Oliva Esther Natalie, Guo Shien, Lord-Bessen Jennifer, Yucel Aylin, Latagliata Roberto, Breccia Massimo, Palumbo Giuseppe A, Sanpaolo Grazia, Riva Marta, Santini Valeria, Platzbecker Uwe, Garcia-Manero Guillermo, Fenaux Pierre, Pelligra Christopher G
Hematology Unit, Grande Ospedale Metropolitano Bianchi Melacrino Morelli, Reggio Calabria, Italy.
Evidera, Waltham, MA, United States.
Front Oncol. 2025 Feb 7;15:1507854. doi: 10.3389/fonc.2025.1507854. eCollection 2025.
Myelodysplastic neoplasms (MDS) are characterized by ineffective hematopoiesis, peripheral blood cytopenias, and an increased risk of progression to acute myeloid leukemia. One of the main treatment goals is improving quality of life (QoL), particularly for patients with lower-risk MDS (LR-MDS) who may live longer with compromised QoL. The QOL-E is a patient-reported outcome (PRO) measure specifically developed to address the lack of a health-related QoL questionnaire for patients with MDS. The objective of this study was to evaluate the psychometric performance of the QOL-E in patients with LR-MDS.
Data from four clinical trials in MDS (MEDALIST, DARB-MDS, EQoL-MDS, and RevMDS trials) were used to assess construct validity, reliability, and responsiveness. The QOL-E was validated by the European Organization for the Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 (QLQ-C30) and clinical outcomes. It contains 29 items with the first two items assessing the patient's general well-being and the 27 remaining items grouped into six domain scores: physical well-being (QOL-FIS), functional well-being (QOL-FUN), social/family well-being (QOL-SOC), sexual well-being (QOL-SEX), fatigue (QOL-FAT), and MDS-specific disturbances (QOL-MDSS). Additionally, meaningful within-patient change (MWPC) thresholds were determined for the domains and summary scores of the QOL-E using anchor-based analyses, supported by distribution-based analyses.
A total of 458 patients were included in the analyses. The QOL-E domain/summary scores demonstrated acceptable convergent/divergent and known-groups validity. Test-retest reliability and internal consistency was confirmed with intraclass correlation coefficients and Cronbach alpha exceeding 0.70 across most QOL-E domains/summary scores. The QOL-E domains/summary scores, except for QOL-SEX, had an adequate ability to detect change from baseline to Week 24. MWPC thresholds were proposed for all other domains and summary scores.
The study results demonstrate that the QOL-E is generally fit for purpose to assess treatment effects in populations with LR-MDS and the proposed MWPC thresholds can be used to assess within-patient treatment effect on PROs, as assessed by the QOL-E, in future studies.
骨髓增生异常综合征（MDS）的特征为造血无效、外周血细胞减少以及进展为急性髓系白血病的风险增加。主要治疗目标之一是改善生活质量（QoL），对于低危MDS（LR-MDS）患者而言尤其如此，他们可能在生活质量受损的情况下存活更长时间。QOL-E是一项专门为解决MDS患者缺乏与健康相关的生活质量问卷这一问题而开发的患者报告结局（PRO）指标。本研究的目的是评估QOL-E在LR-MDS患者中的心理测量性能。
来自四项MDS临床试验（MEDALIST、DARB-MDS、EQoL-MDS和RevMDS试验）的数据用于评估结构效度、信度和反应度。QOL-E通过欧洲癌症研究与治疗组织（EORTC）生活质量问卷核心30项（QLQ-C30）和临床结局进行验证。它包含29个条目，前两个条目评估患者的总体健康状况，其余27个条目分为六个领域得分：身体状况（QOL-FIS）、功能状况（QOL-FUN）、社会/家庭状况（QOL-SOC）、性健康状况（QOL-SEX）、疲劳（QOL-FAT）和MDS特异性干扰（QOL-MDSS）。此外，在基于分布分析的支持下，使用基于锚定的分析确定了QOL-E各领域和总分的有意义的患者内变化（MWPC）阈值。
共有458例患者纳入分析。QOL-E领域/总分显示出可接受的聚合/区分效度和已知组效度。通过组内相关系数和Cronbach α系数证实了重测信度和内部一致性，在大多数QOL-E领域/总分中超过0.70。除QOL-SEX外，QOL-E领域/总分有足够的能力检测从基线到第24周的变化。为所有其他领域和总分提出了MWPC阈值。
研究结果表明，QOL-E总体上适用于评估LR-MDS人群的治疗效果，并且在未来研究中，所提出的MWPC阈值可用于评估QOL-E所评估的患者内治疗对PROs的影响。
