Identification of patient-reported outcomes measures (PROMs) and patient-reported experiences measures (PREMs) in Gaucher disease in Spain

IF 2.6 4区医学 Q1 MEDICINE, GENERAL & INTERNAL

Medicina Clinica Pub Date : 2024-11-15 DOI:10.1016/j.medcli.2024.06.006

Pilar Giraldo , María Camprodón , Paloma Cerro Alcolea , Elena Gras-Colomer , Daiana Ibarretxe , Sunil Lakhwani , Elvira Mora , María Purificación Velasco Calderón , Montserrat Morales-Conejo

{"title":"Identification of patient-reported outcomes measures (PROMs) and patient-reported experiences measures (PREMs) in Gaucher disease in Spain","authors":"Pilar Giraldo , María Camprodón , Paloma Cerro Alcolea , Elena Gras-Colomer , Daiana Ibarretxe , Sunil Lakhwani , Elvira Mora , María Purificación Velasco Calderón , Montserrat Morales-Conejo","doi":"10.1016/j.medcli.2024.06.006","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><div>Patient-reported outcome measures (PROMs) and patient-reported experiences measures (PREMs) are crucial for understanding the impact of GD on quality of life and patient's perceptions on care, but also to guide decision-making processes. Nevertheless, no specific PREMs in GD have been published, neither PROMs for Spanish GD patients have been developed.</div></div><div><h3>Methods</h3><div>Two project coordinators selected key-points to be included in a PROMs/PREMs questionnaire, and the scientific committee and a group of expert patients contributed to the initial draft. Then, 9 meetings with experts were held to discuss controversial points. After, a questionnaire with 103 items regarding symptomatology, aspects of daily life and care experience was developed. Finally, it was conducted a Delphi survey among a multidisciplinary group of experts in GD.</div></div><div><h3>Results</h3><div>Consensus was reached on 85 out of the 103 items. Recommendations on PROMs and PREMs regarding symptomatology, aspects of daily life and care experience were obtained. Consensus was reached on the importance of considering fatigue, concentration problems, and communication issues in GD patients using 5-step analog scales. Panelists recommended asking GD patients about the impact on social functioning and work/school performance. Finally, consensus was reached on considering care experiences, such as treatment satisfaction, treatment interruptions or transitions and healthcare professionals involved in patient's management to perceive patient's perceptions.</div></div><div><h3>Conclusion</h3><div>This expert consensus may help developing GD-specific PROMs/PREMs for improving GD management. Properly developed and validated PROMs/PREMs may help decision-making, establishing patient-tailored therapeutic and follow-up goals.</div></div>","PeriodicalId":18578,"journal":{"name":"Medicina Clinica","volume":"163 9","pages":"Pages 449-457"},"PeriodicalIF":2.6000,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Medicina Clinica","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0025775324004524","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MEDICINE, GENERAL & INTERNAL","Score":null,"Total":0}

引用次数: 0

Abstract

Background

Patient-reported outcome measures (PROMs) and patient-reported experiences measures (PREMs) are crucial for understanding the impact of GD on quality of life and patient's perceptions on care, but also to guide decision-making processes. Nevertheless, no specific PREMs in GD have been published, neither PROMs for Spanish GD patients have been developed.

Methods

Two project coordinators selected key-points to be included in a PROMs/PREMs questionnaire, and the scientific committee and a group of expert patients contributed to the initial draft. Then, 9 meetings with experts were held to discuss controversial points. After, a questionnaire with 103 items regarding symptomatology, aspects of daily life and care experience was developed. Finally, it was conducted a Delphi survey among a multidisciplinary group of experts in GD.

Results

Consensus was reached on 85 out of the 103 items. Recommendations on PROMs and PREMs regarding symptomatology, aspects of daily life and care experience were obtained. Consensus was reached on the importance of considering fatigue, concentration problems, and communication issues in GD patients using 5-step analog scales. Panelists recommended asking GD patients about the impact on social functioning and work/school performance. Finally, consensus was reached on considering care experiences, such as treatment satisfaction, treatment interruptions or transitions and healthcare professionals involved in patient's management to perceive patient's perceptions.

Conclusion

This expert consensus may help developing GD-specific PROMs/PREMs for improving GD management. Properly developed and validated PROMs/PREMs may help decision-making, establishing patient-tailored therapeutic and follow-up goals.

查看原文本刊更多论文

确定西班牙戈谢病患者报告结果指标 (PROM) 和患者报告体验指标 (PREM)。

背景：患者报告的结果测量（PROMs）和患者报告的体验测量（PREMs）对于了解 GD 对生活质量的影响和患者对护理的看法至关重要，同时也能指导决策过程。然而，目前还没有专门针对 GD 的 PREMs 出版，也没有针对西班牙 GD 患者的 PROMs：方法：两名项目协调人选择了要纳入 PROMs/PREMs 问卷的关键点，科学委员会和一组患者专家对初稿做出了贡献。然后，与专家举行了 9 次会议，讨论有争议的问题。之后，制定了一份包含 103 个项目的调查问卷，内容涉及症状、日常生活方面和护理经验。最后，在一个多学科的广东专家小组中进行了德尔菲调查：结果：在 103 个项目中，有 85 个项目达成了共识。就症状学、日常生活和护理体验方面的 PROM 和 PREM 提出了建议。就使用五级模拟量表考虑广东话患者疲劳、注意力不集中和沟通问题的重要性达成了共识。专家组成员建议向广东籍患者询问其对社会功能和工作/学业表现的影响。最后，专家们就考虑护理经验达成共识，如治疗满意度、治疗中断或过渡以及参与患者管理的医护人员对患者感受的看法：结论：这一专家共识有助于开发针对广东的 PROMs/PREMs，以改善广东的管理。经适当开发和验证的 PROMs/PREMs 有助于决策、制定适合患者的治疗和随访目标。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Medicina Clinica 医学-医学：内科

CiteScore

3.10

自引率

5.10%

发文量

295

审稿时长

22 days

期刊介绍： Medicina Clínica, fundada en 1943, es una publicación quincenal dedicada a la promoción de la investigación y de la práctica clínica entre los especialistas de la medicina interna, así como otras especialidades. Son características fundamentales de esta publicación el rigor científico y metodológico de sus artículos, la actualidad de los temas y, sobre todo, su sentido práctico, buscando siempre que la información sea de la mayor utilidad en la práctica clínica.