RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation

NCT ID: NCT03948529

Last Updated: 2025-09-30

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-09-05
• Study Completion Date: 2023-08-21

Brief Summary

Poor graft function (PGF) after allogeneic hematopoietic cell transplantation (allo-HCT) is a misunderstood complication associated with poor outcome and limited therapeutic options. Despite the lack of standardized diagnostic criteria, 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. Not only PGF can alter patients' quality of life by leading to recurrent transfusions, bleeding events and infections, but it is also associated with poor survival after allo-HCT.

Although PGF is relatively frequent, there is no well-codified behavior in the literature or in the recommendations issued by the various learned societies of transplantation.

The aim objective of the investigator's study is to demonstrate that eltrombopag improve PGF after allo-HCT

Conditions

Leukemia; Graft Failure

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

eltrombopag

Eligible patients will receive the investigational drug eltrombopag

Group Type: EXPERIMENTAL

eltrombopag

Intervention Type: DRUG

eltrombopag at the starting dose of 50mg/day. After 2 weeks of eltrombopag initiation and in the absence of platelet response, eltrombopag will be increased every two weeks (50mg increase) up to a maximum dose of 150mg/day (2 maximum escalation from D1, with maximum dose escalation phase of 4 weeks).

Interventions

eltrombopag

eltrombopag at the starting dose of 50mg/day. After 2 weeks of eltrombopag initiation and in the absence of platelet response, eltrombopag will be increased every two weeks (50mg increase) up to a maximum dose of 150mg/day (2 maximum escalation from D1, with maximum dose escalation phase of 4 weeks).

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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 < 30G/L with transfusion requirement) +/- neutropenia (ANC <1G/L) +/- anemia (Hb <8g/dL or transfusion requirement),
• Full donor chimerism on whole blood (≥ 95%),
• Biopsy proven hypocellular marrow without evidence of myelodysplasia
• No evidence for relapse,
• No evidence for active acute or chronic graft versus host disease,
• Absence of active viral infection (EBV, CMV, ADENOVIRUS, PARVOVIRUS B19),
• Absence of B9/B12 deficiency,
• Absence of hypothyroidism,
• Absence of hypogonadism,
• Absence of dialysis,
• Absence of thrombotic microangiopathy,
• Absence of macrophage activation syndrome,
• No other known causes of poor graft function.
• Written informed consent must be obtained before any study-trial specific procedure are performed,
• Affiliation to a social security system.

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),
• Patients with bone morrow fibrosis,
• Patients with a cytogenetic abnormality of chromosome 7
• 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.

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Hospital, Lille

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ibrahim Yakoub-Agha, MD,PhD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Lille

Locations

Hôpital Claude Huriez, CHU

Lille, , France

Countries

France

Other Identifiers

2018-001157-27

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2017_52

Identifier Type: -

Identifier Source: org_study_id