Deeg H Joachim, Jiang Peter Y Z, Holmberg Leona A, Scott Bart, Petersdorf Effie W, Appelbaum Frederick R
Division of Clinical Research, Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue N., D1-100, PO Box 19024, Seattle, WA 98109-1024, USA.
Leuk Res. 2004 Nov;28(11):1177-80. doi: 10.1016/j.leukres.2004.03.016.
Myelodysplastic syndrome (MDS) comprises a spectrum of heterogeneous diseases. Most patients present with ineffective hematopoiesis. The pathophysiology involves immune-mediated effects, cytokine dysregulation, and apoptosis, among others. We treated 14 transfusion-requiring patients with MDS, 10 with refractory anemia (RA) and four with RA with excess blasts (RAEB) with a 4-day course of antithymocyte globulin (ATG) followed by intermittent etanercept for 4 months. Among 13 evaluable patients, five are red blood cell and platelet transfusion independent for intervals extending beyond 2 years, and two have normalized their peripheral blood parameters. One additional patient showed a transient rise of platelet and neutrophil counts, for an overall response rate of 46%. Responding patients showed striking improvements in marrow cell abnormalities as characterized by flow cytometry. These data show that a combination of ATG plus etanercept offers effective palliative therapy for unselected patients with MDS. Further trials incorporating these two agents are warranted.
骨髓增生异常综合征（MDS）包含一系列异质性疾病。大多数患者存在无效造血。其病理生理学涉及免疫介导效应、细胞因子失调以及凋亡等。我们对14例需要输血的MDS患者进行了治疗，其中10例为难治性贫血（RA），4例为难治性贫血伴原始细胞增多（RAEB），给予4天疗程的抗胸腺细胞球蛋白（ATG）治疗，随后间断使用依那西普4个月。在13例可评估患者中，5例在超过2年的时间里无需输注红细胞和血小板，2例外周血参数恢复正常。另有1例患者血小板和中性粒细胞计数短暂升高，总体缓解率为46%。缓解患者的骨髓细胞异常通过流式细胞术检测显示有显著改善。这些数据表明，ATG联合依那西普为未选择的MDS患者提供了有效的姑息治疗。有必要开展纳入这两种药物的进一步试验。
