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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Response Rate and Response Duration After Discontinuation of Treatment With Thrombopoietin Receptor Agonists in 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 Summary: 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.
Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

Current Primary Outcome: 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. ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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. ]
  • Estimation of the suspension criteria in terms of frequency of discontinuation causes for each center. [ Time Frame: 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. ]
  • 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. ]
  • Estimation of treatment duration starting from the date of first dose of TPO-RAs. [ Time Frame: One year from start of recruitment. ]
  • Description of Platelet levels and TPO-RAs dosage at stop. [ Time Frame: 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. ]
  • 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. ]
  • Description of concomitant therapies since TPO-RAs start in terms of type and duration of therapies. [ Time Frame: One year from start of recruitment. ]


Original Secondary Outcome: Same as current

Information By: Gruppo Italiano Malattie EMatologiche dell'Adulto

Dates:
Date Received: November 17, 2014
Date Started: April 2016
Date Completion: January 2017
Last Updated: August 10, 2016
Last Verified: August 2016