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

October 22, 2018 updated by: Gruppo Italiano Malattie EMatologiche dell'Adulto

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

Unknown

Conditions

Intervention / Treatment

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 (Anticipated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Italy
      • Asti, Italy
        • Recruiting
        • S.O.C. di Medicina Interna B - Ospedale - Cardinal Massaia di Asti
        • Principal Investigator:
          • Monia Marchetti
        • Contact:
          • Monia Marchetti
      • Bologna, Italy
        • Recruiting
        • Istituto di Ematologia "Lorenzo e A. Seragnoli"
        • Contact:
          • Nicola Vianelli
        • Principal Investigator:
          • Nicola Vianelli
        • Sub-Investigator:
          • Francesca Palandrini
      • Catania, Italy
        • Not yet recruiting
        • Unità di Onco-Ematologia - Azienda Ospedaliera - Garibaldi
        • Contact:
          • Ugo Consoli
        • Principal Investigator:
          • Ugo Consoli
      • Firenze, Italy
        • Recruiting
        • Unità di Ricerca e di Malattie del sangue - Ematologia San Luca Vecchio Pad. 16 - 1° Piano
        • Contact:
          • Alberto Bosi
        • Principal Investigator:
          • Alberto Bosi
      • Genova, Italy
        • Recruiting
        • Clinica Medica III - Padiglione ex isolamento - Azienda Ospedale Università San Martino
        • Contact:
          • Enrico Balleari
        • Principal Investigator:
          • Enrico Balleari
      • Lecce, Italy
        • Recruiting
        • ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE
        • Principal Investigator:
          • Nicola Di Renzo
        • Sub-Investigator:
          • Margherita Giannoccaro
      • Monza, Italy
        • Recruiting
        • Azienda Ospedaliera "S.Gerardo"
        • Contact:
          • Monia Carpenedo
        • Principal Investigator:
          • Monia Carpenedo
      • Potenza, Italy
        • Recruiting
        • Ematologia - Ospedale San Carlo
        • Contact:
          • Angela Amendola
        • Principal Investigator:
          • Angela Amendola
      • Reggio Emilia, Italy
        • Recruiting
        • Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova
        • Contact:
          • Elena Rivolti
        • Principal Investigator:
          • Elena Rivolti
      • Roma, Italy
        • Recruiting
        • Università Cattolica del Sacro Cuore - Policlinico A. Gemelli
        • Principal Investigator:
          • Valerio De Stefano
        • Sub-Investigator:
          • Elena Rossi
        • Contact:
          • Valerio De Stefano
      • Rome, Italy
        • Recruiting
        • U.O.C. Ematologia - Ospedale S. Eugenio
        • Contact:
          • Laura Scaramucci
        • Principal Investigator:
          • Laura Scaramucci
        • Sub-Investigator:
          • Pasquale Niscola
      • Rome, Italy
        • Recruiting
        • Università degli studi "Sapienza" di Roma
        • Contact:
          • Maria Gabriella Mazzucconi
      • Sassari, Italy
        • Recruiting
        • Ematologia - Dipartimento di Medicina Clinica e Sperimentale

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

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.
Time Frame: One year from start of recruitment.
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).
Time Frame: One year from start of recruitment.
One year from start of recruitment.
Estimation of the suspension criteria in terms of frequency of discontinuation causes for each center.
Time Frame: One year from start of recruitment.
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.
Time Frame: One year from start of recruitment.
One year from start of recruitment.
Estimation of incidence of relapse and loss of response at 6 and 12 months after sustained response achievement.
Time Frame: One year from start of recruitment.
One year from start of recruitment.
Estimation of treatment duration starting from the date of first dose of TPO-RAs.
Time Frame: One year from start of recruitment.
One year from start of recruitment.
Description of Platelet levels and TPO-RAs dosage at stop.
Time Frame: One year from start of recruitment.
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.
Time Frame: One year from start of recruitment.
One year from start of recruitment.
Assessment of an association between previous splenectomy and sustained response after discontinuation of treatment with TPO-RAs.
Time Frame: One year from start of recruitment.
One year from start of recruitment.
Description of concomitant therapies since TPO-RAs start in terms of type and duration of therapies.
Time Frame: One year from start of recruitment.
One year from start of recruitment.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Gruppo Italiano Malattie EMatologiche dell'Adulto

Investigators

  • Study Chair: Maria Gabriella Mazzucconi, Policlinico Umberto I, Hematology Department.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2016

Primary Completion (Anticipated)

December 1, 2018

Study Completion (Anticipated)

December 1, 2018

Study Registration Dates

First Submitted

November 17, 2014

First Submitted That Met QC Criteria

November 19, 2014

First Posted (Estimate)

November 21, 2014

Study Record Updates

Last Update Posted (Actual)

October 23, 2018

Last Update Submitted That Met QC Criteria

October 22, 2018

Last Verified

October 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ITP0714

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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