Dana-Farber Cancer Institute, Boston, MA 02215, USA.
Hematology Am Soc Hematol Educ Program. 2011;2011:76-81. doi: 10.1182/asheducation-2011.1.76.
Aplastic anemia remains a diagnosis of exclusion. Our ability to reliably diagnose, and therefore exclude, a variety of inherited or acquired diseases with similar phenotypes has improved markedly. An efficient diagnostic plan is important because time from diagnosis to treatment is related to outcome regardless of the therapeutic option chosen. HSCT remains the mainstay of therapy for those with matched sibling donors, and results have improved even further in recent years. For those without a sibling donor, the high response and overall survival rates of combined immunosuppressive therapy (IST) have proven robust. Nonetheless, incomplete response, relapse, and progression to myelodysplasia/leukemia have more clearly emerged as significant long-term issues. Improvements in outcome of alternative donor transplantation and the use of established and novel immunosuppressive agents provide multiple alternatives for treating refractory or relapsed patients. Best practices in this regard are not yet clearly established and may vary by a variety of demographic and treatment-specific factors. Regardless of the type of therapeutic approach, patients require ongoing monitoring for occurrence of disease and/or therapy-related side effects.
再生障碍性贫血仍然是一种排他性诊断。我们诊断和排除具有相似表型的各种遗传性或获得性疾病的能力有了显著提高。制定有效的诊断方案非常重要，因为无论选择何种治疗方案，从诊断到治疗的时间都与预后有关。对于有匹配的同胞供体的患者，HSCT 仍然是主要的治疗方法，近年来其治疗效果进一步提高。对于没有同胞供体的患者，联合免疫抑制治疗（IST）的高反应率和总生存率已经得到了充分证明。尽管如此，不完全缓解、复发和进展为骨髓增生异常/白血病更明显地成为了长期存在的重要问题。替代供体移植的治疗效果改善以及现有和新型免疫抑制剂的使用为治疗难治性或复发性患者提供了多种选择。在这方面的最佳实践尚未明确确立，可能因各种人口统计学和治疗特异性因素而有所不同。无论采用何种治疗方法，患者都需要持续监测疾病的发生和/或与治疗相关的副作用。
