RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation (REGALIA)

University Hospital, Lille

Status and phase: Terminated; Phase 2

Conditions: Graft Failure; Leukemia

Treatments: Drug: eltrombopag

Study type: Interventional

Funder types: Other

Identifiers

NCT03948529

2018-001157-27 (EudraCT Number)

2017_52

Details and patient eligibility

About

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

Enrollment: 9 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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9 participants in 1 patient group

eltrombopag

Experimental group

Description:

Eligible patients will receive the investigational drug eltrombopag

Treatment:

Drug: eltrombopag