传统学说认为，在一定遗传背景下，再障作为一组异质性“综合征”可能通过三种机制发病：原、继发性造血干/祖细胞（“种子”）缺陷、造血微环境（“土壤”）及免疫（“虫子”）异常。目前认为T淋巴细胞功能亢进在原发性获得性再障发病机制中占重要地位，再障是T淋巴细胞介导的以造血系统为靶器官的自身免疫性疾病。

临床表现。国际上，再障分为重、轻型，我国相应的分型是急性和慢性再障，主要临床表现为贫血、出血及感染。一般没有淋巴结及肝脾肿大。

治疗。再障患者输注红细胞和血小板对于维持血细胞计数是必需的。输血以能改善患者贫血症状，缓解缺氧状态为宜，无需将血红蛋白水平纠正至正常值。尽量输注输红细胞，全血无红细胞时可以考虑，具体量随病情而定。即使再障患者白细胞或/及血小板数减少，其贫血都应该输浓缩红细胞，而不是输全血。拟行异基因造血干细胞移植者应输注经辐照后的红细胞和血小板。

应尽量减少输血，延长输血间期，避免发生输血性血色病。预防出血除输注血小板外，使用保持口腔卫生、口服止血药物、雄激素激素控制月经等措施，也有所帮助。感染、出血、使用抗生素或抗胸腺细胞免疫球蛋白/抗淋巴细胞免疫球蛋白（ATG/ALG）等治疗。ATG治疗期间及治疗后是否一定要输注辐照血制品尚缺乏循证医学证据。

对于粒缺患者危及生命者可以输注白细胞，粒细胞输注辅助抗生素治疗可取得较好的疗效。造血生长因子仅使用G-CSF、EPO等造血生长因子对再障患者行促造血治疗，临床无显著效果，因此而延误免疫抑制治疗或骨髓移植等有效治疗手段很不值得。皮下注射G-CSF 5μg/kg/d，可能刺激骨髓中残留的粒细胞或者粒细胞的功能，但不推荐将GM-CSF应用于再障患者重症感染的治疗，因为其可能导致严重出血及其他严重毒性反应。G-CSF对造血干/祖细胞有动员作用，而造血细胞进入细胞周期后对免疫因素损伤敏感性增加，有鉴于此，长期大量使用G-CSF应在使用了足够的免疫抑制治疗的前提下。初步资料显示IST联合G-CSF或/和EPO治疗重型再障能够减少感染几率，最终提高生存率，值得进行多中心前瞻性对照研究。但是长期使用造血生长因子的安全性尚未建立。IL-11或TPO在再障患者中促进巨核细胞和血小板生长的作用仍有待证实。

氢气作为一种选择性抗氧化物质，证明对许多氧化应激损伤具有显著的治疗效果。氢气是否也可以治疗该疾病？来自海军总医院的作者钱立仁等曾发表观点论文文章提出，如果从感染、炎症反应、毒性发病机制等角度考虑，可能氢气对再障有一定治疗作用。最近来自第二军医大学的一项研究用动物模型证实了这一说法，虽然目前无法断定氢气对人类再障，但至少使人们朝着这一目标前进了一步。

Therapeutic Effects of Hydrogen-Rich Solution on Aplastic Anemia in Vivo. Cell Physiol Biochem. 2013 Aug 30;32(3):549-560.

Zhao S, Mei K, Qian L, Yang Y, Liu W, Huang Y, Zhang C, Sun X, Liu C, Li B, Gao

F, Cai J, Ni J.

Department of Radiation Medicine, Second Military Medical University, Shanghai,

PR China.

Background: Aplasitc anemia (AA) is a bone marrow failure syndrome characterized by an immune-mediated destruction of hematopoietic stem cells. Though clinical symptoms could be ameliorated by bone marrow transplantation and/or immunosuppressive therapy, frequent recurrence and especially evolution of clonal hematologic diseases remains problematic clinically. Cytokines such as interferon-γ (INF-γ), tumor necrosis factor-α (TNF-α) and interleukin-6 (IL-6) secreted by autologous T cells are closely related with the development of AA. Hydrogen-rich solution was reported to inhibit the levels of cytokines including INF-γ, TNF-α and IL-6 in vivo in recent studies. This study was to investigate the potential therapeutic effects of hydrogen-rich solution on AA in vivo. Methods: AA model was determined in vivo by mice and body weights of the mice were used as the basic physiological index. Peripheral blood cells were calculated to evaluate the hematologic recovery degree. Bone marrow nucleated cells (BMNCs), tissue histology, as well as CFU-S and CFU-GM forming units were used to evaluate the recovery of bone marrow microenvironment. The ratio of CD4⁺ and CD8⁺ cells were examined along with cytokine levels in serum to determine the efficacy of H₂-rich solution on the affected immunological functions. Results: Body weight and number of peripheral blood cells were significantly improved for mice in the H₂-rich solution treated groups as compared with those with AA. The number of BMNCs and CFUs increased markedly and the bone marrow microenvironment was also improved significantly. The experimental group restrained the cell apoptosis, relieved hyperemia and accelerated tissue repair. The number of CD4⁺ and CD8⁺ cells as well as the ratio of CD4/CD8 increased to normal gradually, while the levels of TNF-α, IFN-γ, and IL-6 in serum decreased after H₂-rich solution treatment. Conclusion: Our study firstly showed that hydrogen-rich solution accelerated the recovery of either hematological or immunological recovery on aplastic anemia mice. This finding suggests hydrogen-rich solution as a potential clinical therapeutic agent for AA. © 2013 S. Karger AG, Basel.

http://www.karger.com/Article/FullText/354459