简明的患者报告结果测量能否为癌症患者下肢手术后的身体功能提供有效的测量?
Does a Concise Patient-reported Outcome Measure Provide a Valid Measure of Physical Function for Cancer Patients After Lower Extremity Surgery?
发表日期:2024 Oct 04
作者:
Theresa Nalty, Shalin S Patel, Justin E Bird, Valerae O Lewis, Patrick P Lin
来源:
CLINICAL ORTHOPAEDICS AND RELATED RESEARCH
摘要:
目前用于骨科肿瘤学的功能评估工具是长时间的调查,这会导致患者的调查疲劳,但缺乏辨别经常移动但无法进行剧烈活动的患者群体中有意义的差异的能力。我们试图确定一种基于现有的、经过验证的调查的更简短、新颖的工具是否可以更好地捕捉骨科肿瘤患者样本中的差异。(1) 能否从多伦多肢体挽救评分中的 50 个项目中派生出一个简洁的固定项目功能工具下肢功能量表 (TESS LE) 和下肢功能量表 (LEFS) 在敏感性和特异性方面表现出相似的反应性? (2) 与 TESS LE 和 LEFS 相比,简明工具的精度和准确度如何?功能结果数据被收集并保存在单个机构的纵向维护数据库中。如果患者符合以下条件,则纳入研究:(1) 他们因影响下肢功能的骨或软组织肿瘤而接受过肿瘤切除或非肿瘤骨科手术(例如,骨关节炎的关节置换术),并且 (2) 他们已完成 LEFS 、TESS LE 和患者报告结果测量信息系统 (PROMIS) 全球健康工具至少两次就诊。 2014 年 9 月至 2022 年 4 月期间,我们治疗了 14,234 名原发性骨或软组织肉瘤、骨转移性疾病或慢性癌症护理的骨科后遗症患者。大约 6%(14,234 人中的 854 人)因需要语言翻译而被排除在外。大约 2%(13,380 人中的 278 人)拒绝或无法参加。 72%(13,102 名患者中的 9433 名)接受了下肢手术。其中,4%(9433 名患者中的 339 名)的患者在 ≥ 2 次就诊时完成了 TESS LE、LEFS 和 PROMIS 全球健康工具的第 3 项。在当前研究的患者中,49%(339 名患者中的 167 名)是女性,27%(339 名患者中的 93 名)患有转移癌。百分之十二(339 名患者中的 41 名)在研究期结束前死亡。 Spearman 排序相关、主成分分析 (PCA) 和项目响应理论 (IRT) 建模从 TESS LE 和 LEFS 中识别出 14 个高度区分的项目。进行多元线性逐步回归 (MLSR),因变量是从 TESS LE 和 LEFS 得出的 14 个项目的总分,并标准化为 100 的百分比。MLSR 的 beta 系数用于得出每个项目的权重14 项中。对具有 10 至 17 个变量的模型进行评估,以确保具有 14 个项目的模型满足 PCA、受试者工作特征 (ROC) 曲线和 IRT 建模标准的大部分拟合标准。与 PROMIS 全球健康工具第 3 项的二分变化相比,缩短的 14 项调查、30 项 TESS LE 和 20 项 LEFS 中的变化分数的响应性(敏感性和特异性)通过以下方法进行评估: ROC。对源自 LEFS 和 TESS LE 的 14 个项目的一致性(准确度和精密度)进行了评估。缩短的 14 项目调查、TESS LE 和 LEFS 对 PROMIS 标准目标的响应性(敏感性和特异性)全球健康工具(第 3 项)类似,ROC 曲线的曲线下面积 (AUC) 范围为 0.62 至 0.65。 TESS LE 的 14 项调查的响应度显示,敏感性为 96%,特异性为 90%,AUC 为 0.98(p < 0.001)。 14 个项目对 LEFS 的敏感性为 95%,特异性为 86%,AUC 为 0.96。 TESS LE 的 14 个项目的效度通过一致性来衡量,精密度为 0.98,准确度为 0.97。 14 个项目与 LEFS 的一致性显示精确度为 0.98,准确度为 0.83。从 TESS LE 和 LEFS 结果测量中患者报告的反应得出的简明 14 个项目显示出与原始 TESS LE 和 LEFS 相似的反应性(敏感性和特异性)。针对肿瘤和非肿瘤适应症进行下肢骨科手术后的癌症患者进行 LEFS。简明的 14 项与 TESS LE 和 LEFS 具有类似的能力,可以告诉临床医生或患者与其他患者相比,他们的功能如何。这 14 项比 TESS LE 和 LEFS 的 50 项之和要短,同时保留了描述骨科肿瘤患者广泛的下肢功能的能力。我们将这项包含 14 项的调查命名为下肢肿瘤功能评估工具。证据级别 II 级,诊断研究。版权所有 © 2024,骨与关节外科医生协会。
Current functional assessment tools for orthopaedic oncology are long surveys that contribute to patients' survey fatigue and yet lack the ability to discern meaningful differences in a patient population that is often mobile but unable to perform strenuous activities. We sought to determine whether a shorter, novel tool based on existing, validated surveys could better capture differences in a sample of orthopaedic oncology patients.(1) Can a concise fixed-item functional tool derived from the 50 items in the Toronto Extremity Salvage Score for the lower extremity (TESS LE) and the Lower Extremity Functional Scale (LEFS) demonstrate similar responsiveness in terms of sensitivity and specificity? (2) What is the precision and accuracy of the concise tool compared with the TESS LE and LEFS?Functional outcome data were collected and maintained in a longitudinally maintained database at a single institution. Patients were included in the study if (1) they had undergone a tumor excision or a nononcologic orthopaedic procedure (for example, arthroplasty for osteoarthritis) for a bone or soft tissue tumor affecting lower extremity function, and (2) they had completed the LEFS, TESS LE, and Patient-Reported Outcomes Measurement Information System (PROMIS) global health tool on at least two clinic visits. Between