将病理生理异常综合征 (MDS) 患者的健康相关生活质量 (HRQoL) 视为治疗的重要目标，然而，在临床初诊时，关于这些患者的HRQoL几乎没有了解。我们报告了新诊断MDS患者的HRQoL情况，按照国际预后评分系统 (IPSS) 和IPSS-修订版 (IPSS-R) 分类，根据性别和年龄组别划分，旨在为这些患者建立欧洲肿瘤研究与治疗组织生活质量问卷-核心30 (EORTC QLQ-C30) 的参考值。基于927名患者的分析，中位年龄为73.3岁 (66.0-79.2)，其中506名患有低风险疾病，421名患有高风险疾病，根据IPSS分类。使用EORTC QLQ-C30评估HRQoL，并观察到年龄组别和性别之间以及低风险和高风险疾病之间的显著差异。例如，在高风险疾病患者中，年龄最小的组 (即30-59岁) 相对于年龄较大的组在各种功能和症状领域中倾向于报告临床意义上较差的结果。我们还开发了2个回归模型，用于预测根据IPSS或IPSS-R对患者进行分类的EORTC QLQ-C30参考分数。调查诊断时临床重要问题和症状的患病率显示了疾病的重大负担，超过50%的患者报告低风险和高风险疾病中的身体功能和呼吸困难存在临床重要问题。我们的研究结果有助于改善未来MDS研究中HRQoL结果的解释，并更好地从常规实践环境中对HRQoL数据进行评估。版权所有 © 2023 作者。由 Wolters Kluwer Health, Inc. 代表欧洲血液学协会出版。

Health-related quality of life (HRQoL) is an important goal of therapy for patients with myelodysplastic syndromes (MDS); however, little is known about HRQoL of these patients at clinical presentation. We report HRQoL profile of newly diagnosed patients with MDS across both the the International Prognostic Scoring System (IPSS) and IPSS-Revised (IPSS-R) classifications, stratified by sex and age group categories, aiming to also establish European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core30 (EORTC QLQ-C30) reference values for these patients. Analysis was based on 927 patients with a median age of 73.3 years (, 66.0-79.2), of whom 506 and 421 with lower- and higher-risk disease respectively, according to the IPSS classification. HRQoL was assessed with the EORTC QLQ-C30 and substantial differences by age groups and sex, between and within lower- and higher-risk disease categories were observed. For example, within higher-risk disease patients, the youngest group (ie, 30-59 years) tended to report clinically meaningful worse outcomes across various functional and symptom domains compared with older age groups. We also developed 2 regression models allowing for the prediction of EORTC QLQ-C30 reference scores for patients classified according to either the IPSS or the IPSS-R. Investigation of prevalence rates for clinically important problems and symptoms at diagnosis revealed a substantial burden of the disease with >50% of patients reporting clinically important problems with physical functioning and dyspnea in both lower- and higher-risk disease. Our findings may help to enhance the interpretation of HRQoL outcomes in future MDS studies and to better contextualize HRQoL data from routine practice settings.Copyright © 2023 the Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the European Hematology Association.