Poor Graft Function (PGF) after allogeneic hematopoietic cell transplantation (allo-HCT). PGF is commonly defined as follows: one or several significant cytopenias after allo-HCT persisting or developing after allo-HCT despite full donor chimerism and in the absence of relapse or other causes (in particular graft-versus-host disease [GVHD]).
Conditions
Interventions
Trade Name: Revolade
Pharmaceutical Form: Film-coated tablet
Sponsors
CHRU of Lille
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Patient age = 6 years old - Diagnosis of poor graft function defined as: • Patient = day+60 after allo-HCT, • Persisting thrombocytopenia on two different samples over at least two weeks (platelet =65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: • Criteria for poor graft function not fulfilled (see above), • Patients aged less than 6 years old (or unable to swallow), • Hepatic impairment (Child-Pugh = 5), • Hypersensitivity to eltrombopag or to any of the excipients, • Patients with any contra-indication to eltrombopag, filgrastim, • Unable to understand the investigational nature of the study or give informed consent, • History of congestive heart failure, arrhythmia requiring chronic treatment, arterial or venous, • Thrombosis (not excluding line thrombosis) within the last 1 year, or myocardial infarction within 3 months before enrollment, • ECOG Performance Status of 3 or greater, • Pregnant and/or lactating women, • Freedom privacy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate that eltrombopag improves PGF.;Secondary Objective: - To decrease the need for transfusions, - To decrease the rate of bleeding events, - To decrease the rate of infectious events, - To improve quality of life parameters, - To demonstrate that eltrombopag improves bone marrow cellularity, - To evaluate overall survival and non-relapse mortality, - To evaluate adverse events. ;Primary end point(s): Platelet response defined as a platelet =20G/L at 12 weeks measured on at least two serial measurements performed 1 week apart and sustained for 1 month or more without support of platelet transfusions.;Timepoint(s) of evaluation of this end point: As specified in the primary end point : platelet =20G/L at 12 weeks of treatment. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Time toerythroid response defined as an increase of at least 1.5g/dL without transfusion that is sustained for at least 2 weeks, - Time to neutrophil response defined as an increase of ANC above 1G/L, that is sustained for at least 7 days, - Marrow best response at 12 and 24 weeks, - Transfusion requirements at 12 and 24 weeks for BRC and platelets as compared with transfusions requirements during the eight weeks preceding study entry, - Severe adverse events, - Quality of life parameters at 12 and 24 weeks, - Immune function (T/B/NK cells counts) at 12 and 24 weeks, - 6-month overall survival, - 6-month relapse-free survival, - 6-month non relapse mortality, - 6-month cumulative incidence of underlying disease relapse. ;Timepoint(s) of evaluation of this end point: As spicified for each end point. | — |
Countries
France
Contacts
Public ContactKarim DAHACHE (CRA)
Clinical Research Federation
Outcome results
None listed