A Research on Haploidentical Transplantation in Severe Aplastic Anemia Using Reduced-intensity Fludarabine-based Conditioning
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 60
- 试验地点
- 2
- 主要终点
- Engraftment
研究概览
简要总结
This is a prospective case-control study on SAA patients treated with HSCT, order to further discuss and assess the safety, feasibility and effectiveness of HFD-HSCT which performed with reduced-intensity fludarabine-based conditioning regimen.Our findings would indicate that SAA patients who lack MSD benefited most if HFD-HSCT was performed with reduced-intensity fludarabine-based conditioning regimen, and our improved outcomes with HFD-HSCT may lead to a salvaged therapy and an expanded direct role for SAA in the future.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
盲法说明
no masking
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •(i) Diagnosis of SAA, very SAA or SAA and paroxysmal nocturnal hemoglobinuria (PNH) according to the International Aplastic Anemia Study Group; (ii) SAA patients no response to previous IST; (iii) adequate performance status [Eastern Cooperative Oncology Group (ECOG) score 0-2].
排除标准
- •(i) Congenital forms of aplastic anemia; (ii)Patients with any severe pulmonary, cardiac, liver, or renal diseases or active infection.
研究组 & 干预措施
MSD-HSCT
This group received treatment of matched sibling donor - hematopoietic stem cell transplantation (MSD-HSCT).
干预措施: MSD-HSCT (Other)
HFD-HSCT
This group received treatment of haploid family donor - hematopoietic stem cell transplantation (HFD-HSCT).
干预措施: HFD-HSCT (Other)
结局指标
主要结局
Engraftment
时间窗: In the first months after infusion
Neutrophil engraftment was defined as the first of three consecutive days in which the neutrophil counts (ANC) exceeded 0.50 × 109/L, and platelet engraftment was defined as the first of five consecutive days in which the platelet count exceeded 20 × 109/L without transfusion. GF was classified as follows: (1) primary non-engraftment (failure to reach a neutrophil count of 0.5×109/L after transplant); (2) rejection (decrease in blood counts to \< 0.5×109/L neutrophils, after achieving a neutrophil count of 0.5×109/L); (3) late graft failure (decrease of blood counts after day 100 to \< 1.0×109/L neutrophils and \< 30×109/L platelets).
Toxicity grading
时间窗: TRT was defined as toxic effects occurring within 40 days after HSCT
The transplantation-related toxicity (TRT) was graded using the National Cancer Institute Common Toxicity Criteria for Adverse Events version 4.0. Organ damage due to GVHD or infectious complications were excluded.
Chimerism analyses +30
时间窗: Days +30 after HSCT
Chimerism would be evaluated in recipient BM cells usually on days +30 after HSCT using cytogenetic G-banding or fluorescence in situ hybridization. Sex-matched donor-recipient chimerism was assessed using PCR-based analyses of polymorphic minisatellite or microsatellite regions. HLA typing was performed for patients with HLA-haploidentical donors.
Chimerism analyses +100
时间窗: Days +100 after HSCT
Chimerism would be evaluated in recipient BM cells usually on days +180 after HSCT using cytogenetic G-banding or fluorescence in situ hybridization. Sex-matched donor-recipient chimerism was assessed using PCR-based analyses of polymorphic minisatellite or microsatellite regions. HLA typing was performed for patients with HLA-haploidentical donors.
Chimerism analyses +180
时间窗: Days +180 after HSCT
Chimerism would be evaluated in recipient BM cells usually on days +100 after HSCT using cytogenetic G-banding or fluorescence in situ hybridization. Sex-matched donor-recipient chimerism was assessed using PCR-based analyses of polymorphic minisatellite or microsatellite regions. HLA typing was performed for patients with HLA-haploidentical donors.
Chimerism analyses +365
时间窗: Days +365 after HSCT
Chimerism would be evaluated in recipient BM cells usually on days +365 after HSCT using cytogenetic G-banding or fluorescence in situ hybridization. Sex-matched donor-recipient chimerism was assessed using PCR-based analyses of polymorphic minisatellite or microsatellite regions. HLA typing was performed for patients with HLA-haploidentical donors.
OS 1-year
时间窗: 1-year after HSCT
OS was defined as the time from transplantation to death from any cause or the last follow-up.
OS 2-year
时间窗: 2-year after HSCT
OS was defined as the time from transplantation to death from any cause or the last follow-up.
OS 5-year
时间窗: 5-year after HSCT
OS was defined as the time from transplantation to death from any cause or the last follow-up.
EFS 1-year
时间窗: 1-year after HSCT
EFS was defined as survival with a response to therapy. Death, GF and relapse were considered as treatment failure. EFS was defined as survival with a response to therapy. Death, GF and relapse were considered as treatment failure.
EFS 2-year
时间窗: 2-year after HSCT
EFS was defined as survival with a response to therapy. Death, GF and relapse were considered as treatment failure.
EFS 5-year
时间窗: 5-year after HSCT
EFS was defined as survival with a response to therapy. Death, GF and relapse were considered as treatment failure.
次要结局
未报告次要终点
研究者
Wu Xiaoxiong
Head, Research group of the Center of Hematology
Chinese PLA General Hospital