严重肿瘤患者脱落的特征和患者报告结局：一项探索性研究。
Characteristics and patient-reported outcomes associated with dropout in severely affected oncological patients: an exploratory study.
机构信息
Berlin Institute of Health at Charité -Universitätsmedizin Berlin, Charitéplatz 1, 10117, Berlin, Germany.
Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Institute of Biometry and Clinical Epidemiology, Charitéplatz 1, 10117, Berlin, Germany.
出版信息
BMC Med Res Methodol. 2021 Apr 20;21(1):77. doi: 10.1186/s12874-021-01259-0.
BACKGROUND
Patient-reported outcome measures (PROMs) are commonly-used surrogates for clinical outcomes in cancer research. When researching severe diseases such as cancer, it is difficult to avoid the problem of incomplete questionnaires from drop-outs or missing data from patients who pass away during the observation period. The aim of this exploratory study was to explore patient characteristics and the patient-reported outcomes associated with the time-to-dropout.
METHODS
In an Oncological Social Care Project (OSCAR) study, the condition of the participants was assessed four times within 12 months (t0: baseline, t1: 3 months, t2: 6 months, and t3: 12 months) by validated PROMs. We performed competing-risk regressions based on Fine and Gray's proportional sub-distribution hazards model for exploring factors associated with time-to-dropout. Death was considered a competing risk.
RESULTS
Three hundred sixty-two participants were analyzed in the study. 193 (53.3%) completed a follow-up after 12 months, 67 (18.5%) patients dropped out, and 102 patients (28.2%) died during the study period. Poor subjective social support was related to a higher risk of drop-out (SHR = 2.10; 95%CI: 1.01-4.35). Lower values in health-related quality of life were related to drop-out and death. The sub-scales global health status/QoL, role functioning, physical functioning, and fatigue symptom in the EORTC QLQ-C30 were key characteristics of early drop-out.
CONCLUSION
Severely affected cancer patients with poor social support and poor quality of life seem more likely to drop out of studies than patients with higher levels of social support and a better quality of life. This should be considered when planning studies to assess advanced cancer patients. Methods of close continued monitoring should be actively used when patient experiences a substantial deterioration in their health-related quality of life and symptoms during the study. Results for such studies have to be interpreted with caution in light of specific drop-out mechanisms.
TRIAL REGISTRATION
OSCAR study was registered to the German Clinical Trials Register (DRKS-ID: DRKS00013640 ). Registered 29 December 2017.
背景
患者报告结局测量（PROMs）是癌症研究中常用的临床结局替代指标。在研究癌症等严重疾病时，由于患者在观察期内退出或死亡而导致问卷不完整或数据缺失的问题难以避免。本探索性研究旨在探讨与退出时间相关的患者特征和患者报告结局。
方法
在一项肿瘤社会关怀项目（OSCAR）研究中，通过验证后的 PROMs 在 12 个月内对参与者的状况进行了四次评估（t0：基线，t1：3 个月，t2：6 个月，t3：12 个月）。我们基于 Fine 和 Gray 的比例亚分布风险模型进行竞争风险回归，以探索与退出时间相关的因素。死亡被视为竞争风险。
结果
在这项研究中分析了 362 名参与者。193 名（53.3%）患者在 12 个月后完成了随访，67 名（18.5%）患者退出，102 名（28.2%）患者在研究期间死亡。主观社会支持较差与退出风险增加相关（SHR=2.10；95%CI：1.01-4.35）。健康相关生活质量较低与退出和死亡相关。EORTC QLQ-C30 的全球健康状况/QoL、角色功能、身体功能和疲劳症状等子量表是早期退出的关键特征。
结论
与社会支持较高和生活质量较好的患者相比，社会支持较差和生活质量较低的严重癌症患者似乎更有可能退出研究。在计划评估晚期癌症患者的研究时应考虑到这一点。当患者的健康相关生活质量和症状在研究期间出现实质性恶化时，应积极使用密切持续监测的方法。鉴于特定的退出机制，必须谨慎解释此类研究的结果。
试验注册
OSCAR 研究在德国临床试验注册处（DRKS-ID：DRKS00013640）注册。于 2017 年 12 月 29 日注册。
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