Clinical trial NCT02298075: Response Rate and Duration After Discontinuation Thrombopoietin Receptor Agonists Primary Immune Thrombocytopenia
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 type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Immune Thrombocytopenia
Keywords: Immune thrombocytopenia
Primary
Thrombopoietin receptor agonist
Lead sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor class: OTHER
First posted: 2014-11-21
Enrollment: 148
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients affected by primary immune thrombocytopenia (pITP)

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.

Eligibility 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.

Primary outcomes:
measure: 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.
timeFrame: One year from start of recruitment.
Secondary outcomes:
measure: 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).
timeFrame: One year from start of recruitment.
measure: Estimation of the suspension criteria in terms of frequency of discontinuation causes for each center.
timeFrame: One year from start of recruitment.
measure: 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.
timeFrame: One year from start of recruitment.
measure: Estimation of incidence of relapse and loss of response at 6 and 12 months after sustained response achievement.
timeFrame: One year from start of recruitment.
measure: Estimation of treatment duration starting from the date of first dose of TPO-RAs.
timeFrame: One year from start of recruitment.
measure: Description of Platelet levels and TPO-RAs dosage at stop.
timeFrame: One year from start of recruitment.
measure: 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.
timeFrame: One year from start of recruitment.
measure: Assessment of an association between previous splenectomy and sustained response after discontinuation of treatment with TPO-RAs.
timeFrame: One year from start of recruitment.
measure: Description of concomitant therapies since TPO-RAs start in terms of type and duration of therapies.
timeFrame: One year from start of recruitment.
Other outcomes:
Locations:
city: Asti
country: Italy
facility: S.O.C. di Medicina Interna B - Ospedale - Cardinal Massaia di Asti
city: Bologna
country: Italy
facility: Istituto di Ematologia "Lorenzo e A. Seragnoli"
city: Catania
country: Italy
facility: Unità di Onco-Ematologia - Azienda Ospedaliera - Garibaldi
city: Firenze
country: Italy
facility: Unità di Ricerca e di Malattie del sangue - Ematologia San Luca Vecchio Pad. 16 - 1° Piano
city: Genova
country: Italy
facility: Clinica Medica III - Padiglione ex isolamento - Azienda Ospedale Università San Martino
city: Lecce
country: Italy
facility: ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE
city: Monza
country: Italy
facility: Azienda Ospedaliera "S.Gerardo"
city: Potenza
country: Italy
facility: Ematologia - Ospedale San Carlo
city: Reggio Emilia
country: Italy
facility: Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova
city: Roma
country: Italy
facility: Università Cattolica del Sacro Cuore - Policlinico A. Gemelli
city: Rome
country: Italy
facility: U.O.C. Ematologia - Ospedale S. Eugenio
city: Rome
country: Italy
facility: Università degli studi "Sapienza" di Roma
city: Sassari
country: Italy
facility: Ematologia - Dipartimento di Medicina Clinica e Sperimentale
Study officials:
affiliation: Policlinico Umberto I, Hematology Department.
name: Maria Gabriella Mazzucconi
role: STUDY_CHAIR
References: