How I treat acquired aplastic anemia
How I treat acquired aplastic anemia
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DOI：
10.1182/blood-2016-08-693481
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发表时间：
2017-03-16
期刊：
影响因子：
20.3
通讯作者：
Bacigalupo, Andrea
中科院分区：
文献类型：
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作者：
Bacigalupo, Andrea
获得性重型再生障碍性贫血（SAA）是一种罕见的血液病，与显着的发病率和死亡率。造血干细胞的免疫破坏在发病机制中起着重要作用，如免疫抑制剂的成功治疗所示，导致一定比例的患者不依赖输血或外周血计数完全恢复。生长因子可以与免疫抑制治疗（IST）相结合，并可能提高反应率，最近与血小板生成素类似物。合成代谢类固醇与IST结合仍可能发挥作用。IST的问题是无法响应和晚期克隆性疾病的发展。骨髓移植（BMT）是另一种治疗选择：匹配的同胞供体仍然是最佳选择。对于缺乏匹配的家庭供体的患者，可以很容易地找到不相关的供体，尽管大多数患者是白人。其他BMT选择包括无关的脐带血或不匹配的家庭供体。急性和慢性移植物抗宿主病仍然是骨髓移植的重要并发症.患者年龄是IST和BMT结果的强有力预测因素，在设计治疗策略时必须考虑。早期诊断和治疗以及长期监测仍然是成功治疗SAA的关键步骤。
Acquired severe aplastic anemia (SAA) is a rare hematologic disease associated with significant morbidity and mortality. Immune destruction of hemopoietic stem cells plays an important role in pathogenesis, as shown by successful treatment with immunosuppressive agents, leading to transfusion independence or complete recovery of peripheral blood counts in a proportion of patients. Growth factors can be combined with immunosuppressive therapy (IST) and may improve response rates, as recently shown with thrombopoietin analogs. Anabolic steroids may still play a role in combination with IST. The problem with IST is failure to respond and the development of late clonal disorders. Bone marrow transplantation (BMT) is the other therapeutic option: a matched sibling donor remains the best choice. For patients lacking a matched family donor, unrelated donors can be readily found, although mostly for patients of Caucasian origin. Other BMT options include unrelated cord blood or mismatched family donors. Acute and chronic graftversus- host disease remain important complications of BMT. Patient age is a strong predictor of outcome for both IST and BMT, and must be considered when designing therapeutic strategies. Early diagnosis and treatment, as well as long-term monitoring, remain crucial steps for successful treatment of SAA.