RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation

NCT03948529 | Terminated Phase 2 DMC

• 2 other identifiers: 2017_522018-001157-27
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

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

Keywords

Poor Graft Function (PGF),; Allogeneic hematopoietic cell transplantation (Allo-HCT); Eltrombopag.

Outcome Measures

Primary Outcomes (1)

• Platelet response

  Platelet response defined as a platelet count ≥ 30G/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.

  at 12 weeks

Secondary Outcomes (8)

• Time to erythroid response

  at 12 and 24 weeks

• Time to neutrophil response

  at least 7 days

• Percentage of patients presenting best bone marrow response at 12 and 24 weeks of treatment assessed by bone marrow aspirate and bone marrow biopsy with fibrosis staining.

  at 12 and 24 weeks

• Transfusion requirements

  at 12 and 24 weeks

• Proportion of patients presenting grade 3 or 4 adverse events from the first to the last administration of eltombopag.

  from 1st administration of eltrombopag to 1 month after the last administration of eltrombopag,

Study Arms (1)

eltrombopag

EXPERIMENTAL

Eligible patients will receive the investigational drug eltrombopag

Drug: eltrombopag

Interventions

eltrombopag 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).

eltrombopag

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University Hospital, Lille: lead

Study Sites (1)

Hôpital Claude Huriez, CHU

Lille, France

Location

MeSH Terms

Conditions

Leukemia

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Ibrahim Yakoub-Agha, MD,PhD

  University Hospital, Lille

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 28, 2019
• First Posted: May 14, 2019
• Study Start: September 5, 2019
• Primary Completion: February 24, 2023
• Study Completion: August 21, 2023
• Last Updated: September 30, 2025

Record last verified: 2025-09

Locations

FR (1)