Core set of patient-reported outcomes for myelodysplastic syndromes: an EUMDS Delphi study involving patients and hematologists.
Core set of patient-reported outcomes for myelodysplastic syndromes: an EUMDS Delphi study involving patients and hematologists.
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DOI：
10.1182/bloodadvances.2021004568
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发表时间：
2022-01-11
期刊：
影响因子：
7.5
通讯作者：
Stauder R
中科院分区：
文献类型：
--
作者：
Stojkov I;Conrads-Frank A;Rochau U;Koinig KA;Arvandi M;Puntscher S;van Marrewijk C;Fenaux P;Symeonidis A;Chermat F;Garelius H;Bowen D;Mittelman M;Mora E;de Witte T;Efficace F;Siebert U;Stauder R
系统地开发了MDS的一套核心优点，涉及患者和血液学家进行了2轮德尔菲调查。核心PROs将支持统一的结果测量，并促进在MDS疾病护理中纳入可靠的患者信息。患者报告的结局（PROs）是骨髓增生异常综合征（MDS）患者护理中相关且有价值的终点。然而，缺乏由患者和血液学家得出的基于共识的MDS PROs选择。我们的目标是为MDS患者开发一套核心的PROs，作为欧洲白血病网MDS （EUMDS）注册的一部分。根据国际指南，从MDS研究的综合文献检索中确定候选PROs。总体而言，通过两轮德尔菲调查，40名MDS患者和38名血液学家在第一轮和38名患者和32名血液学家中选择并评估了40名PROs。基于一致性量表和预定义的纳入标准，患者和血液学家都选择“一般生活质量”作为核心PRO。血液学家还选择了“输血依赖负担”和“工作能力/日常生活活动”作为核心PROs。第二轮德尔福融资增加了PRO评级协议。第一轮有28名专业医师与血液科医师的评分差异有统计学意义（Mann-Whitney U检验；P < 0.05），第二轮有19名专业医师的评分差异有统计学意义，其中患者对“疾病知识”和“卫生保健服务信心”的评分明显较高。两组间总体平均PRO评分相关性为中等（Spearman’s rank相关系数= 0.5;P < 0.05）。这是由患者和血液学家共同制定的一套核心pro的第一个共识，为日常实践和临床研究中以患者为中心的护理奠定了基础。
Systematically developed set of core PROs in MDS, involving patients and hematologists in a 2-round Delphi survey. Core PROs will support unified outcome measurement and facilitate inclusion of reliable patient information in MDS disease care. 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.
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影响因子：
2.7
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通讯作者：
Efficace, F.
影响因子：
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作者：
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