RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation: a Prospective, Phase II Study by the SFGM-TC
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Platelet response
研究概览
简要总结
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
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
eltrombopag
Eligible patients will receive the investigational drug eltrombopag
干预措施: eltrombopag (Drug)
结局指标
主要结局
Platelet response
时间窗: at 12 weeks
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.
次要结局
- 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)
- Time to erythroid response(at 12 and 24 weeks)
- Time to neutrophil response(at least 7 days)
- 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,)
- Immune function (T/B/NK cells counts)(at 12 and 24 weeks)
- Overall survival, relapse-free survival and non-relapse mortality(at 24 weeks of treatment)
- Quality of life evaluation using the European Organisation for Research Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30 questionnaire).(at 12 and 24 weeks)