确定不良事件通用术语标准（PRO-CTCAE®）患者报告结果版中有意义的个人水平恶化变化阈值。

IF 3.3 3区医学 Q1 HEALTH CARE SCIENCES & SERVICES

Quality of Life Research Pub Date : 2024-11-06 DOI:10.1007/s11136-024-03819-5

Minji K Lee, Sandra A Mitchell, Ethan Basch, Gina L Mazza, Blake T Langlais, Gita Thanarajasingam, Brenda F Ginos, Lauren Rogak, Eric A Meek, Jennifer Jansen, Allison M Deal, Philip Carr, Victoria S Blinder, Mattias Jonsson, Gita N Mody, Tito R Mendoza, Antonia V Bennett, Deborah Schrag, Amylou C Dueck

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引用次数: 0

摘要

背景：我们从选定的患者报告结果版不良事件通用术语标准（PRO-CTCAE®）项目及其综合评分中得出了有意义的个人水平的恶化变化阈值：我们使用了两个数据源，一个是 PRO-TECT 试验（Alliance AFT-39），该试验收集了 26 个美国社区肿瘤诊所的晚期癌症成人患者的 PRO-CTCAE 数据；另一个是 PRO-CTCAE 验证研究，该研究收集了 9 个美国癌症中心或社区肿瘤诊所接受化疗或放疗的成人患者的 PRO-CTCAE 数据。这两项研究都采用了选定的 PRO-CTCAE 项目和 EORTC QLQ-C30 量表。从概念上讲，相关的 QLQ-C30 领域被用作锚，以估算相应的 PRO-CTCAE 项目及其综合评分恶化的有意义变化阈值。与QLQ-C30量表相关性ǀρǀ≥ 0.30的项目或综合得分被纳入其中。在相应的QLQ-C30量表上达到或超过10分恶化的患者，其PRO-CTCAE评分和复合评分的变化将被估算。在 PRO-TECT 中，计算基线与 3 个月时间点之间的变化分数；在 PRO-CTCAE 验证研究中，计算基线与 1 到 7 周后的单次随访之间的变化分数。对于每个 PRO-CTCAE 项目，变化分值范围为-4 到 4；对于综合项目，变化分值范围为-3 到 3：在 PRO-TECT 和验证研究中，分别有 406 名和 792 名患者获得了 QLQ-C30 和 PRO-CTCAE 的变化评分。就 QLQ-C30 量表而言，在 PRO-TECT 数据中，QLQ-C30 恶化 10 分或以上的患者比例为 15% 至 30%，在验证数据中为 13% 至 34%。在所有 PRO-CTCAE 项目中，PRO-TECT 数据中基于锚点的恶化有意义变化估计值从 0.05 到 0.30（平均 0.19）不等，验证数据中从 0.19 到 0.53（平均 0.36）不等。对于复合材料，PRO-TECT 数据中的误差范围为 0.06 至 0.27（平均值为 0.17），验证数据中的误差范围为 0.22 至 0.51（平均值为 0.37）：结论：在这两个数据集中，所有项目和综合评分恶化的最小有意义个体水平变化阈值均为1分：NCT03249090（AFT-39）、NCT02158637（MC1091）。

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Identification of meaningful individual-level change thresholds for worsening on the patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE®).

Background: We derived meaningful individual-level change thresholds for worsening in selected patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE®) items and their composite scores.

Methods: We used two data sources, the PRO-TECT trial (Alliance AFT-39) that collected PRO-CTCAE data from adults with advanced cancer at 26 United States (U.S.) community oncology practices and the PRO-CTCAE validation study that collected PRO-CTCAE data from adults undergoing chemotherapy or radiation therapy at nine U.S. cancer centers or community oncology practices. Both studies administered selected PRO-CTCAE items and EORTC QLQ-C30 scales. Conceptually, relevant QLQ-C30 domains were used as anchors to estimate meaningful change thresholds for deterioration in corresponding PRO-CTCAE items and their composite scores. Items or composites with ǀρǀ ≥ 0.30 correlation with QLQ-C30 scales were included. Changes in PRO-CTCAE scores and composites were estimated for patients who met or exceeded a 10-point deterioration on the corresponding QLQ-C30 scale. Change scores were computed between baseline and the 3-month timepoint in PRO-TECT, and in the PRO-CTCAE validation study between baseline and a single follow-up visit that occurred between 1 and 7 weeks later. For each PRO-CTCAE item, change scores could range from - 4 to 4; for a composite, change scores could range from - 3 to 3.

Results: Change scores in QLQ-C30 and PRO-CTCAE were available in 406 and 792 patients in PRO-TECT and the validation study, respectively. Across QLQ-C30 scales, the proportion of patients with a 10-point or greater worsening on QLQ-C30 ranged from 15 to 30% in the PRO-TECT data and 13% to 34% in the validation data. Across PRO-CTCAE items, anchor-based meaningful change estimates for deterioration ranged from 0.05 to 0.30 (mean 0.19) in the PRO-TECT data and from 0.19 to 0.53 (mean 0.36) in the validation data. For composites, they ranged from 0.06 to 0.27 (mean 0.17) in the PRO-TECT data and 0.22 to 0.51 (mean 0.37) in the validation data.

Conclusion: In both datasets, the minimal meaningful individual-level change threshold for worsening was one point for all items and composite scores.

Clinicaltrials: gov: NCT03249090 (AFT-39), NCT02158637 (MC1091).

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来源期刊

Quality of Life Research 医学-公共卫生、环境卫生与职业卫生

CiteScore

6.50

自引率

8.60%

发文量

224

审稿时长

3-8 weeks

期刊介绍： Quality of Life Research is an international, multidisciplinary journal devoted to the rapid communication of original research, theoretical articles and methodological reports related to the field of quality of life, in all the health sciences. The journal also offers editorials, literature, book and software reviews, correspondence and abstracts of conferences. Quality of life has become a prominent issue in biometry, philosophy, social science, clinical medicine, health services and outcomes research. The journal''s scope reflects the wide application of quality of life assessment and research in the biological and social sciences. All original work is subject to peer review for originality, scientific quality and relevance to a broad readership. This is an official journal of the International Society of Quality of Life Research.