Non-Transplant Therapies Aplastic Anemia.

Editor's Notes

• #17: 这一观念，也得到了大部分国内专家的共识。
• #18: Neal S. Young在《再障病理生理学和治疗方面的现有观念》中也提到，ATG联合环孢素仍作为标准的一线免疫抑制治疗 。
• #19: 免疫抑制治疗和造血干细胞移植是目前再障主要的治疗方法，英国再障诊断和治疗指南中指出，对于缺少匹配供者的患者，ATG联合环孢素的免疫抑制疗法是首选治疗。
• #20: 关于免疫抑制治疗的疗效，国内外的报道不尽相同，但大约都在70-80%之间。右图显示在一个大样本的研究中，应用马ATG联合环孢素的标准免疫抑制治疗重再患者的生存率，没有并发症的患者10年生存率为95%，而发生并发症事件的10年生存率则为65%。 A large cohort (N=243) of NIH patients who responded to treatment with the standard regimen of horse ATG plus cyclosporine was analyzed. Shown are long-term outcomes including the negative impact of a complicating event. Events were defined as relapse (need for further immunosuppression after protocol treatment) and clonal evolution (myelodysplasia/acute myeloid leukemia; almost always accompanied by a new cytogenetic abnormality in the BM). Approximately half of the patients did not experience a clonal event and poor survival was largely a consequence of disease progression. Data were censored for transplantation.
• #21: 关于免疫抑制治疗的疗效，国内外的报道不尽相同，但大约都在70-80%之间。右图显示在一个大样本的研究中，应用马ATG联合环孢素的标准免疫抑制治疗重再患者的生存率，没有并发症的患者10年生存率为95%，而发生并发症事件的10年生存率则为65%。 A large cohort (N=243) of NIH patients who responded to treatment with the standard regimen of horse ATG plus cyclosporine was analyzed. Shown are long-term outcomes including the negative impact of a complicating event. Events were defined as relapse (need for further immunosuppression after protocol treatment) and clonal evolution (myelodysplasia/acute myeloid leukemia; almost always accompanied by a new cytogenetic abnormality in the BM). Approximately half of the patients did not experience a clonal event and poor survival was largely a consequence of disease progression. Data were censored for transplantation.
• #22: 近十年基于马ATG的免疫抑制治疗SAA的疗效结果，最右侧显示了这些研究的总体生存率。 PRCT, prospective randomized clinical trial, POLCT, prospective open-label clinical trial, RETR, retrospective study; GITMO, Gruppo Italiano Trapianto di Midollo Osseo; EBMT, European Group for Blood and Marrow Transplantation; NIH, National Institutes of Health; ATG, antithymocyte globulin; CyA, ciclosporin A; G-CSF, granulocyte colony-stimulating factor. Age: median age (years). Response: overall response rate. Clonal evol.: evolution to myelodysplastic syndrome/acute myeloid leukaemia (paroxysmal nocturnal haemoglobinuria). Survival: overall survival rate. *PRCT ATG ± CyA; only data from the CyA arm are included. Twenty-seven of patients received more than one course of ATG. PRCT ATG + CyA ± G-CSF: data from both arms are included (No G-CSF/Yes G-CSF). §PRCT ATG + CyA ± MMF: data from both arms are merged. –PRCT ATG + CyA ± RAPA: data from both arms are merged.
• #23: FAA: fulminant aplastic anemia; vSAA: very severe aplastic anemia; SAA: severe aplastic anemia; FFS: Failure-free survival; OS: Overall survival. The 5-year FFS (a) and the 5-year OS (b) according to the severity of AA. The 5-year FFS was 48.2 % in the FAA group, 65.1 % in the vSAA group, and 68.1 % in the SAA group, respectively. However, no significant difference was demonstrated (p=0.206). The 5-year OS was comparable among the three groups: 88.5 % in the FAA group, 95.8 % in the vSAA group, and 96.8 % in the SAA group, respectively