed Description

Increasing evidence exists in the literature on the persistent response after TPO-RAs discontinuation. However, the available data consist of description of case reports from different patients' series. At this time, systematic data collections on this topic do not exist and the real incidence of such persistent response is unknown. Therefore, because of the increasing interest in this new and challenging therapeutic field, also for the therapeutic implications, we would like to investigate the impact of TPO- RAs on the achievement of sustained and persistent responses after their discontinuation in the Italian adult patients affected by pITP who have been referred to the GIMEMA Centers.

• Study Type: Observational
• Enrollment (Actual): 148

Contacts and Locations

Study Locations

• Italy
  • Asti, Italy
    • S.O.C. di Medicina Interna B - Ospedale - Cardinal Massaia di Asti
  • Bologna, Italy
    • Istituto di Ematologia "Lorenzo e A. Seragnoli"
  • Catania, Italy
    • Unità di Onco-Ematologia - Azienda Ospedaliera - Garibaldi
  • Firenze, Italy
    • Unità di Ricerca e di Malattie del sangue - Ematologia San Luca Vecchio Pad. 16 - 1° Piano
  • Genova, Italy
    • Clinica Medica III - Padiglione ex isolamento - Azienda Ospedale Università San Martino
  • Lecce, Italy
    • ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE
  • Monza, Italy
    • Azienda Ospedaliera "S.Gerardo"
  • Potenza, Italy
    • Ematologia - Ospedale San Carlo
  • Reggio Emilia, Italy
    • Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova
  • Roma, Italy
    • Università Cattolica del Sacro Cuore - Policlinico A. Gemelli
  • Rome, Italy
    • U.O.C. Ematologia - Ospedale S. Eugenio
  • Rome, Italy
    • Università degli studi "Sapienza" di Roma
  • Sassari, Italy
    • Ematologia - Dipartimento di Medicina Clinica e Sperimentale

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients affected by primary immune thrombocytopenia (pITP)

Description

Inclusion Criteria:

• Signed written informed consent according to ICH/EU/GCP and national local laws;
• Persistent or chronic pITP adult patients who have been treated with TPO-RAs therapy after failure of one or more therapy lines, splenectomy included;
• Age > 18 years.

Exclusion Criteria:

• Active malignancy;
• Active malignancy;
• HCVAb, HIVAb, HBsAg, HBcAb seropositive status;
• Chronic liver disease;
• Treatment with Rituximab less than 8 weeks before TPO-RAs start;
• Recent splenectomy (less than 8 weeks) before TPO-RAs start. Active malignancy;
• HCVAb, HIVAb, HBsAg, HBcAb seropositive status;
• Chronic liver disease;
• Treatment with Rituximab less than 8 weeks before TPO-RAs start;
• Recent splenectomy (less than 8 weeks) before TPO-RAs start. HCVAb, HIVAb, HBsAg, HBcAb seropositive status;
• Chronic liver disease;
• Treatment with Rituximab less than 8 weeks before TPO-RAs start;
• Recent splenectomy (less than 8 weeks) before TPO-RAs start.

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Estimation of sustained response rate after discontinuation of treatment with TPO-RAs in terms of patients still alive in Complete Response or R at more than 4 weeks after discontinuation.	One year from start of recruitment.

Secondary Outcome Measures

Outcome Measure	Time Frame
Frequency of the type of response in patients treated with TPO-RAs in absence of concomitant or rescue medication, in order to estimate the rate of Response (R) and Complete Response (CR).	One year from start of recruitment.
Estimation of the suspension criteria in terms of frequency of discontinuation causes for each center.	One year from start of recruitment.
Estimation of the duration of sustained response in terms of patients alive in CR or R at 6 and 12 months after sustained response achievement.	One year from start of recruitment.
Estimation of incidence of relapse and loss of response at 6 and 12 months after sustained response achievement.	One year from start of recruitment.
Estimation of treatment duration starting from the date of first dose of TPO-RAs.	One year from start of recruitment.
Description of Platelet levels and TPO-RAs dosage at stop.	One year from start of recruitment.
Description of patients' characteristics in term of: biological and clinical features, disease duration at the beginning of TPO-RAs, number and type of therapy lines preceding TPO-RAs treatment, platelet levels at TPO-RAs start.	One year from start of recruitment.
Assessment of an association between previous splenectomy and sustained response after discontinuation of treatment with TPO-RAs.	One year from start of recruitment.
Description of concomitant therapies since TPO-RAs start in terms of type and duration of therapies.	One year from start of recruitment.

Collaborators and Investigators

• Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
• Investigators: Study Chair: Maria Gabriella Mazzucconi, Policlinico Umberto I, Hematology Department.

Publications and helpful links

Helpful Links

• GIMEMA Foundation website

Study record dates

Study Major Dates

• Study Start: April 1, 2016
• Primary Completion (Actual): April 17, 2019
• Study Completion (Actual): April 17, 2019

Study Registration Dates

• First Submitted: November 17, 2014
• First Submitted That Met QC Criteria: November 19, 2014
• First Posted (Estimated): November 21, 2014

Study Record Updates

• Last Update Posted (Actual): April 29, 2025
• Last Update Submitted That Met QC Criteria: April 28, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Keywords: Primary; Immune thrombocytopenia; Thrombopoietin receptor agonist
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: ITP0714

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO