Perioperative Eltrombopag in Patients With Inherited Thrombocytopenia (ELPOT)
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|ClinicalTrials.gov Identifier: NCT03638817|
Recruitment Status : Recruiting
First Posted : August 20, 2018
Last Update Posted : January 23, 2019
|Condition or disease||Intervention/treatment||Phase|
|Thrombocytopenia||Drug: Eltrombopag||Phase 2|
The hypothesis of the trial is that preoperative treatment by a thrombopoietin mimetic (eltrombopag) will be effective and safe and will avoid requirement of PC administration in a majority of IT patients Eltrombopag is a thrombopoietin mimetic available orally, not licenced for the treatment of IT. Preliminary data in short series of IT patients indicate that eltrombopag, at the doses used in primary immune thrombocytopenia, increases the platelet counts after 2-4 weeks of treatment and reduces spontaneous bleeding in a significant proportion of subjects. The tolerance of short-term treatment is good. The experience of eltrombopag for the management of perioperative thrombocytopenia in IT is anecdotic. Avoiding the administration of platelet concentrates in these patients, especially children, would represent a direct benefit by preventing adverse reactions to transfusion of blood products and human leukocyte antigen (HLA) immunisation.
Eltrombopag will be prescribed after the inclusion visit at the standard dose of 50 mg/day with dose adjustment on the platelet count (+/- 25 mg) after 2 weeks, for a maximum of 4 weeks before the invasive procedure. If the predefined safety level of platelet count required for the procedure is reached, the treatment will be discontinued and the patient operated without prophylactic administration of PC. In case of bleeding of undetermined cause per-and post-operatively, rescue PC will be given.
Clinical and biological follow-up will be performed until the end-of-study visit, 4 weeks after the intake of the last tablet of eltrombopag.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Evaluation of Perioperative Eltrombopag for the Management of Elective Surgery and Invasive Acts in Patients With Inherited Thrombocytopenia|
|Actual Study Start Date :||December 14, 2018|
|Estimated Primary Completion Date :||March 2021|
|Estimated Study Completion Date :||September 2021|
Eltrombopag will be prescribed at doses recommended in primary immune thrombocytopenia (50, 25 or 75 mg), starting 4 weeks before the procedure and stopped 2 days before. PC will be administrated prophylactically if the platelet count is < 80 G/L or per/post-operatively in case of bleeding of undetermined cause.
Antifibrinolytics will be authorized and low molecular weight heparin prescribed if indicated for the prophylaxis of postoperative venous thrombosis according to the standard dose and duration, , irrespective of the platelet count
- Perioperative management by eltrombopag in inherited thrombocytopenia [ Time Frame: up to 4 weeks after completion of treatment ]The response to Eltrombopag is a composite criteria including the level of platelet count 2 days before the procedure and the requirement of PC administration at any time in the study period. The "study period" is running from the start of treatment (inclusion visit) to 4 weeks after completion of treatment. A platelet count remaining below 80 G/L preoperatively, whether or not eltrombopag was taken, is a criterion of failure of treatment.
- Adverse events [ Time Frame: up to 4 weeks after completion of treatment ]Adverse events and adverse reactions occurring at any time during the study period will be collected. Adverse events may be clinical and biological (especially liver function tests).
- Excessive bleeding [ Time Frame: up to 4 weeks after completion of treatment ]Excessive or unusual bleeding occurring at any time during the study period are major adverse events. An independent event adjudication committee (EAC) will review all bleeding events.
- Vascular thrombosis [ Time Frame: up to 4 weeks after completion of treatment ]Symptomatic thrombosis (venous or arterial) occurring at any time during the study period will be diagnosed by appropriate objective methods and reviewed by the EAC.
- Doses of eltrombopag on-treatment [ Time Frame: 2 and 4 weeks after the beginning of the treatment ]The total doses of eltrombopag given in the preoperative period will be recorded, as the dose and duration of treatment required to obtained the safety level
- Platelet kinetics [ Time Frame: up to 4 weeks after completion of treatment ]Serial blood sampling during the study period will be performed for measuring the rise of platelet count on-treatment and its decline after completion of treatment.
- Platelet size [ Time Frame: Inclusion and before the procedure ]Mean platelet volume will be measured by flow cytometry.on blood samples obtained for platelet counts at inclusion, during hospitalisation and end-of study visit
- Baseline of Serum Thrombopoietin [ Time Frame: Inclusion visit ]Serum thrombopoietin will be measured once, at the inclusion visit.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03638817
|Contact: Pierre SIE, Prof.||561322289 ext firstname.lastname@example.org|
|university hospital Toulouse||Recruiting|
|Contact: Pierre SIE, MD|
|Principal Investigator:||Pierre SIE, Prof.||University Hospital, Toulouse|