PDF(Sci-hub)
Abstract：
The European Leukemia Net (ELN) guidelines for treatment of myelodysplastic syndromes (MDS) connect heterogeneous MDS subgroups with a number of therapeutic options ranging from best supportive care to allogeneic stem cell transplantation (alloSCT). However, it is currently unknown whether adherence to guideline recommendations translates into improved survival. The sizeable database of the Duesseldorf MDS Registry allowed us to address this question. We first performed a retrospective analysis including 1698 patients (cohort 1) to whom we retrospectively applied the ELN guidelines. We compared patients treated according to the guidelines with patients who deviated from it, either because they received a certain treatment though it was not recommended or because they did not receive that treatment despite being eligible. We also performed a prospective study with 381 patients (cohort 2) who were seen in our department and received guideline-based expert advice. Again, we compared the impact of subsequent guideline-adherent versus non-adherent treatment. For the majority of treatment options (best supportive care, lenalidomide, hypomethylating agents, low-dose chemotherapy, and intensive chemotherapy), we found that adherence to the ELN guidelines did not improve survival in cohort 1. The same was true when patient management was prospectively enhanced through guideline-based treatment advice given by MDS experts (cohort 2). The only exceptions were alloSCT and iron chelation (ICT). Patients receiving ICT and alloSCT as recommended fared significantly better than those who were eligible but received other treatment. Our analysis underscores the limited survival impact of most MDS therapies and suggests to pursue alloSCT in all suitable candidates. Graphical abstract.
摘要：
欧洲白血病网（ELN）治疗骨髓增生异常综合征（MDS）的指南将异质性MDS亚组与许多治疗选择联系起来，从最佳支持治疗到异基因干细胞移植（alloSCT）。然而,目前尚不清楚坚持指南建议是否能提高生存率.杜塞尔多夫MDS注册表的庞大数据库使我们能够解决这个问题。我们首先进行了一项回顾性分析，包括1698名患者（队列1），我们回顾性地应用了ELN指南。我们将根据指南治疗的患者与偏离指南的患者进行了比较，要么是因为他们接受了某种治疗，尽管不推荐，要么是因为他们没有接受这种治疗，尽管他们是合格的。我们还对381名患者（队列2）进行了一项前瞻性研究，这些患者在我们部门就诊并接受了基于指南的专家建议。再一次,我们比较了后续指南依从和非依从治疗的影响.对于大多数治疗选择(最佳支持治疗，来那度胺,低甲基化剂，低剂量化疗，和强化化疗)，我们发现,在队列1中,坚持ELN指南并不能改善生存率.当通过MDS专家提供的基于指南的治疗建议（队列2）前瞻性地加强患者管理时，情况也是如此。唯一的例外是alloSCT和铁螯合(ICT)。按照建议接受ICT和alloSCT的患者的表现明显优于符合条件但接受其他治疗的患者。我们的分析强调了大多数MDS疗法对生存的影响有限，并建议在所有合适的候选人中采用alloSCT。图形抽象。
