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

Sponsor

Gruppo Italiano Malattie EMatologiche dell'Adulto (Other)

Overall Status

Unknown status

CT.gov ID

NCT02298075

Collaborator

(none)

100

Enrollment

Locations

Duration (Months)

7.7

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Immune Thrombocytopenia	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 Design

Study Type:

Observational

Anticipated Enrollment :

100 participants

Observational Model:

Other

Time Perspective:

Retrospective

Official Title:

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

Study Start Date :

Apr 1, 2016

Anticipated Primary Completion Date :

Dec 1, 2018

Anticipated Study Completion Date :

Dec 1, 2018

Outcome Measures

Primary Outcome Measures

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

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.]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

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