Efficacy and Safety of Allogeneic Hematopoietic Cell Transplantation Using Post-transplantation Cyclophosphamide for Graft-versus-host Disease Prophylaxis in Higher-risk Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia, and Secondary AML Patients
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 113
- 试验地点
- 1
- 主要终点
- GVHD-free, relapse-free survival
研究概览
简要总结
This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival.
The secondary end points of the study include engraftment, relapse incidence, non-relapse mortality, graft-versus-host disease, donor chimerism, immune reconstitution, infections, and survivals (overall and event-free).
详细描述
This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival.
The secondary end points of the study include engraftment, relapse incidence, non-relapse mortality, graft-versus-host disease, donor chimerism, immune reconstitution, infections, and survivals (overall and event-free).
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
- 15 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •MDS defined by WHO classification, CMML, or AML evolving from MDS A. International Prognostic Scoring System (IPSS) > 1.0 or bone marrow blast ≥ 5% at any time points before HCT or B. AML progressed from MDS or C. CMML with bone marrow blast ≥ 5% at any time points before HCT
- •Patients receiving first HCT
- •Patients with appropriate hematopoietic cell donors A. HLA-matched sibling donor B. Unrelated donor C. HLA-mismatched familial donor
- •15 years old or older , under 75 years
- •Adequate performance status (Karnofsky score of 70 or more)
- •Adequate hepatic function (AST or ALT < 3 x upper normal limits and bilirubin < 1.5 x upper normal limit).
- •Adequate renal function (creatinine < 2.0 mg/dL or creatinine clearance ≥ 50 mL/min)
- •Adequate cardiac function (left ventricular ejection fraction ≥ 50% on heart scan or echocardiogram)
- •Adequate pulmonary function: DLCO, FEV1, and FVC ≥ 45% predicted by pulmonary function tests
- •Signed and dated informed consent must be obtained from both recipient and donor.
排除标准
- •Presence of significant active infection
- •Presence of uncontrolled bleeding
- •Any coexisting major illness or organ failure
- •Patients with psychiatric disorder or mental deficiency severe as to make compliance with the treatment unlike, and making informed consent impossible
- •Nursing women, pregnant women, women of childbearing potential who do not want adequate contraception
- •Patients with a diagnosis of prior malignancy unless disease-free for at least 5 years following therapy with curative intent (except curatively treated nonmelanoma skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia)
- •Presence of contraindications to cyclophosphamide (patients receiving pentostatin, symptomatic cystitis, urinary tract obstruction, with a history of hypersensitivity reactions to the component of the drug)
研究组 & 干预措施
PTCy
Patients who receive post-transplantation cyclophosphamide
干预措施: Cyclophosphamide (Drug)
结局指标
主要结局
GVHD-free, relapse-free survival
时间窗: 8 years
This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival.
次要结局
未报告次要终点
研究者
Je-Hwan Lee
M.D. Professor
Asan Medical Center