Cazzola M
Division of Hematology, University of Pavia School of Medicine, IRCCS Policlinico S. Matteo, Italy.
Int J Hematol. 2000 Aug;72(2):134-8.
At present, the only 2 treatments that can prolong survival in patients with myelodysplastic syndrome (MDS) are allogeneic stem cell transplantation and intensive chemotherapy. Alternatives to myeloablative or conventional chemotherapy include: (1) supportive therapy, (2) stimulation of normal residual hematopoietic progenitors, and (3) manipulation of myelodysplastic hematopoiesis. These alternative therapeutic strategies can be accomplished using various therapeutic tools. Supportive therapy remains the mainstay in the management of MDS patients and desferrioxamine should be administered to individuals who have a regular need for blood transfusion. The only hematopoietic growth factors that can be useful in the treatment of selected MDS patients are recombinant human erythropoietin (rhEpo) and granulocyte colony-stimulating factor (G-CSF). Overall, 15% to 20% of patients with MDS respond to rhEpo treatment. Factors predicting response include serum erythropoietin levels <100 to 200 mU/mL, low-risk MDS, and no or low need for transfusion. G-CSF alone should be used only for short-term treatments during severe infection episodes that do not respond to conventional therapy. About 40% of MDS patients respond to a combined treatment of rhEpo plus G-CSF with amelioration of anemia. Cytoprotective antiapoptotic agents such as amifostine, alone or in combination, may improve blood values in occasional MDS patients. MDS patients with immunologically mediated myelosuppression may respond favorably to antithymocyte globulin or cyclosporin A (CyA). Although rhEpo and CyA may be used in individual patients who appear likely to respond, the remaining therapeutic tools must be considered strictly experimental; phase III clinical trials are required to establish whether they can be useful in the treatment of MDS patients. More generally, because of the current uncertainties concerning MDS treatment, participation of patients in clinical trials should be always encouraged.
目前，骨髓增生异常综合征（MDS）患者中仅有的两种可延长生存期的治疗方法是异基因干细胞移植和强化化疗。清髓性或传统化疗的替代方法包括：（1）支持性治疗，（2）刺激正常残留造血祖细胞，以及（3）调控骨髓增生异常的造血过程。这些替代治疗策略可通过各种治疗手段实现。支持性治疗仍是MDS患者管理的主要手段，对于有定期输血需求的个体应给予去铁胺。在选定的MDS患者治疗中可能有用的唯一造血生长因子是重组人促红细胞生成素（rhEpo）和粒细胞集落刺激因子（G-CSF）。总体而言，15%至20%的MDS患者对rhEpo治疗有反应。预测反应的因素包括血清促红细胞生成素水平<100至200 mU/mL、低危MDS以及无输血需求或低输血需求。G-CSF仅应在对传统治疗无反应的严重感染发作期间用于短期治疗。约40%的MDS患者对rhEpo加G-CSF的联合治疗有反应，贫血症状得到改善。细胞保护抗凋亡药物如氨磷汀单独或联合使用，可能会使部分MDS患者的血液指标得到改善。具有免疫介导的骨髓抑制的MDS患者可能对抗胸腺细胞球蛋白或环孢素A（CyA）反应良好。虽然rhEpo和CyA可用于可能有反应迹象的个体患者，但其余治疗手段必须被视为严格意义上的实验性方法；需要进行III期临床试验来确定它们是否可用于MDS患者的治疗。更普遍地说，由于目前MDS治疗存在不确定性，应始终鼓励患者参与临床试验。
