Department of Hematology, Atomic Bomb Disease Institute, Nagasaki University, 1-12-4 Sakamoto, Nagasaki, Nagasaki, 852-8523, Japan,
Int J Hematol. 2013 Dec;98(6):687-93. doi: 10.1007/s12185-013-1468-8. Epub 2013 Nov 20.
To investigate the long-term usefulness of immunosuppressive therapy (IST) for Japanese patients with lower-risk myelodysplastic syndromes, we retrospectively analyzed 29 MDS patients who were treated with cyclosporine A alone or with anti-thymocyte globulin at a single institute in Japan. A total of 58.6 % of patients showed hematological response to IST. Overall survival of all patients was 74.5 % at 5 years and 48.3 % at 10 years. The major adverse event was the elevation of creatinine level (grade 1 and 2). Eleven patients were still on IST at the time of analysis with, at least, some clinical benefits. Pneumonia was the most frequent cause of death (eight of 12 deaths), followed by bleeding (three of 12); most of the patients who died were non-responders. The presence of paroxysmal nocturnal hemoglobinuria-type cells was significantly associated with both response to IST and long-term survival by univariate analysis. The 10-year overall survival of responders (72.2 %) was significantly superior to that of non-responders (15.6 %, P < 0.0001). These results suggest that IST using cyclosporine A provides long-term benefit for Japanese patients with lower-risk MDS.
为了研究免疫抑制疗法（IST）对日本低危骨髓增生异常综合征（MDS）患者的长期疗效，我们回顾性分析了在日本一家单中心接受环孢素 A 单药或抗胸腺细胞球蛋白治疗的 29 例 MDS 患者。IST 治疗后，58.6%的患者出现血液学反应。所有患者的 5 年总生存率为 74.5%，10 年总生存率为 48.3%。IST 的主要不良反应为血肌酐升高（1 级和 2 级）。分析时，仍有 11 例患者继续接受 IST 治疗，至少有部分临床获益。导致死亡的最常见原因是肺炎（12 例死亡中的 8 例），其次是出血（12 例死亡中的 3 例）；大多数死亡患者为无反应者。单因素分析显示，阵发性睡眠性血红蛋白尿样细胞的存在与 IST 反应和长期生存均显著相关。IST 治疗后有反应者的 10 年总生存率（72.2%）显著优于无反应者（15.6%，P<0.0001）。这些结果表明，环孢素 A 诱导的 IST 可为日本低危 MDS 患者带来长期获益。
