NCT07338422尚未招募不适用
Nonrandomized Controlled Study of HLA-Haploidentical Hematopoietic Stem Cell Transplantation Versus Immunosuppressive Therapy as First-Line Treatment for Severe Aplastic Anemia
干预措施
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 116
- 主要终点
- Failure Free Survival, FFS
研究概览
简要总结
This study aims to compare the efficacy and safety of HLA-haploidentical hematopoietic stem cell transplantation (HLA-haplo HSCT) versus optimal immunosuppressive therapy (IST) as first-line treatments for severe aplastic anemia (SAA) through a real-world cohort design. The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.
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
入排标准
- 年龄范围
- 14 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •(1) Confirmed diagnosis of severe aplastic anemia, including very severe aplastic anemia, severe aplastic anemia, and hepatitis-associated severe aplastic anemia.
- •(2) Age 14-70 years. (3) No HLA-matched related donor available. (4) HIV negative, HBV negative, HCV negative. (5) No absolute contraindications to transplantation or immunosuppressive therapy.
- •(6) Signed informed consent form must be obtained before study procedures begin; for subjects aged 18 years or older, informed consent shall be signed by the patient themselves or direct family members. Considering the patient's medical condition, if the patient's own signature is medically inadvisable, the informed consent shall be signed by a legal guardian or the patient's direct family member.
排除标准
- •Inherited bone marrow failure syndromes (IBMFS), including Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond syndrome (SDS), etc.;
- •Clonal cytogenetic abnormalities or bone marrow examination suggesting pre-MDS or MDS;
- •Known severe allergy to ATG;
- •Previous allogeneic or autologous hematopoietic stem cell transplantation;
- •Previous solid organ transplantation;
- •Uncontrolled infection at enrollment, or requiring mechanical ventilation or hemodynamic instability;
- •Active HIV replication at enrollment, detectable HCV antibody positivity and HCV-RNA positivity within 90 days prior to enrollment, or HBsAg positivity; known seropositivity for HIV or active hepatitis C virus;
- •History of malignant tumors (except resected basal cell carcinoma or treated cervical carcinoma in situ);
- •Psychiatric disorders or other conditions that prevent compliance with study treatment and monitoring requirements;
- •Inability or unwillingness to sign the consent form;
- •Other special circumstances deemed ineligible by the investigator.
研究组 & 干预措施
IST
Other
干预措施: IST (ATG + CsA+TPORA) (Procedure)
HID-HSCT
Experimental
干预措施: tranplantation (Procedure)
结局指标
主要结局
Failure Free Survival, FFS
时间窗: 2 year
survival with complete response whereas death, graft failure and relapse are considered treatment failures
次要结局
- Overall Survival, OS(2 year)
- Treatment Related Mortality, TRM(2 year)
- hematology remission(2 year)
- Infection incidence(2 year)
- GVHD incidence(2 year)
- QoL evaluation(2 year)
研究者
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