Section of Hematology, Department of Internal Medicine, Yale University School of Medicine, New Haven, CT, USA.
Yale Cancer Center, New Haven, CT, USA.
Expert Rev Hematol. 2020 May;13(5):447-460. doi: 10.1080/17474086.2020.1744433. Epub 2020 Mar 29.
: The myelodysplastic syndromes (MDS) vary in their risk of disease progression; progression includes increasingly severe bone marrow failure, reclassification as acute myeloid leukemia (AML), and death. Prognostic tools guide recommendations for allogeneic stem cell transplantation (alloSCT), the only curative option. AlloSCT is typically reserved for patients with higher-risk MDS as defined by existing prognostic tools, although additional clinical and biological factors in lower-risk patients may influence this dogma.: This review discusses the current understanding of MDS risk stratification as it pertains to the use of alloSCT in subpopulations of MDS patients with a particular focus on the use of alloSCT in patients with lower-risk disease.: Though high-quality data are lacking, some lower-risk MDS patients may benefit from alloSCT, which offers the only prospect of cure. Understanding the etiologic role and prognostic impact of recurring genetic events may improve existing risk stratification and become integral facets of prognostic schemata. The identification of additional factors influencing the prognoses of patients currently lumped together as 'lower-risk' will likewise improve the selection of MDS patients for early intervention or aggressive therapies such as alloSCT.
骨髓增生异常综合征（MDS）在疾病进展的风险上存在差异；进展包括骨髓衰竭逐渐加重、重新分类为急性髓系白血病（AML）和死亡。预后工具指导异基因造血干细胞移植（alloSCT）的建议，alloSCT 是唯一的治愈选择。alloSCT 通常保留给现有预后工具定义的高危 MDS 患者，尽管低危患者的其他临床和生物学因素可能影响这一观念。
本综述讨论了 MDS 风险分层的当前理解，因为它与在 MDS 患者亚群中使用 alloSCT 有关，特别是在低危疾病患者中使用 alloSCT 的情况。
虽然缺乏高质量的数据，但一些低危 MDS 患者可能从 alloSCT 中受益，alloSCT 提供了唯一的治愈前景。了解反复发生的遗传事件的病因作用和预后影响可能会改善现有的风险分层，并成为预后方案的重要组成部分。确定影响当前被归类为“低危”的患者预后的其他因素也将改善对 MDS 患者进行早期干预或积极治疗（如 alloSCT）的选择。
