Diagnosis and Management of Aplastic Anemia
Diagnosis and Management of Aplastic Anemia
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DOI：
10.1182/asheducation-2011.1.76
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发表时间：
2011-12-01
影响因子：
3
通讯作者：
Guinan, Eva C.
中科院分区：
文献类型：
--
作者：
Guinan, Eva C.
再生障碍性贫血仍然是一种排除性诊断。我们可靠地诊断并因此排除各种具有相似表型的遗传性或获得性疾病的能力已显著提高。一个有效的诊断计划是重要的，因为从诊断到治疗的时间与结果有关，无论选择何种治疗方案。HSCT仍然是那些有匹配的同胞供体的患者的主要治疗方法，近年来结果进一步改善。对于那些没有同胞供体的患者，联合免疫抑制治疗（IST）的高应答率和总生存率已被证明是稳健的。尽管如此，不完全缓解、复发和进展为骨髓增生异常/白血病已更清楚地成为重要的长期问题。替代供体移植结果的改善以及已建立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替代方案。这方面的最佳做法尚未明确确立，可能因各种人口统计和治疗特定因素而异。无论治疗方法的类型如何，患者都需要持续监测疾病和/或治疗相关副作用的发生。
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.