Functional Unit of Haematology, AOU Careggi, University of Florence, Largo Brambilla 3, Firenze, Italy.
Oncologist. 2011;16 Suppl 3:35-42. doi: 10.1634/theoncologist.2011-S3-35.
Myelodysplastic syndromes (MDS) are heterogeneous clonal diseases characterized by cytopenias resulting from ineffective hemopoiesis. Anemia affects the vast majority of patients with MDS and contributes substantially to their symptoms. For more than 20 years, recombinant human erythropoietin has been available for clinical use, and it has been employed in an attempt to relieve MDS-related anemia. Erythropoietin-alpha, erythropoietin-beta, and more recently darbepoetin have been found to increase hemoglobin levels and abolish transfusion dependence in 19%-68% of MDS cases. This wide range in clinical response depends on several biological and clinical variables that allow the selection of patients with the highest probability of successful treatment. These agents are a mainstay in MDS therapy, but many issues are still open in terms of the initiation of therapy, the optimal dosage of erythropoietic stimulating agents (ESAs), the most efficient type of ESA, and the duration and outcome of such treatments. In this review, the mechanisms of response and predictive factors as well as an analysis of the clinical activity of ESAs in MDS therapy are presented.
骨髓增生异常综合征（MDS）是一组异质性克隆性疾病，其特征为无效造血导致的血细胞减少。贫血影响绝大多数 MDS 患者，并对其症状有重大影响。二十多年来，重组人红细胞生成素已可用于临床应用，并被用于尝试缓解 MDS 相关贫血。Epoetin-α、Epoetin-β 和最近的达贝泊汀已被发现可使 19%-68%的 MDS 病例的血红蛋白水平升高并消除输血依赖。这种广泛的临床反应取决于几个生物学和临床变量，这些变量允许选择最有可能成功治疗的患者。这些药物是 MDS 治疗的主要方法，但在治疗的起始、红细胞生成刺激剂（ESA）的最佳剂量、最有效的 ESA 类型以及此类治疗的持续时间和结果方面，仍有许多问题悬而未决。在这篇综述中，介绍了 ESA 在 MDS 治疗中的反应机制和预测因素以及临床活性分析。
