Division of Cancer Epidemiology and Genetics, National Cancer Institute, Rockville, MD 20892, United States.
Leuk Res. 2013 Jul;37(7):848-51. doi: 10.1016/j.leukres.2013.04.013. Epub 2013 May 1.
Aplastic anemia (AA), myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are rare disorders of bone marrow failure. Once considered distinct entities, these three diseases are now believed to have overlapping pathophysiologies. The Aplastic Anemia and MDS International Foundation, a nonprofit organization that supports patients and families living with bone marrow failure disorders, sponsored a scientific symposium in Bethesda, MD, in March 2012. This report summarizes the symposium presentations by 30 of the world's leading AA, MDS, and PNH researchers on recent findings, current areas of controversy, and recommendations for basic and clinical research to advance the field.
再生障碍性贫血(AA)、骨髓增生异常综合征(MDS)和阵发性睡眠性血红蛋白尿症(PNH)是罕见的骨髓衰竭疾病。这三种疾病曾经被认为是不同的实体,但现在认为它们具有重叠的病理生理学。再生障碍性贫血和 MDS 国际基金会是一个支持患有骨髓衰竭疾病的患者和家庭的非营利组织,于 2012 年 3 月在马里兰州贝塞斯达举办了一次科学研讨会。本报告总结了 30 位世界领先的 AA、MDS 和 PNH 研究人员在最近的发现、当前争议领域以及推进该领域的基础和临床研究建议方面的研讨会演讲。