AstraZeneca, Oncology Digital Health R&D, Gaithersburg, MD, USA.
Center for Health Measurement, Department of Population Health Sciences, Duke University School of Medicine, Durham, NC, USA.
Qual Life Res. 2024 Oct;33(10):2869-2880. doi: 10.1007/s11136-024-03740-x. Epub 2024 Jul 24.
The Functional Assessment of Cancer Therapy item (FACT-GP5) has the potential to provide an understanding of global treatment tolerability from the patient perspective. Longitudinal evaluations of the FACT-GP5 and challenges posed by data missing-not-at-random (MNAR) have not been explored. Robustness of the FACT-GP5 to missing data assumptions and the responsiveness of the FACT-GP5 to key side-effects are evaluated.
In a randomized, double-blind study (NCT00065325), postmenopausal women (n = 618) with hormone receptor-positive (HR+), advanced breast cancer received either fulvestrant or exemestane and completed FACT measures monthly for seven months. Cumulative link mixed models (CLMM) were fit to evaluate: (1) the trajectory of the FACT-GP5 and (2) the responsiveness of the FACT-GP5 to CTCAE grade, Eastern Cooperative Oncology Group (ECOG) Performance Status scale, and key side-effects from the FACT. Sensitivity analyses of the missing-at-random (MAR) assumption were conducted.
Odds of reporting worse side-effect bother increased over time. There were positive within-person relationships between level of side-effect bother (FACT-GP5) and severity of other FACT items, as well as ECOG performance status and Common Terminology Criteria for Adverse Events (CTCAE) grade. The number of missing FACT-GP5 assessments impacted the trajectory of the FACT-GP5 but did not impact the relationships between the FACT-GP5 and other items (except for nausea [FACT-GP2]).
Results support the responsiveness of the FACT-GP5. Generally speaking, the responsiveness of the FACT-GP5 is robust to missing assessments. Missingness should be considered, however, when evaluating change over time of the FACT-GP5.
NCT00065325.
癌症治疗功能评估量表-患者报告结局（FACT-GP5）有潜力从患者角度了解整体治疗耐受性。尚未探讨 FACT-GP5 的纵向评估和数据缺失非随机（MNAR）带来的挑战。评估 FACT-GP5 对缺失数据假设的稳健性和 FACT-GP5 对关键副作用的反应性。
在一项随机、双盲研究（NCT00065325）中，618 例激素受体阳性（HR+）、晚期乳腺癌绝经后女性接受氟维司群或依西美坦治疗，并在 7 个月内每月完成 FACT 量表评估。采用累积链接混合模型（CLMM）评估：（1）FACT-GP5 的轨迹；（2）FACT-GP5 对 CTCAE 分级、东部肿瘤协作组（ECOG）体能状态量表和 FACT 关键副作用的反应性。对随机缺失（MAR）假设进行了敏感性分析。
报告更严重副作用困扰的几率随时间增加而增加。副作用困扰程度（FACT-GP5）与其他 FACT 项目的严重程度以及 ECOG 体能状态和不良事件通用术语标准（CTCAE）分级之间存在个体内的正相关关系。FACT-GP5 缺失评估的数量会影响 FACT-GP5 的轨迹，但不会影响 FACT-GP5 与其他项目之间的关系（除了恶心[FACT-GP2]）。
结果支持 FACT-GP5 的反应性。一般来说，FACT-GP5 的反应性对缺失评估具有稳健性。然而，在评估 FACT-GP5 随时间的变化时，应考虑缺失情况。
NCT00065325。
2003 年。
