A nationwide survey of hypoplastic myelodysplastic syndrome (a multicenter retrospective study)
A nationwide survey of hypoplastic myelodysplastic syndrome (a multicenter retrospective study)
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全国发育不全性骨髓增生异常综合征调查（多中心回顾性研究）
DOI：
10.1002/ajh.24905
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发表时间：
2017
影响因子：
12.8
通讯作者：
et al
中科院分区：
文献类型：
--
作者：
Kobayashi T;Nannya Y;Ichikawa M;Oritani K;Kanakura Y;Tomita A;Kiyoi H;Kobune M;Kato J;Kawabata H;Shindo M;Torimoto Y;Yonemura Y;Tohyama K;et al
骨髓发育不良综合征(HMDS)是一种特殊的疾病，具有骨髓(BM)细胞减少和骨髓衰竭(BMF)死亡的风险。为了阐明HMDS的特征，收集了2003年4月至2012年3月期间确诊的129例HMDS患者的数据，来自20个机构和国家特发性骨髓衰竭综合征研究小组中央审查小组，并与115例非HMDS患者进行了比较。FAB中RA较多，CMMoL、RAEB-t较少，世界卫生组织(WHO)分类中RCUD、MDS-U较多，RCMD较少，差异有统计学意义。HMDs患者的总生存期(OS)和无进展生存期(PFS)均高于非HMDs患者，尤其是年龄在≥50岁且修订的国际预后评分系统(IPSS-R)风险较低的患者。在竞争性风险分析中，在IPSS或IPSS-R风险较低的患者中，HMD显示出急性髓细胞白血病进展的风险降低，而在≥50岁的患者中，死于BMF的风险更高。在COX比例风险分析中，表现状态差(PS≥2)和IPSS-R的高核型风险(高和极高)是死亡和急性髓系白血病进展的重要危险因素。
Hypoplastic myelodysplastic syndrome (hMDS) is a distinct entity with bone marrow (BM) hypocellularity and the risk of death from BM failure (BMF). To elucidate the characteristics of hMDS, the data of 129 patients diagnosed between April 2003 and March 2012 were collected from 20 institutions and the central review team of the National Research Group on Idiopathic Bone Marrow Failure Syndromes, and compared with 115 non‐hMDS patients. More RA and fewer CMMoL and RAEB‐t in French‐American‐British (FAB) and more RCUD and MDS‐U and fewer RCMD in World Health Organization (WHO) classifications were found in hMDS than non‐hMDS with significant differences. The overall survival (OS) and AML progression‐free survival (AML‐PFS) of hMDS were higher than those of non‐hMDS, especially in patients at age ≥50 and of lower risk in Revised International Prognostic Scoring System (IPSS‐R). In competing risks analysis, hMDS exhibited decreased risk of AML‐progression in lower IPSS or IPSS‐R risk patients, and higher risk of death from BMF in patients at age ≥50. Poor performance status (PS ≥2) and high karyotype risks in IPSS‐R (high and very high) were significant risk factors of death and AML‐progression in Cox proportional hazards analysis.