September 2014 and April 2022, we treated 14,234 patients for primary bone or soft tissue sarcoma, metastatic disease to bone, or orthopaedic sequelae of chronic cancer care. Approximately 6% (854 of 14,234) were excluded due to the need of a language translator. Approximately 2% (278 of 13,380) refused or were unable to participate. Seventy-two percent (9433 of 13,102) of the patients had an operation on a lower extremity. Of these, 4% (339 of 9433) of the patients completed the TESS LE, LEFS, and Item 3 of the PROMIS global health tool on ≥ 2 clinic visits. Of the patients in the current study, 49% (167 of 339) were women, and 27% (93 of 339) had metastatic carcinoma. Twelve percent (41 of 339) of the patients died before the end of the study period. Spearman rank-order correlation, principal component analysis (PCA), and item response theory (IRT) modeling identified 14 highly discriminating items from the TESS LE and LEFS. Multiple linear stepwise regression (MLSR) was performed with the dependent variable being the summary score of the 14 items derived from the TESS LE and LEFS and standardized to a percentage of 100. The beta coefficient from the MLSR was used to derive a weight for each of the 14 items. Evaluation of the model with 10 to 17 variables was performed to ensure that the model with the 14 items met the most criteria for fit with the PCA, the receiver operating characteristic (ROC) curve, and the IRT modeling criteria. The responsiveness (sensitivity and specificity) of the change scores in the shortened 14-item survey, the 30-item TESS LE, and the 20-item LEFS as compared with the dichotomized changes in Item 3 of the PROMIS global health tool was evaluated using ROCs. The concordance (accuracy and precision) of the 14 items derived from the LEFS and TESS LE was evaluated.The responsiveness (sensitivity and specificity) of the shortened 14-item survey, the TESS LE, and the LEFS to the criterion target of the PROMIS global health tool (Item 3) was similar, with areas under the curve (AUCs) ranging from 0.62 to 0.65 for the ROC curves. The responsiveness of the 14-item survey to the TESS LE showed sensitivity of 96% and specificity of 90%, with an AUC of 0.98 (p < 0.001). The responsiveness of the 14 items to the LEFS showed sensitivity of 95% and specificity of 86%, with an AUC of 0.96. The validity of the 14 items to the TESS LE was measured by concordance, with a precision of 0.98 and an accuracy of 0.97. Concordance of the 14 items to the LEFS showed a precision of 0.98 and accuracy of 0.83.The concise 14 items derived from patient-reported responses in the TESS LE and LEFS outcome measures showed similar responsiveness (sensitivity and specificity) as the original TESS LE and LEFS for cancer patients after lower extremity orthopaedic surgery performed for oncologic and nononcologic indications. The concise 14 items have a similar ability to the TESS LE and LEFS to tell the clinician or patient how they are functioning compared with other patients. These 14 items are shorter than the combined 50 items of the TESS LE and LEFS while retaining the capacity to describe a broad range of lower extremity function for orthopaedic oncology patients. We have named the 14-item survey the Lower Extremity Oncology Functional Assessment Tool.Level of Evidence Level II, diagnostic study.Copyright © 2024 by the Association of Bone and Joint Surgeons.