Nalty Theresa, Patel Shalin S, Bird Justin E, Lewis Valerae O, Lin Patrick P
Department of Orthopaedic Oncology, MD Anderson Cancer Center, Houston, TX, USA.
The Learning Cancer Outcomes Research Program (LCORP) at MD Anderson Cancer Center, Houston, TX, USA.
Clin Orthop Relat Res. 2025 Jan 1;483(1):62-75. doi: 10.1097/CORR.0000000000003257. Epub 2024 Oct 4.
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.
QUESTIONS/PURPOSES: (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 (LEO).Level of Evidence Level II, diagnostic study.
目前用于骨肿瘤学的功能评估工具是冗长的调查问卷，这会导致患者出现调查疲劳，而且在辨别经常活动但无法进行剧烈活动的患者群体中的有意义差异方面能力不足。我们试图确定基于现有经过验证的调查问卷的更简短新颖工具是否能更好地捕捉骨肿瘤学患者样本中的差异。
问题/目的：（1）从下肢多伦多肢体挽救评分（TESS LE）的50项和下肢功能量表（LEFS）中得出的简洁固定项功能工具在敏感性和特异性方面能否表现出相似的反应性？（2）与TESS LE和LEFS相比，该简洁工具的精度和准确性如何？
功能结局数据在单一机构的纵向维护数据库中收集和保存。纳入本研究的患者需满足以下条件：（1）因影响下肢功能的骨或软组织肿瘤接受过肿瘤切除或非肿瘤性骨科手术（如骨关节炎的关节置换术）；（2）至少在两次门诊就诊时完成了LEFS、TESS LE和患者报告结局测量信息系统（PROMIS）全球健康工具。2014年9月至2022年4月期间，我们治疗了14234例原发性骨或软组织肉瘤、骨转移瘤或慢性癌症护理的骨科后遗症患者。约6%（14234例中的854例）因需要语言翻译而被排除。约2%（13380例中的278例）拒绝或无法参与。72%（13102例中的9433例）患者接受了下肢手术。其中，4%（9433例中的339例）患者在≥2次门诊就诊时完成了TESS LE、LEFS和PROMIS全球健康工具的第3项。在本研究的患者中，49%（339例中的167例）为女性，27%（339例中的93例）患有转移性癌。12%（339例中的41例）患者在研究期结束前死亡。Spearman等级相关、主成分分析（PCA）和项目反应理论（IRT）建模从TESS LE和LEFS中确定了14个具有高度区分性的项目。以从TESS LE和LEFS中得出并标准化为100%的14个项目的总分作为因变量进行多元线性逐步回归（MLSR）。MLSR的β系数用于得出14个项目中每个项目的权重。对具有10至17个变量的模型进行评估，以确保包含14个项目的模型在PCA、受试者工作特征（ROC）曲线和IRT建模标准方面符合最多的拟合标准。使用ROC评估缩短的14项调查问卷、30项TESS LE和20项LEFS的变化分数与PROMIS全球健康工具第3项的二分变化相比的反应性（敏感性和特异性）。评估从LEFS和TESS LE得出的14个项目的一致性（准确性和精度）。
缩短的14项调查问卷、TESS LE和LEFS对PROMIS全球健康工具（第3项）标准目标的反应性（敏感性和特异性）相似，ROC曲线下面积（AUC）范围为0.62至0.65。14项调查问卷对TESS LE的反应性显示敏感性为96%，特异性为90%，AUC为0.98（p < 0.001）。14个项目对LEFS的反应性显示敏感性为95%，特异性为86%，AUC为0.96。14个项目对TESS LE的有效性通过一致性来衡量，精度为0.98，准确性为0.97。14个项目与LEFS的一致性显示精度为0.98，准确性为0.83。
从TESS LE和LEFS结局测量中患者报告的反应得出的简洁的14个项目与原始的TESS LE和LEFS在下肢骨科手术（肿瘤性和非肿瘤性适应证）后癌症患者中的反应性（敏感性和特异性）相似。这简洁的14个项目与TESS LE和LEFS具有相似的能力，能够向临床医生或患者说明他们与其他患者相比的功能状况。这14个项目比TESS LE和LEFS总共50个项目更短，同时保留了描述骨肿瘤学患者广泛下肢功能的能力。我们将这14项调查问卷命名为下肢肿瘤功能评估工具（LEO）。证据水平：II级，诊断性研究。
