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嵌合抗原受体 T 细胞(CAR-T)疗法治疗患者中患者报告结局(PROs)采集数字系统的开发和可行性测试的混合方法研究方案(PRO-CAR-T 研究)。

Protocol for a mixed-methods study to develop and feasibility test a digital system for the capture of patient-reported outcomes (PROs) in patients receiving chimeric antigen receptor T-cell (CAR-T) therapies (the PRO-CAR-T study).

机构信息

Centre for Patient Reported Outcome Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK

National Institute of Health and Care Research (NIHR) Blood and Transplant Research Unit (BTRU) in Precision Cellular Therapeutics, Birmingham, UK.

出版信息

BMJ Open. 2024 Mar 29;14(3):e085392. doi: 10.1136/bmjopen-2024-085392.


DOI:10.1136/bmjopen-2024-085392
PMID:38553074
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10982800/
Abstract

INTRODUCTION: Chimeric antigen receptor (CAR) T-cell therapies are novel, potentially curative therapies for haematological malignancies. CAR T-cell therapies are associated with severe toxicities, meaning patients require monitoring during acute and postacute treatment phases. Electronic patient-reported outcomes (ePROs), self-reports of health status provided via online questionnaires, can complement clinician observation with potential to improve patient outcomes. This study will develop and evaluate feasibility of a new ePRO system for CAR-T patients in routine care. METHODS AND ANALYSIS: Multiphase, mixed-methods study involving multiple stakeholder groups (patients, family members, carers, clinicians, academics/researchers and policy-makers). The intervention development phase comprises a Delphi study to select PRO measures for the digital system, a codesign workshop and consensus meetings to establish thresholds for notifications to the clinical team if a patient reports severe symptoms or side effects. Usability testing will evaluate how users interact with the digital system and, lastly, we will evaluate ePRO system feasibility with 30 CAR-T patients (adults aged 18+ years) when used in addition to usual care. Feasibility study participants will use the ePRO system to submit self-reports of symptoms, treatment tolerability and quality of life at specific time points. The CAR-T clinical team will respond to system notifications triggered by patients' submitted responses with actions in line with standard clinical practice. Feasibility measures will be collected at prespecified time points following CAR T-cell infusion. A qualitative substudy involving patients and clinical team members will explore acceptability of the ePRO system. ETHICS AND DISSEMINATION: Favourable ethical opinion was granted by the Health and Social Care Research Ethics Committee B(HSC REC B) (ref: 23/NI/0104) on 28 September 2023. Findings will be submitted for publication in high-quality, peer-reviewed journals. Summaries of results, codeveloped with the Blood and Transplant Research Unit Patient and Public Involvement and Engagement group, will be disseminated to all interested groups. TRIAL REGISTRATION NUMBER: ISCTRN11232653.

摘要

简介:嵌合抗原受体 (CAR) T 细胞疗法是治疗血液系统恶性肿瘤的新型、潜在治愈性疗法。CAR T 细胞疗法与严重的毒性相关,这意味着患者在急性和急性后治疗阶段需要接受监测。电子患者报告结局(ePRO),即通过在线问卷提供的健康状况自我报告,可以补充临床医生的观察,有可能改善患者的结局。本研究将开发并评估一种新的 CAR-T 患者常规护理中 ePRO 系统的可行性。

方法和分析:多阶段、混合方法研究,涉及多个利益相关者群体(患者、家属、照顾者、临床医生、学者/研究人员和政策制定者)。干预措施的开发阶段包括德尔菲研究,以选择数字系统的 PRO 措施;代码设计研讨会和共识会议,以确定如果患者报告严重症状或副作用,向临床团队发出通知的阈值。可用性测试将评估用户与数字系统的交互方式;最后,我们将在 30 名 CAR-T 患者(年龄 18 岁以上的成年人)中评估 ePRO 系统的可行性,该系统在常规护理的基础上使用。可行性研究参与者将使用 ePRO 系统在特定时间点提交症状、治疗耐受性和生活质量的自我报告。CAR-T 临床团队将根据患者提交的回复触发系统通知,采取符合标准临床实践的行动。将在 CAR T 细胞输注后预先指定的时间点收集可行性措施。一项涉及患者和临床团队成员的定性子研究将探索 ePRO 系统的可接受性。

伦理和传播:2023 年 9 月 28 日,健康和社会保健研究伦理委员会 B(HSC REC B)(参考号:23/NI/0104)授予了有利的伦理意见。研究结果将提交给高质量的同行评审期刊发表。与血液和移植研究单位患者和公众参与和参与小组共同制定的结果摘要将分发给所有感兴趣的群体。

试验注册号:ISCTRN11232653。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/49b8/10982800/fa6608268e89/bmjopen-2024-085392f01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/49b8/10982800/fa6608268e89/bmjopen-2024-085392f01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/49b8/10982800/fa6608268e89/bmjopen-2024-085392f01.jpg

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[1]
Protocol for a mixed-methods study to develop and feasibility test a digital system for the capture of patient-reported outcomes (PROs) in patients receiving chimeric antigen receptor T-cell (CAR-T) therapies (the PRO-CAR-T study).

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引用本文的文献

[1]
Measure selection for an electronic patient-reported outcome (ePRO) system for CAR T-cell therapy patients: a modified Delphi consensus study.

EClinicalMedicine. 2025-5-28

本文引用的文献

[1]
Chimeric Antigen Receptor T-cell Therapy in Hematologic Malignancies and Patient-reported Outcomes: A Scoping Review.

Hemasphere. 2022-12-1

[2]
Longitudinal Collection of Patient-Reported Outcomes and Activity Data during CAR-T Therapy: Feasibility, Acceptability, and Data Visualization.

Cancers (Basel). 2022-5-31

[3]
The Unique Symptom Burden of Patients Receiving CAR T-Cell Therapy.

Semin Oncol Nurs. 2021-12

[4]
Key methodological considerations for usability testing of electronic patient-reported outcome (ePRO) systems.

Qual Life Res. 2019-10-18

[5]
Fit Between Individuals, Tasks, Technology, and Environment (FITTE) Framework: A Proposed Extension of FITT to Evaluate and Optimise Health Information Technology Use.

Stud Health Technol Inform. 2019-8-21

[6]
The Other Side of CAR T-Cell Therapy: Cytokine Release Syndrome, Neurologic Toxicity, and Financial Burden.

Am Soc Clin Oncol Educ Book. 2019-1

[7]
Risks and Benefits of Chimeric Antigen Receptor T-Cell (CAR-T) Therapy in Cancer: A Systematic Review and Meta-Analysis.

Transfus Med Rev. 2019-2-14

[8]
Maximising the impact of patient reported outcome assessment for patients and society.

BMJ. 2019-1-24

[9]
User-Innovated eHealth Solutions for Service Delivery to Older Persons With Hearing Impairment.

Am J Audiol. 2018-11-19

[10]
How do patient reported outcome measures (PROMs) support clinician-patient communication and patient care? A realist synthesis.

J Patient Rep Outcomes. 2018-9-15

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