Vitale A, Testi A M, Moleti M L, Vignetti M, Arcese W, Fenu S, Cedrone M, De Felice L, Amadori S, Mandelli F
Department of Human Biopathology, University La Sapienza, Rome, Italy.
Ann Hematol. 1994 May;68(5):241-5. doi: 10.1007/BF01737424.
Seven children with a primary myelodysplastic syndrome were seen at our center over a 9-year period. Two presented with refractory anemia, three with refractory anemia with excess of blasts, and two with refractory anemia with excess of blasts in transformation. All children received supportive therapy, including blood transfusions in five of them. Three patients developed acute myeloid leukemia and were treated with intensive chemotherapy, followed by allogeneic or autologous marrow transplantation in the two responders. All three died of either infection or progressive disease. The other four patients are still alive a median of 71 months (range 38-130) after diagnosis. These results confirm the difficulties in managing patients with myelodysplastic syndromes.
在9年的时间里，我们中心共接诊了7名原发性骨髓增生异常综合征患儿。其中2例表现为难治性贫血，3例为伴有过多原始细胞的难治性贫血，2例为转化中的伴有过多原始细胞的难治性贫血。所有患儿均接受了支持性治疗，其中5例接受了输血治疗。3例患者发展为急性髓系白血病并接受了强化化疗，2例缓解者随后接受了异基因或自体骨髓移植。3例均死于感染或疾病进展。其他4例患者在诊断后中位生存71个月（范围38 - 130个月），目前仍然存活。这些结果证实了管理骨髓增生异常综合征患者存在困难。
