Department of Medical Oncology, IRCCS Ospedale San Raffaele Scientific Institute, Milan, Italy.
Bristol Myers Squibb, Princeton, NJ, USA.
Ann Surg Oncol. 2021 Nov;28(12):7545-7554. doi: 10.1245/s10434-021-09816-z. Epub 2021 Apr 3.
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.
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.
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.
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.
ClinicalTrials.gov NCT01964430; Eudra CT 2013-003398-91.
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。
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。
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）。
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。
ClinicalTrials.gov NCT01964430；Eudra CT 2013-003398-91。
