评估 EORTC QLQ-PAN26 模块中用于评估接受手术切除胰腺腺癌患者健康相关生活质量的最小重要差异和应答者定义。
Evaluation of Minimal Important Difference and Responder Definition in the EORTC QLQ-PAN26 Module for Assessing Health-Related Quality of Life in Patients with Surgically Resected Pancreatic Adenocarcinoma.
机构信息
Department of Medical Oncology, IRCCS Ospedale San Raffaele Scientific Institute, Milan, Italy.
Bristol Myers Squibb, Princeton, NJ, USA.
出版信息
Ann Surg Oncol. 2021 Nov;28(12):7545-7554. doi: 10.1245/s10434-021-09816-z. Epub 2021 Apr 3.
BACKGROUND
Although the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-PAN26 is widely used to assess health-related quality of life (HRQoL), its group-level minimal important difference (MID) and individual-level responder definition (RD) are not established; we calculated MID and RD using HRQoL data from the APACT trial in patients with surgically resected pancreatic cancer who received adjuvant chemotherapy.
METHODS
HRQoL was assessed using EORTC QLQ-C30 and QLQ-PAN26 at baseline, during treatment, at end of treatment, and during follow-up. Distribution-based MIDs were estimated using 0.5 × baseline standard deviation (SD) and reliability-based (intraclass correlation) standard error of measurement (SEM). Anchor-based MIDs and RDs (anchor, QLQ-C30 overall health) were estimated using a linear mixed model.
RESULTS
Overall, 772 patients completed the baseline assessment. Distribution-based MIDs (0.5 × SD) for QLQ-PAN26 scales ranged from 12 to 13, except hepatic symptoms (≈8), pancreatic pain (≈10), and sexual dysfunction (≈17); those for stand-alone items ranged from 12 to 16. The SEM values were similar. Among scales/items sufficiently correlated (r > 0.30) with the anchor, MIDs ranged from 5 to 9. Within-patient QLQ-PAN26 RD estimates varied by direction (deterioration vs. improvement) and scale/item, but all values were lower than the true possible within-patient change (e.g. 16.7 points for a two-item scale) given a one-category change on the raw scale.
CONCLUSIONS
Compared with distribution-based MIDs, anchor-based MIDs were twice as sensitive in detecting group-level changes in QLQ-PAN26 scales/items. For interpreting clinically meaningful change, RDs cannot be less than the true minimum of the scale. The group-level MID may help clinicians/researchers interpret HRQoL changes.
TRIAL REGISTRATION
ClinicalTrials.gov NCT01964430; Eudra CT 2013-003398-91.
背景
尽管欧洲癌症研究与治疗组织（EORTC）的 QLQ-PAN26 广泛用于评估健康相关生活质量（HRQoL），但其组级最小重要差异（MID）和个体级应答者定义（RD）尚未确定；我们使用接受辅助化疗的手术切除胰腺癌患者的 APACT 试验中的 HRQoL 数据计算了 MID 和 RD。
方法
在基线、治疗期间、治疗结束时和随访期间，使用 EORTC QLQ-C30 和 QLQ-PAN26 评估 HRQoL。使用 0.5×基线标准差（SD）和基于可靠性（内类相关）测量误差（SEM）的分布型 MID 进行估计。基于锚定的 MID 和 RD（锚定，QLQ-C30 总体健康）使用线性混合模型进行估计。
结果
总体而言，772 例患者完成了基线评估。除了肝症状（≈8）、胰腺痛（≈10）和性功能障碍（≈17）外，QLQ-PAN26 量表的分布型 MID（0.5×SD）范围为 12 到 13；单项 MID 范围为 12 到 16。SEM 值相似。在与锚定足够相关（r>0.30）的量表/项目中，MID 范围为 5 到 9。患者内 QLQ-PAN26 RD 估计值因方向（恶化与改善）和量表/项目而异，但所有值均低于原始量表上一个类别变化时的真实患者内变化（例如，两个项目量表为 16.7 分）。
结论
与分布型 MID 相比，基于锚定的 MID 在检测 QLQ-PAN26 量表/项目的组级变化方面更加敏感。为了解释有临床意义的变化，RD 不能小于量表的真实最小值。组级 MID 可能有助于临床医生/研究人员解释 HRQoL 变化。
试验注册
ClinicalTrials.gov NCT01964430；Eudra CT 2013-003398-91。
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