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

Sponsors

Lead Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

Source Gruppo Italiano Malattie EMatologiche dell'Adulto
Brief Summary

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.

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.

Overall Status Recruiting
Start Date April 2016
Completion Date December 2018
Primary Completion Date December 2018
Study Type Observational
Primary 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
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.
Enrollment 100
Condition
Intervention

Intervention Type: Drug

Intervention Name: Eltrombopag

Description: Investigation of the impact of TPO-RAs on the achievement of sustained and persistent response after discontinuation in adult patients affected by pITP.

Other Name: Thrombopoietin Receptor Agonists (TPO-RAs)

Intervention Type: Drug

Intervention Name: Romiplostin

Description: Investigation of the impact of TPO-RAs on the achievement of sustained and persistent response after discontinuation in adult patients affected by pITP.

Other Name: Thrombopoietin Receptor Agonists (TPO-RAs)

Eligibility

Sampling Method: Non-Probability Sample

Criteria:

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.

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Maria Gabriella Mazzucconi Study Chair Policlinico Umberto I, Hematology Department.
Overall Contact

Last Name: Enrico Crea

Email: [email protected]

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

Italy

Verification Date

October 2018

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Patient Data No
Study Design Info

Observational Model: Other

Time Perspective: Retrospective

Source: ClinicalTrials.gov