Institute of Public Health, Medical Decision Making, and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics, and Technology, Hall in Tirol, Austria.
Department of Internal Medicine V (Hematology and Oncology), Innsbruck Medical University, Innsbruck, Austria.
Blood Adv. 2022 Jan 11;6(1):1-12. doi: 10.1182/bloodadvances.2021004568.
Patient-reported outcomes (PROs) are relevant and valuable end points in the care of patients with myelodysplastic syndromes (MDS). However, a consensus-based selection of PROs for MDS, derived by both patients and hematologists, is lacking. We aimed to develop a core set of PROs for patients with MDS as part of the prospective European LeukemiaNet MDS (EUMDS) Registry. According to international guidelines, candidate PROs were identified from a comprehensive literature search of MDS studies. Overall, 40 PROs were selected and evaluated in a two-round Delphi survey by 40 patients with MDS and 38 hematologists in the first round and 38 patients and 32 hematologists in the second round. Based on an agreement scale and predefined inclusion criteria, both patients and hematologists selected "general quality of life" as a core PRO. Hematologists also selected "transfusion-dependency burden" and "ability to work/activities of daily living" as core PROs. The second Delphi round increased PRO rating agreements. Statistically significant rating differences between patients and hematologists were observed for 28 PROs (Mann-Whitney U test; P < .05) in the first round and for 19 PROs in the second round, with "disease knowledge" and "confidence in health care services" rated notably higher by patients. The overall mean PRO ratings correlation between the 2 groups was moderate (Spearman's rank correlation coefficient = 0.5; P < .05). This first consensus on a core set of PROs jointly developed by patients and hematologists forms the basis for patient-centered care in daily practice and clinical research.
患者报告的结局（PROs）是骨髓增生异常综合征（MDS）患者治疗中相关且有价值的终点。然而，缺乏患者和血液学家共同选择的 MDS 患者 PRO 共识。我们旨在作为前瞻性欧洲白血病网 MDS（EUMDS）注册研究的一部分，为 MDS 患者制定一组核心 PRO。根据国际指南，从 MDS 研究的全面文献搜索中确定了候选 PRO。总体而言，在第一轮中，40 名 MDS 患者和 38 名血液学家对 40 个 PRO 进行了两轮 Delphi 调查，并在第二轮中对 38 名患者和 32 名血液学家进行了评估。根据一致尺度和预定的纳入标准，患者和血液学家均选择“一般生活质量”作为核心 PRO。血液学家还选择“输血依赖负担”和“工作/日常生活能力”作为核心 PRO。第二轮 Delphi 增加了 PRO 评分的一致性。在第一轮中，患者和血液学家对 28 个 PRO（Mann-Whitney U 检验；P <.05）的评分存在统计学显著差异，在第二轮中对 19 个 PRO 的评分存在差异，患者对“疾病知识”和“对医疗服务的信心”的评分明显更高。两组之间总体平均 PRO 评分相关性中等（Spearman 秩相关系数= 0.5；P <.05）。这是患者和血液学家共同制定的一组核心 PRO 的首次共识，为日常实践和临床研究中的以患者为中心的护理奠定了基础。
