Department of Internal Medicine, Cleveland Clinic, 9500 Euclid Avenue, Cleveland, OH, 44195, USA.
Department of Leukemia, Cleveland Clinic Taussig Cancer Center, 9500 Euclid Avenue, CA-6, Cleveland, OH, 44195, USA.
Curr Hematol Malig Rep. 2020 Apr;15(2):149-154. doi: 10.1007/s11899-020-00562-9.
The lack of fully effective therapies to alter the natural course of myelodysplastic syndromes (MDS) leads to chronic morbidity, mortality and affects quality of life (QoL). Since existing therapies outside of hematopoietic cell transplantation (HCT) are not curative, there is a growing interest in incorporating patient-reported outcomes (PROs) as meaningful endpoints for these patients in research and clinical practice.
Currently, there are limited numbers of studies reporting the impact of MDS therapeutics on PROs to guide clinical decision-making and increase patient satisfaction. However, clinical trials that have incorporated QoL outcomes have demonstrated positive results with the use of growth factors, hypomethylating agents, and lenalidomide. Here we review and highlight the importance of harnessing the power of PROs as part of a comprehensive efficacy evaluation to ultimately deliver superior patient-centered care across the spectrum of MDS. As our understanding of MDS continues to increase, adapting the metrics of these outcome measurements will be equally important as the alteration of the natural history in developing new therapies.
由于缺乏能够改变骨髓增生异常综合征（MDS）自然病程的完全有效的治疗方法，导致该病患者慢性发病、死亡率高，并影响生活质量（QoL）。由于造血细胞移植（HCT）以外的现有治疗方法无法治愈该病，因此人们越来越感兴趣地将患者报告的结果（PROs）作为这些患者在研究和临床实践中的有意义的终点纳入其中。
目前，很少有研究报告 MDS 治疗方法对 PROs 的影响，以指导临床决策并提高患者满意度。然而，已经纳入生活质量结局的临床试验表明，使用生长因子、低甲基化剂和来那度胺具有积极的结果。在这里，我们回顾并强调了利用 PROs 作为综合疗效评估的一部分的重要性，以最终在 MDS 整个范围内提供以患者为中心的卓越护理。随着我们对 MDS 的认识不断提高，调整这些结局测量指标的衡量标准与改变新疗法的自然史同样重要。
