Giesinger Johannes M, La Nasa Giorgio, Sparano Francesco, Angermeyer Matthias, Morelli Emanuela, Mulas Olga, Efficace Fabio, Caocci Giovanni
Medical University of Innsbruck, University Hospital of Psychiatry II, Innsbruck, Austria.
Ematologia e CTMO, Ospedale Businco, ARNAS "G. Brotzu", Cagliari, Italy.
Clin Pract Epidemiol Ment Health. 2021 Dec 31;17(1):307-314. doi: 10.2174/1745017902117010307. eCollection 2021.
Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis and blood cytopenia with a variable risk of progression to acute myeloid leukemia. The main goal of therapy for the large majority of patients is to improve health-related quality of life (HRQoL). Its rigorous assessment is now recommended in international MDS guidelines. Our review provides an overview of HRQoL results from randomized controlled trials (RCTs) in MDS patients. The literature search undertaken in PubMed identified 10 RCTs with HRQoL endpoints (all secondary) published between August 2008 and September 2020. These RCTs have helped to better understand the impact of therapies from the patient perspective and have generated valuable information that can be used to further support clinical decisions. However, the number of RCTs in MDS patients, including HRQoL endpoints, is still low. Given the importance of symptom relief and HRQoL improvement in the treatment of MDS patients, the assessment of the patient perspective in future RCTs is highly recommended to keep expanding the knowledge of the impact of new MDS therapies.
骨髓增生异常综合征（MDS）的特征是造血无效和血细胞减少，进展为急性髓系白血病的风险各异。绝大多数患者的主要治疗目标是改善健康相关生活质量（HRQoL）。国际MDS指南现推荐对其进行严格评估。我们的综述概述了MDS患者随机对照试验（RCT）中的HRQoL结果。在PubMed上进行的文献检索确定了2008年8月至2020年9月期间发表的10项有HRQoL终点（均为次要终点）的RCT。这些RCT有助于从患者角度更好地理解治疗的影响，并产生了可用于进一步支持临床决策的有价值信息。然而，包括HRQoL终点在内的MDS患者RCT数量仍然较少。鉴于缓解症状和改善HRQoL在MDS患者治疗中的重要性，强烈建议在未来的RCT中评估患者的观点，以不断扩展对新MDS疗法影响的认识。
